Tuesday, 8 December 2015

Cystic Fibrosis & Fitness: Part 3

I decided to quit my gyms.

Yes you read right...gyms (as in plural).

I was a member of Nuffield gym in Manchester at the Printworks as well as my local gym, LiveWire.

Though fear not, I am now a member of Pure gym.

The difference?

Per year I am saving £502.92 on gym membership.

The main difference between Pure and the other gyms is no pool, which I rarely used at Nuffield and never used at all at LiveWire.

What Pure lacks pool wise certainly makes up for in gym equipment by the quality, quantity and variety. Considering it costs considerably less than the other gyms combined, you certainly get your money's worth.

Part of the money saved has gone towards a Personal Trainer. His name is Samson, whose profile is on the Altrincham page of the Pure Gym website.

One thing I like about Samson is he specialises in biomechanics and learning I have CF has sparked an interest with him. He certainly researched what CF is and what makes it tick, which he took into consideration when making my workout program.

He has given me a really good program to work on, which is more vigorous than what I had.

So what is the difference between my previous program and the one I have now?

My previous program saw me doing 4 sets of 8-12 reps of each exercise with 2 minutes rest between sets as shown in my previous fitness blog.

In this program I warm up with cardio, do 3 super sets and finish with core exercises and stretches.

For those who don't know what a super set is, I do 4 sets of 12 reps of exercises of 2 different muscle groups one after the other with a 45 seconds rest.

Example of a super set:
  • 12 reps chest press machine
  • 12 reps over head extension
  • 45 second rest
  • repeat another 3 times
Sounds easy? Compare this to the previous technique, you'll realise this is more vigorous and works the muscles more. Especially with the 45 seconds rest rather than the 2 minutes as previous.

After each workout I've worked up a sweat whereby I have to shower before I leave.

If anything I've surprised myself as I didn't realise how strong I was until I started training with my Personal Trainer.

Even he is impressed with my form.

It's great to have once more a Personal Trainer there to encourage and motivate.

Monday, 16 November 2015

QBW251 - Day 28: second to last

Today was my second to last visit to the hospital in regards to the drug trial.

The visit consisted of:
  • Body weight/BMI
  • Vital signs
  • Body temperature
  • Urine collection
  • Blood sampling
  • Lung function
  • Sputum sampling
  • Sweat chloride assessment
  • Pulse oximetry
  • CFQ-R questionnaire
In 2 weeks time I will be attending my last visit to the hospital.

This has been what feels like a short journey for me. Simply because I'm a very busy man and time flies when you're busy.

Though as I said, I'm pretty sure there will be more to say on this trial in the months to come.

As an interesting last word to today's news, through making friends with many members of the CF community around the world, I've had many good conversations with people who ask how the trial is going.

One conversation lead to the interesting mention that for CF people with the F508del mutation, there is a drug on the market called Orkambi.

Information about this drug can be found on this website: www.orkambi.com

There is a video on the website that elaborates on what this drug does.

Sunday, 8 November 2015

QBW251 - Day 17: getting more interesting by the minute

Thursday, 5 November 2015
You may recall that on days 2, 3 and 4 of taking QBW251 I started to have some form of effect in one way or the other.

Whether this was side effects such as feeling sick or having headaches, or other effects such as having the feeling my sinuses were more clear and able to breath better whilst exercise, this gave me some form of indication the drug was doing its job...or maybe a coincidence? Who knows?

Now today (day 17 into the trial; day 3 of coming off QBW251), I've started to have effects as the drug is making its way out of my body.

I noticed a slight increase in coughing in particular first thing in the morning.

Day 20 (Sunday, 8 November 2015)
Today I've been to the gym to do a chest workout. Breathing is as normal as it was when on QBW251.

Is this an indication the drug is still active in my body?

Could it be that in the last month I've simply become stronger and the effect of the drug be a coincidence?

I have 2 more visits to the hospital in regards to the trial:
  • Day 28 (Monday, 16 November 2015)
  • Day 42 (Monday, 30 November 2015)
And then...drums rolling here...3-4 months after the final date, I will know whether or not I had the placebo and results of the various tests I've had during my 42 days.

Monday, 2 November 2015

Cystic Fibrosis & Fitness: Part 2

It's been over a year since I last wrote a blog about CF & Fitness.

What a year it has been too.

In October 2014 I was doing the following program:
Exercise Weight (kg) No of sets Reps per set
Workout 1
Incline dumbbell bench press 12 4 8-12
Chest press machine 25 4 8-12
Incline dumbbell fly 10 4 8-12
Overhead extension 12 3 10-12
Cable rope press down 12.5 3 10-12
Cable kickback 2.5 3 10-12
Workout 2
Leg press 50 6 8-12
Leg extension 30 2 8-12
Leg curl 30 2 8-12
Dumbbell shoulder press 10 3 8-12
4 3 8-12
Workout 3
Vertical traction 35 4 8-12
Dumbbell row 10 4 8-12
Underarm barbell row 25 4 8-12
Dumbbell row 8 3 8-12
Cable curls 12.5 3 8-12
Hammer curl 6 3 8-12

In addition to this I also did cardio choice of rowing, cycling, jogging or swimming.

In 2015 I started working in a retirement home, where my physical activity level rose considerably.

Physical activity has gone from:

37 hours low activity job and 3 hours workout in the gym per week


25 hours high activity job and 3 hours workout in the gym per week.

A BIG difference eh?

As a result of this, I've had to decrease the weights used in my workouts slightly and by October 2015 I noticed as I got use to my high physical activity job, I gained my strength in the gym back.

Family and friends have commented how bulky I've become as I gained strength over the last couple of years.

I've found it quite an achievement where I'm at now going from a life of no physical activity to not getting enough.

QBW251 - Day 14: bye bye QBW251

Today I had an appointment at Wythenshawe Hospital in regards to the drug trial.

The sad part of today is that it's the last day I'm on QBW251.

However this doesn't mean I'm at the end of the trial. I still have to return on day 28 and 42 for further tests...I'm guessing to see changes in the body as the drug leaves the system.

Weight: 68.3kg (up by 1.2kg since last Monday).

I've gained since 18 March 2015 6.1kg (1 stone)...amazing.

This morning a repeat of some screening tests as followed:
  • CFQ-R questionnaire
  • Body weight/BMI
  • Vital signs
  • Body temperature
  • ECG
  • Urine collection
  • Blood sampling
  • Sweat chloride assessment
  • Pulse oximetry
With the blood sampling, they had to take blood pre-dose and 2 hours post-dose.

Again they managed to take the first lot of bloods first time. Yay!

However I'm yet to wait if the second lot of bloods will be taken without a glitch.

An interesting comment made about the drug is although the tests will help see how my body reacts to the drug as it enters the system, no change in lung function doesn't necessarily mean the drug isn't doing its job. There could be a chance I've simply not been on the drug long enough for it to have an affect on lung function.

It will be strange not having the drug after my last dose tonight. However I am looking forward to "what happens next".

I would say this isn't the end of the story, but the beginning.

Saturday, 31 October 2015

QBW251 - when is publicity too much?

This isn't an attack on anybody, nor should this be used as an attack.

Someone made a comment about whether or not I should quiet things down on the publishing front about the trial of QBW251 in case it turns out I've created all this hype when it could be I'm on the placebo.

Although I can see where they are coming from, what I highlighted was I made it clear from the beginning nobody knows if I'm on the drug or placebo.

All I can do is guess merely by the effects the drug is having that I could possibly well be on the actual drug.

Although there is a 50% chance I'm wrong, I have felt subtle changes since I started this trial, which could indicate I am not on the placebo.

Whether I am correct or this is by chance is another matter.

The fact the trial is ongoing to me is the main thing.

Whether or not I'm on the placebo doesn't matter. It's an unknown journey for me and I love sharing this journey with you all.

We all know there are drugs out there already for other genotypes doing the same work.

What we need to do is raise awareness about Cystic Fibrosis, its effects on the CF community and the importance this trial will have on our lives.

It's about time CF got exposed as it seems to be something not enough people know about.

QBW251 - Breathing easy in Whitby

It's been a week since I last put fingers to keyboard for updating people with the goings on in the QBW251 trial. So what has been happening? I hear you all say.

Here is a lowdown of events from day 6 to today (day 12)...

DAY 6 (Sunday, 25 Oct 2015)
Earlier this week I reported I had a sensation in my lungs and sinuses as though they were clearing themselves more.

Today at around 2:30pm whilst doing a workout at the gym, I noticed I was breathing easier than normal as I pushed the weights.

Could this be further evidence of me being on the drug and not the placebo or coincidence?

Although the only people to confirm this are those in charge of the trial, the effects I'm experiencing do bring me hope it's not a coincidence and the drug is doing as it should.

Again time will tell. In the mean time, one can't help but be excited by all this. It's about time the Cystic Fibrosis community had some form of hope of a better life thanks to modern medicine.

DAY 7 (Monday, 26 Oct 2015)
Today I had an appointment at Wythenshawe Hospital in regards to the drug trial. Basically to see how things are ticking.

Weight: 67.1kg (up by 1.3kg since trial started).

FEV1: 2.06; FVC: 4.52 (normal readings - question is, should this have gone up as result of drug working?).

The best part of today is the nurse was able to take blood samples first time. Yay!

DAY 12 (Friday, 30 Oct 2015)
Last day of holiday in Whitby.

Today I went to Whitby town centre and climbed 199 steps up to Whitby Abbey.

Guess what?

No breathless feeling.

Could this be another sign of drug working?

Or has doing fitness for the last couple of years paid off?

Again only time will tell.

Saturday, 24 October 2015

QBW251 - Warrington Guardian article from Saturday, 24 October 2015

Adam Everett, Reporter / Saturday, 24 October 2015 / News

Cystic fibrosis sufferer holds back tears as he tries breakthrough drug for the first time

Peter Trengove is taking part in the trial
of a new drug called QBW251

QBW251 could help reverse the effects of Cystic Fibrosis

A CYSTIC fibrosis suffered held back tears of happiness as he tried a new breakthrough drug for the first time.

Peter Trengrove suffers from the life-shortening inherited disease which is caused by a faulty gene, but the 36-year-old has become one of the first people to try QBW251 – a drug which could help reverse the effects caused by cystic fibrosis.

The Woolston resident has reported no side effects thus far, with people in the USA and Australia watching on via Peter’s live blogging of the trial.

On the blog Peter describes how he held back the tears as he tried the drug for the first time and described it as a new dawn.

Cystic fibrosis causes respiratory and digestive problems and the disease claimed Peter’s older brother’s life at the age of six, while Peter himself nearly died when he was just three days old because of it.

He said: “There’s a real excitement in the cystic fibrosis community about this drug.

“Since I started telling people on Facebook about this trial I've had people from the UK, USA and Australia contacting me asking about the trial drug and wishing me the best of luck.

“No one knows what’s going to happen so all eyes are on me for answers of this historic breakthrough - I feel I’ve become a celebrity.”

To keep up to date with Peter’s trial go to mycamstudionetwork.blogspot.co.uk

QBW251 - Day 5: scent sense

I had an interesting morning today.
I woke up at 8:30am with a headache slightly worse than yesterday.

Holly (my Cocker Spaniel) was lay beside me sniffing at my face.  It was as though she could smell something new about me.  As though she was detecting the drug working.

I once read about dogs and cats being able to sense things.  Whether a coincidence or she is picking up changes to my body as a result of this drug...this gets more interesting by the day.

At 8:45am I had a yogurt and then took the drug with water.

By 9:00am the headache subsided, which shows the importance of the water intake as instructed.

Friday, 23 October 2015

QBW251 - Placebo or not Placebo?...that is the question

DAY 2 (Wednesday, 21 October 2015)
Well, so far I've had no side effects or any other effects from the drug...though there's still time.

This was the first thought of today. However as the day passed, things started to happen. Whether a coincidence was another question.

At 11:20am I had a sensation in my lungs and sinuses as though they were clearing themselves more. I could feel what felt like thick mucus making its way up and out. However I chose to remain skeptical, though thought it was worth noting as feedback for my next study trail visit.

I was in CF clinic at 2:00pm. All is looking good. Effect of drug hasn't shown in lung function. Though its early days yet. The drug will probably take approx 4 days before effects, if any, are noticed. Whether I will notice them without tests or not is yet to be known.

I talked with the Doctor about the Kalydeco video I shared in an earlier post. I mentioned to him that over the years of being asked what CF is, I found it hard to put into words what it's all about. However having watched the video, this made CF so easy to understand and for me to describe the basic function that I'm surprised a drug like this hasn't been found much much sooner. This made the Doctor laugh.

Obviously had the human body been mechanical, switching on the faulty gene would be a lot easier.

DAY 3 (Thursday, 22 October 2015)
At 9:10am felt a little sick, which lasted approximately 3 hours...maybe a coincidence, but this is one of the side effects of taking QBW251.

No other side effects were experienced.

DAY 4 (Friday, 23 October 2015)
Weight 65.7kg.

The staff found it astonishing that I was able to take 6 tablets in 1 go.

When I said I'm use to swallowing a good mouthful, they started laughing. People have such filthy minds when they're around me! LOL

At 11:50am whilst at work, I started to feel a little sick again like yesterday.

At 1:50pm I started with a mild headache - another known side effect. Followed 5 minutes later with slight dizzy feelings.

Again maybe coincidence, but today I've had 3 different known side effects to the drug.

Whether signs this is THE drug or is the placebo and a coincidence is another matter.

Tuesday, 20 October 2015

QBW251 - Day1: The First Dose

Here is an account for what happened today:
7:30am Arrived at the hospital.
7:50am Oximeter check done.
8:00am Paperwork check done.

Took 2 attempts to get cannula in.

Cannula put in and bloods taken.
9:10am Ate toast prior to first dose.

I never thought how much this trial would affect me mentally.

 So many thoughts and emotions to deal with.

 It's lovely to have so many supporters.

What's more is, the support is worldwide.

I'm overwhelmed.
9:25am I'm staring at the new drug! Holding back tears here.
9:30am Final checks are being made prior to first dose.
9:35am I've seen the tablet. It's a capsule. Looks like the blood red capsules similar to Rifampicin, but probably same size as creon.

The nurses are counting the capsules to make sure all is present and correct.
9:45am First dose taken. I'm cured...or am I?

Placebo or not placebo, that is the question.
10:00am First blood sample taken.
10:15am Second blood sample taken.
10:30am Blood pressure and pulse rate taken.
10:40am ECG done.
10:45am Blood sample taken.
11:10am I've lost count to how many people are following me today.

This has gone viral in USA and Australia as well as UK.
11:30am Blood pressure, and pulse rate taken.
11:40am ECG and blood sample taken.

Mmmm yummy...this didn't last long!
Lunch time...chicken roast.  Mmmm!
12:45pm Blood sample taken.
1:30pm Blood pressure and pulse rate taken.
1:40pm Blood sample taken.
1:45pm Urine sample taken and physical examination done.
3:30pm Blood pressure and pulse taken.
3:40pm ECG done.
3:45pm Urine sample taken.
4:00pm Tuna salad baguette "snack". Yummy!
6:00pm Allowed home.
This concludes the first day of my trial.

How do I feel?

On top of the world!!!

Monday, 19 October 2015

QBW251 - Day -1: Baseline

Checking myself out - wit woo!
I woke up at 5:40am to the alarm of a clock coming from within a dream I was having. Typical. 20 minutes before my real alarm clock was due.

Ah well, at least I was up early and not late...that would've been another story.

I quickly showered and freshened myself up ready for the big day.

Clothes were laid out ready and a quick look in the mirror at me, as I am now, before I set off to a new life.

I left the house to a dark neighbourhood. Everyone still sleeping as I got in my car and silently left the area to the Medical Research Unit at Wythenshawe.

As I joined the network of traffic on the M6, I compared the motorway to an artery, it's travellers as the blood cells giving life to the island on which we live, which ironically is shaped like a begging dog.

I listened to an assortment of TV themes I downloaded. All of which I found symbolic to this day.

As I drove to Wythenshawe, I watched the sky turn from black to purple to blue. A new dawn, a new day, a new life (must write a song about that).

I parked outside the centre and entered the building which would help set a new life for the CF world.

As the staff were setting up to start today's tests, I was informed I'm the only CF person in the world on this drug trial at present.

I was told the recruiting for this trial is ongoing for taking on more CF volunteers, so WATCH THIS SPACE!

Here is an account of what happened today:

7:30am Completed "Quality of Life" questionnaire.
7:50am Weight 66.7kg (gained 0.9kg since last visit).

Temperature and blood pressure done.
8:15am ECG done.
8:30am Urine sample done - they're always taking the piss! LOL
9:00am I did the breathing tests again.

On this occasion towards the end of the first test, my mouth opened slightly after being told not to release my lips from the mouthpiece. Had to repeat the test. Oops!

Though I passed the test - I can breath! LOL
10:20am Lung function FEV1 2.08; FVC 4.51
10:30am Sweat chloride test. Sensation feels like pins and needles.
11:30am Sweat test complete.
11:45am Bloods taken...took 4 attempts. Ouch!
12:54pm All done for the day. Having lunch and then going home.

Sunday, 18 October 2015

QBW251 - Warrington Guardian artcle from Thursday, 8 October 2015

QBW251 - last thoughts before Day -1

Since the announcement was made that I'd be taking part in this drug trial, I've had many interest from many people.

It's strange really. I expected there to be interest from other people in regards to this trial, but I suppose I didn't realise how big the interest would be.

I was asked the other day how do you feel about taking part in this drug? An answer to which came about whilst I was at work having a quiet moment as I went about my duties.

I'm going through all sorts of emotions.

There's so much to take in and accept at present.

Since birth I've been used to my condition and over the years I've heard in the media of many breakthroughs towards a cure to CF. Most of which were, well, not utter rubbish, but signs of a better understanding of what makes CF tick, which would benefit progression of finding better treatments.

I didn't really know this type of treatment would become a reality in my lifetime. I thought I'd be dead well before something like this would come to life.

How wrong was I?

I said before there are eyes watching my every move on Facebook waiting for a new blog to be written.

There are CF people out there who are on the edge of their seats as they wait for me to write the blog longed to be seen...what effects am I feeling from taking this miracle drug?

Even I feel teased by this question. I don't even know if I will have the actual drug or a placebo. No one will know if I've had the drug until the trial is complete.

The only answer I will be able to give is, basically, let's see what happens.

Will I feel any effects?

Will I be on the placebo and any the effects would be by sheer cruel coincidence and all psychologyical?

Will the effects be due to me being on the actual drug?

What if nothing happens at all?

Many questions...all of which I'm having to keep myself busy so not to think about any of this.

With people at work when I've finished my shift at 2pm, if my best mate is available, I'd give him a call or Facetime and talk about anything just so my mind is focused on something other than wondering the answers to the questions above.

Don't get me wrong, I am excited about this life long wait of a drug that will make living with CF a lot easier.

I simply want everything to go really well and for this trial to result in many, who like me, have waited since they were born for a drug lke this to be available on the market so we can all breath easy.

In the mean time, all I can leave you with for now is in 36 hours time, I will probably be taking the first dose of QBW251.

Monday, 5 October 2015

QBW251 - 2 weeks to go...

2 weeks to go before I take the first dose of what could be a "history in the making" drug.  I'm rather excited about this.  Mind open and faith in medical science strong as I'm confident all will go the way it should.

The attraction the news article has brought on Facebook has seen people from Warrington, Manchester, Birmingham, and even Melbourne, Australia. Mainly friends and relatives, but also a few people I've never seen before wishing me all the best with this trial.

I sort of feel like a celebrity. I'm even tempted (as a joke) to put on Facebook "signed photos £5 each". Perhaps lick the stamp for the envelope and mark it as "almost cured DNA"...oh hang on, stamps are self-adhesive nowadays aren't they? Hey ho...another money making idea out the window.

I truly do hope the blogs I've been doing plus the article will encourage other CF people who fit the criteria to come forward and volunteer too. I feel this is an opportunity not to be missed.

Anyone interested and thinking about doing this trial, but have concerns, please don't hesitate to either ask me or your CF consultant. I've found the staff very supportive.

Well? What are you waiting for?

Friday, 2 October 2015

QBW251 - Warrington Guardian article from Friday, 2 October 2015

You've all probably seen this article by now. Though in case you haven't:

Adam Everett, Reporter / Friday, 2 October 2015 / News
Cystic fibrosis sufferer trials breakthrough drug

Peter Trengove will trial a new drug called QBW251,
which he says he has been waiting for all his life.
A CYSTIC fibrosis sufferer will trial a new breakthrough drug that he says he has been waiting for all his life.

Peter Trengove from Woolston suffers from the life-shortening inherited disease which is caused by a faulty gene.

This faulty gene controls the movement of salt and water in and out of cells, meaning that the lungs and digestive system of cystic fibrosis sufferers become clogged with mucus – making it hard to breathe and digest food.

Peter’s older brother Paul died of cystic fibrosis aged six, and Peter himself nearly died at three days old because of the disease.

But now the 36-year-old will trial a new drug called QBW251, which could help reverse the effects cause by the faulty gene and become the closest thing to a cure for cystic fibrosis yet.

Peter, who has a prescription of 15 medications to battle the disease, said: “The condition can cause life-threatening lung infections and serious digestive problems where reduced absorption of fats and proteins results in vitamin deficiency and malnutrition.

“It is one of the most commonly inherited chronic lung diseases in children and young adults."

“The affect cystic fibrosis can have on a person’s life depends on the strain – in my case I have a mild strain on both lungs and digestive system whereby you can’t tell I have cystic fibrosis."

“Although I lead as normal a life as possible there have been occasions in my life whereby even I nearly passed way from severe illness."

“The earliest age was at three days old following a major operation to remove black sticky stools from my bowel – this left me seriously ill whereby it was touch and go."

“Luckily I’ve lived to tell the tale."

“Other cystic fibrosis people with a more severe strain could spend what feels like most of their life in hospital."

“They could find it hard to secure a job due to how ill they are, find it hard to keep their weight up, be on intravenous antibiotics, be on oxygen or sadly die at a young age."

“Whilst I was attending a health check I was approached by a nurse who told me a trial study of a new drug was taking place whereby the drug worked with cystic fibrosis of my genotype."

“I was later given further information once I showed interest in taking part whereby I was delighted to find out it was a breakthrough I had waited a lifetime to see happen."

“In my parents case they’ve been waiting 44 years as they lost their first son Paul to cystic fibrosis in 1977 at six years old."

“Cystic fibrosis doesn’t only affect me, but my parents will have suffered sleepless night worrying about my health in times when I was ill.”

“Should this drug work this will have a huge impact on over 10,000 cystic fibrosis sufferers who regularly face the risk of chronic lung infections and spend a lot of time in hospital as a result.”

If you have Cystic Fibrosis or know of someone with Cystic Fibrosis and interested in taking part in this study, contact 0800 655 6553 for more information.

Thursday, 24 September 2015

QBW251 - what's it all about?

What is Cystic Fibrosis?

CF is a life-shortening inherited disease, affecting over 10,000 people in the UK.

You can't catch or develop CF. It's something you're born with. Most cases in the UK are now diagnosed soon after birth.

It causes thick, sticky mucus to build up in the lungs, digestive tract and other areas of the body.

The build up of mucus is caused by a defect in the CF gene chromosome, which results in a disruption of the trafficking chloride (salt) channels (called Cystic Fibrosis Trans-membrane conductance Regulator or CFTR) that are found on the cell surface (CFTR membrane).

In the normal healthy state, this allows the easy passage of substances to and from the cell surface. However in CF the disruption in the flow of these substances to and from the cell surface leads to the collection of sticky mucus.

The condition can cause life-threatening lung infections and serious digestive problems where reduced absorption of fats and proteins results in vitamin deficiency and malnutrition.

It is one of the most commonly inherited chronic lung diseases in children and young adults and is a life-threatening condition.

How will QBW251 help with CF?

QBW251 is a drug that may improve the activity of the defective chloride channel CFTR.

It is suggested that helping CFTR work better by adding QBW251 will result in improved salt transport at the cells surface. Therefore an improvement in the thick, sticky mucus, and an improvement in lung disease in CF patients.

Here is a video presentation for a drug called "Kalydeco", which does the same as QBW251, but for a different genotype.

What are the side effects of QBW251?

All drugs carry a risk of side effects. In Parts 1 and 2 of the study (healthy volunteers), there were no serious side effects seen and no side effects were seen that were related to volunteers discontinuing with the study.

The most common side effects of QBW251 observed in humans include: headache (15%, 15 patients in every 100), cough (6%, 6 patients in every 100), dizziness (5%, 5 patients in every 100), and flatulence (4%, 4 patients in every 100).

Apart from cough, these side effects were also commonly observed in humans treated with placebo. There were no abnormal clinical results (laboratory tests, vital signs and ECG) that were associated with exposure to QBW251 at any dose or exposure in either study part.

Changes in adrenal glands and kidneys have been seen in animals that have been given extremely high doses of QBW251 and there is a potential risk of diarrhoea. None of these adverse effects have been seen in the healthy volunteers who participated in parts 1 and 2 of this study.

Is QBW251 available on the market?

QBW251 is an “investigational drug”, which means that it is a medicine that is being tested and has not been approved by the regulatory agencies (health authorities) around the world.

QBW251 is currently not “on the market” in any country. The study drug is being used in this study for research purposes only, and will not be available to you to continue taking after this study has finished.

If I was to participate in the study, what will I have to do?

Visit Length of visit
Consent (Visit 0) 1.5 hours Read and sign consent forms.
Screening (Visit 1) 6 hours Study Doctor collects:
  • Basic information about you
  • Medical history
  • Details of current medical condition
  • Medication you are currently taking
  • Body measurements i.e. height, weight and vital signs (pulse, blood pressure, respiratory rate & body temperature)
  • Blood samples for CF genotyping, safety bloods (biochemistry, haematology) & HIV and Hepatitis blood screens
  • Alcohol, drugs & cotinine (to find if you smoke)
  • Pregnancy & follicle stimulation hormone test (for females)
  • Urine collection
  • ECG
  • Lung function
  • Pulse oximetry
  • Sputum collection
Day -1,
Baseline (Visit 2)
6 hours Baseline study evaluations will be conducted.

Repeat of some screening tests as followed:
  • Repeat assessment of the inclusion/exclusion criteria
  • Medical history & current condition
  • Physical examination
  • Pregnancy test (for females)
  • Body measurements
  • Vital signs
  • Blood sampling
  • ECG
  • Lung function
  • Sweat chloride assessment
  • Pulse oximetry
  • CF Quality of Life (CFQ-L) questionnaire
Day 1 (Visit 3) 9 hours On the evening before between 9pm & midnight, a salivary cortisol sample will be collected.

Blood samples for pharmacokinetics and blood, plasma and serum biomarkers will be taken before you receive your first dose of QBW251.

After this, a further 7 pharmacokinetic blood samples will be collected at 15 min, 30 min, 1 hour, 2 hours, 3 hours, 4 hours and 6 or 8 hours post treatment.

Repeat of some screening tests as followed:
  • Repeat body temperature 4 hours post treatment
  • Repeat vital signs and ECG tracing at 1, 2 and 6 or 8 hours post treatment
  • Biochemistry and haematology sampling along with repeat urinalysis at 4 & 6 or 8 hours post treatment.
The second dose of QBW251 will be administered 12 hours after the first dose.
Day 4 (Visit 4) 3 hours The next dose of QBW251 will be administered.

Repeat of some screening tests as followed:
  • Body weight/BMI
  • Vital signs
  • Body temperature
  • ECG
  • Urine collection
  • Blood sampling
  • Pulse oximetry
Day 7 (Visit 5) 5 hours On the evening before between 9pm & midnight, a salivary cortisol sample will be collected.

Repeat of some screening tests as followed:
  • Body weight/BMI
  • Vital signs
  • Body temperature
  • ECG
  • Urine collection
  • Blood sampling
  • Pulse oximetry
  • Lung function
  • Sweat chloride assessment
Day 14 (Visit 6) 5 hours On the evening before between 9pm & midnight, a salivary cortisol sample will be collected.

Repeat of some screening tests as followed:
  • CFQ-R questionnaire
  • Body weight/BMI
  • Vital signs
  • Body temperature
  • ECG
  • Urine collection
  • Blood sampling
  • Sweat chloride assessment
  • Pulse oximetry
Day 15,
Follow up (Visit 7)
1 hours Repeat of some screening tests as followed:
  • Lung function
  • Pulse oximetry
Day 28,
Follow up (Visit 8)
4 hours Repeat of some screening tests as followed:
  • Body weight/BMI
  • Vital signs
  • Body temperature
  • Urine collection
  • Blood sampling
  • Lung function
  • Sputum sampling
  • Sweat chloride assessment
  • Pulse oximetry
  • CFQ-R questionnaire
Day 42,
Follow up - End of Study (Visit 9)
3-4 hours On the evening before between 9pm & midnight, a salivary cortisol sample will be collected.

Repeat of some screening tests as followed:
  • Pregnancy test (for females)
  • Body weight/BMI
  • Vital signs
  • Body temperature
  • ECG
  • Blood sampling
  • Urine collection
  • Sputum sampling
  • Sweat chloride assessment
  • Pulse oximetry
  • CFQ-R questionnaire

You will need to:

  • Carry with you at all times a card (the same size as a credit card) which the study doctor will give you during the study. Cards like this are given to everyone who takes part in this kind of study; they include phone numbers to contact in any emergency.
  • Attend all of the study visits, and follow the instructions you are given. All of these visits will take place in your study hospital.
  • You may eat and drink normally whilst you are in the study, although we ask you to watch how much salt you eat and to eat some food just before you take each dose of your study treatment.
  • You will need to avoid being in direct sunlight, even if you cover your skin with clothes. If this will be difficult, your study doctor might suggest that you use tropical sub-blocking creams.
  • Tell your study doctor about any other medicines you are taking during the study. This is very important. This includes prescription drugs and over the counter medications (including creams and ointments, vitamins and supplements) that you take or use now, or intend to take — even ones you buy without a prescription. There are some drugs you are not allowed to take whilst you are on this study.
  • Tell your study doctor about any other conditions that you now or have suffered in the past.
  • Tell your study doctor if you have any unusual symptoms during the study.
  • Tell your study doctor if you want to stop being in the study at any time.
  • If you are a woman who could become pregnant:
    • You should not plan to become pregnant during the study, as we don’t yet know whether the study medicine is safe for an unborn baby.

      Women who are pregnant or nursing a child cannot take part in this study. You must confirm, to the best of your knowledge, that you are not now pregnant, and that you do not intend to become pregnant during the trial. The risks to an unborn human baby or a nursing child from the medications you are required to take in this study are not known.

      If you are female, to take part in the study you must be post-menopausal (no menses for at least 12 months) or be surgically sterile or use contraception methods listed below.

      The documented methods of surgical sterilization and contraception methods include:
      • having had a hysterectomy (removal of the uterus with or without the ovaries), or
      • bilateral oophorectomy (removal of both ovaries), or
      • A tubal ligation (having your tubes tied), or
      • Total abstinence (no sexual relations), when this is in line with your preferred and usual lifestyle. Periodic abstinence like calendar, ovulation, symptothermal, post-ovulation methods, and withdrawal are not acceptable methods of contraception, or
      • Your sole partner is another woman, or
      • Your male partner has already been sterilized with the appropriate documentation. The sterilized male partner should be your sole partner, or
      • Using both of the following:
        • Placement of an intrauterine device (IUD) or intrauterine system (IUS), and
        • Use of an occlusive cap (diaphragm or cervical/vault cap) by you, or a condom by your male partner combined with a spermicidal foam/gel/film/cream/vaginal suppository.

Wednesday, 23 September 2015

QBW251 - Day -2: breathe in and relax

Day -2

Here is an account of what happened today:
6:00am Got up and showered.

View from bedroom window

Ready for the big day
Started my travel to hospital...
7:50am Arrived at the hospital.
8:40am By this time I had my BMI, temperature, blood pressure and ECG done.

Hardest part was to keep quiet during tests as talking would hinder the test and therefore would have to start again.  I wasn't allowed to do anything at all during the tests such as read, use mobile or listen to music for the same reason.
9:00am I was asked to do a urine sample mid-steam.  I asked where the nearest brook was.  I think the staff realised by this time I have a good sense of humour.
9:30am I did a breathing test 1/3, which took 7 minutes to complete.  I passed...I can breath!
9:45am Breathing test 2/3.  Guess what?  I passed again!  LOL
10:15am Breathing test 3/3.  I think I've convinced them now I can breathe.  No more breathing tests.
11:15am Lung function FEV1=2.15; FVC=4.65.
12:00pm Blood samples taken and all is done for today until Monday, 19 October 2015.
Home in time for lunch.

Tuesday, 22 September 2015

QBW251 - tick, initial, signature, date

Day -3

You may ask why I've put the day as -3 (minus three)?

Today was all about going through all the information with the Doctor about what the drug trial will involve.

I had to sign paperwork to show I understand what is to happen and that I consent to basically being a guinea pig.

The volunteers suite is stunning.  Whilst undergoing screening during this trial, I will have access to facilities such as a snooker room, cyber cafe, entertainment and relaxation room (where I can either play games on an X-box or watch DVDs, a small cafeteria and a self service refreshments section.  Plus my own relaxation garden.

I feel as though I'm going to be well looked after during this trial.

The people I saw were nice and very helpful in answering any questions asked.  Most of which were more because I was interested in what's going on rather than concerned.

Tomorrow, Day -2 I will have a screening assessment to see if I'm eligible to take part in this study.  Of course we all know already I am eligible.  However this is protocol.  This will involve taking basic information such as medical history,details of current medication and medical condition.  My BMI will be taken and vital signs i.e. pulse, blood pressure, respiratory rate and temperature.  I will undergo a full physical examination.

Blood samples will be taken for CF genotyping, safety bloods and other blood screens.

Other tests will include alcohol, drug and smoke tests.  Apparently I don't need to do a pregnancy test.  Though they will be taking the piss (a urine sample), doing an ECG and lung function.

Basically, by the end of the day, I will be poked, prodded, and pricked...nothing new here.  It will be like having an annual health assessment, but more thorough.

Early night to bed tonight.  I'm up at 6:30am so I can get ready to leave at 7am to be at the hospital for 8am.

More writings tomorrow to let you know how things go...goodnight from me and goodnight from him (now where did that quote come from?).

Monday, 21 September 2015

QBW251 - the trial study

On Wednesday, 16 September 2015 I had a port flush appointment at Wythenshawe Hospital.

During my appointment I was approached about taking part in a study to find out how the study drug, QBW251, works in the bodies of patients with CF.

I was given the opportunity to discuss this with family and friends before making up my mind.  However at the time of asking it wasn't made clear to me what drug I would be testing.

I requested more information on this to understand what was to happen.

Based on little information given at the time, I looked back over the last year or so to news items related to breakthrough medication for CF.

A story came to mind about a breakthrough in CF treatment for the lungs whereby the medication reversed or corrected the faulty gene (or something to that effect).

I suddenly had a feeling had I declined this opportunity, I would regret it.  So having spoken quickly to my dad and partner (by text), their encouragement confirmed this felt like the right thing to do.

Attempts were made to ring the hospital to confirm my interest in the study and it was a matter of time before I received the call I had been waiting for...the call that would be the beginning of a possible life changing experience.

Tomorrow (Tuesday, 22 September 2015), I will be attending my first appointment to go through processes and officially give my consent to be part of this study.

Come October, I will start trials of QBW251, a drug that may, when given to humans, improve the activity of the defective chloride channel CFTR, helping it to work better resulting in improved salt transport at the cells surface, lessen the thick, sticky mucus, and reduce lung disease in CF patients.

The only twist in this study is there is a chance I will be given a placebo...watch this space to see what happens over the next 2 months...