For Science ... (drug trial participant)

Miscellaneous and Personal Stuff I Must Share
1

As I have previously mentioned on these boards, I have chronic Hepatitis B, for which I have been treated with antivirals for the last four years. Having recently returned to my native NZ, I had an appointment with a hepatologist to arrange continued treatment. After she approved my access to the appropriate drugs, she asked if I would be interested in a phase 2 clinical trial for a therapeutic vaccine for HBV.

Basically, the theory is that those with chronic HBV have an incomplete immune response to the virus. The theraputic vaccine is different to the current vaccine in that it expresses internal HBV proteins rather than surface ones. The experimental treatment runs alongside my existing oral antiviral program. As a small benefit, due to the trial rules I get to not change the drug I am taking - there is a slightly cheaper option that is just as good but I tolerate the current drug really well and I prefer to continue it.

So, being an adventurous soul, I decided to sign up. I was screened for height, weight, drug use, yeast allergy, compliance with my current regime, and a few other things. My examining doctor was a former head of dept of infectious diseases, so knew his stuff. He wasn’t quite convinced by the theraputic vaccine approach, so he set my expectations quite low - this is more about data gathering and probably won’t produce a cure directly.

Yesterday, they took baseline bloods (12 tubes), randomly allocated me to a dosing level (2, from 0, 2, 8 or 40) and gave me my first shot. I had to be observed for an hour in case of an adverse reaction. I get more bloods done in 2 weeks time. This repeats for another 3 months, then I have monthly bloods for another 6-7 months.

I do get paid a small fee at the end (I’m planning on a new guitar, but my wife has other plans), and travel expenses. But it wasn’t the money, it was the opportunity to help make some progress on finding a cure - the drugs I am on cost the NZ govt $800(NZD) per month, and I will be on them for a lifetime without a cure.

Oh, and my motivation can be summed up in the final panel here …

2

Good luck to you! I’m surprised they told you which dose you were getting in the randomization. Seems to put that whole double blind thing out the window!

3

As someone who works in the pharmaceutical industry (although not R&D), thank you!

4

USCDiver: They often (but not always) hold off on double-blind until phase III testing, depending on the study’s structure, so that doesn’t surprise me.

Thanks for participating in what will hopefully be the advancement of scientific knowledge, si_blakely! I’ve worked in that ‘setting’ (clinic/hospital-based, with patients) of medical research for over a decade now. I’m glad to hear the doctor was downplaying the possibilities; it seems like almost everyone comes in expecting to be given The Magic Cure even after you’ve explained this is why we’re doing research, we’re not sure it’ll work. Some people need a good dose of reality talk along with the study medication. :slight_smile:

5

I’ve done a few drug studies.
I got free physicals, free meds and paid for it too.

The last one included some genetic testing which they said would not benefit me but would benefit future generations as they were trying to match which drugs are most effective with genetic types.
I had to sign a bunch of consent forms and all they were doing was taking an extra vial of blood.

6

Yep - they took buccal swabs for HLA testing, and I have allowed samples to be kept for later research (Biomarker and DNA investigations into success/failure, as I understand it).

I made the nurse laugh - I had 4 swabs left and right, upper and lower, timed rubbing for 15 seconds each. My comment -
“CSI makes this look much easier”.

I was just glad I was in the tranche getting a dose - I would have felt a bit let down if I was in the 0 branch. But I was somewhat relieved not to be in the 40 tranche - they get 8 shots per treatment, 2 in each arm and leg.

Pleased to report that I have had no reactions (adverse or otherwise) in the two days since the jab. So I am feeling pretty happy. And my schedule has been adjusted to fit in with my workday much better. They have already planned around my 4 week absence in the US for work - I miss one blood test and one of my treatments is delayed a week, which is acceptable.

7

Bumping this thread after almost 2 years to provide a HAPPY update…

I finished the initial trial, which was an immunological approach (basically a vaccine for a core HBV protein).

I stayed on a followup program monitoring my immune system and taking DNA.

I started a new trial based on the original trial, that included a immuno-modulation drug (already approved, used for other diseases).

I was in an early phase, intending to test HBV response to a low dose of the immuno-modulator (no additional vaccine exposure).
One month in, my liver enzymes were spiking, and the medical staff strongly recommend that I did not to travel to the US for a week training course for work. It could have been an autoimmune response to the immuno-modulator (a known risk).
My liver results settled down to normal over the course of the trial which finished 4 weeks ago.

I saw a consultant last week, and following a final confirmatory blood test, I no longer need to take anti-viral medication for HBV:
i.e. it appears to work (subject to monitoring for a few months to ensure I do not revert).
This is the first time it has happened on the trial, and obviously it gives the researchers great hope that this can be developed into a reliable and successful treatment program.

I’m hoping I get my own reference in the eventual paper - Patient A :smiley:

I can’t provide too many more details of the treatment itself (blah, blah, commercial sensitivity, blah, blah, NDA) but it was very low impact on my life and health while it happened.

Science: it works

8

A final bump on this thread:

My study published - PS-044 abstract here:
A phase1 study evaluating anti-PD-1 treatment with or without GS-4774 in HBeAg negative chronic hepatitis B patients - Journal of Hepatology

One TAF patient indeed :rolleyes:

9

A priest friend of mine volunteered for an NIH AIDs vaccine study at Vanderbilt University. They wanted people that were celibate. My sister and I were inspired to do the same thing, so we both volunteered for a year of celibacy (not like we had any likely prospects anyway) and involved ourselves in the study. At the end of the study we found out she got the placebo and I got the vaccine. That was in 1990, and they told us they thought they’d have a workable vaccine in 10 years. It hasn’t happened yet.

As an interesting side note, at the picnic for study participents, I found out that of all the volunteers throughout the country, I had the strongest immune response to the vaccine. Pretty cool.

StG

10

A priest friend of mine volunteered for an NIH AIDs vaccine study at Vanderbilt University. They wanted people that were celibate. My sister and I were inspired to do the same thing, so we both volunteered for a year of celibacy (not like we had any likely prospects anyway) and involved ourselves in the study. At the end of the study we found out she got the placebo and I got the vaccine. That was in 1990, and they told us they thought they’d have a workable vaccine in 10 years. It hasn’t happened yet.

As an interesting side note, at the picnic for study participents, I found out that of all the volunteers throughout the country, I had the strongest immune response to the vaccine. Pretty cool.

StG

11

I received an invitation in the mail to participate in a clinical study. apparently some of the participants are to receive testosterone-lowering drugs.

I decided to decline this opportunity.

12

That’s weird - report 24 minutes apart. I have no clue how that happened.

StG

13

I missed these threads previously, so I’m glad you bumped it.

Congratulations! How are you doing since the update last August?

14

Absolutely fine - my specialist (Dr Gane, NZ Innovator of the Year 2017) is being cautious, but should finally discharge me in a couple of months.

15

So, you HepB is…cured? Not just managed, cured?

16

Cured - full seroconversion.

Only ~300million chronic HepB sufferers to go …

17

Good news for me as I am one of the unlucky HBV chronics. I’ll send link to my doc.

Any idea how long it will take for that particular drug to be accessible in local drug stores? Since this disease is quite common I’d expect it would speed up that process …

18

This was a phase 1 trial for safety - probably another 5-10 years to develop, test and get approval for a working protocol.

Antiviral treatment is still the best option.

19

Congratulations, Mr. One TAF patient. I bet you still do the happy dance from time to time.

20

Congratulations! May your success be echoed in the trials that follow for other patients.