PV Research study

[ERei]

There was a full page article in my local newspaper to be part of an Phase 3 investigational drug study for ppl with PV. The drug is called rusfertide - it says it is designed to control the amount of iron available to make red blood cells. There is no financial compensation to be part of the study but it will help advance PV research.

For those who may be interested the website is: [e-mail]

Tel: [phone]

[AnPV]

Hi, I take part of this study (i started a few weeks ago). I decided to participate because my treatment exists out of many plebotomies (I don't want to start HU because of my age and interferon is still not available in my country). The injections with Rusfertide should replace those plebotomies. I have no side effects, but maybe that's because I receive a placebo in this first part of the study :-). The good thing with this study: after 32 weeks everyone receives the medications for almost another 2 years and a half. Are there other members of this community who take part as well? Enyoy this day!

[AnBee in reply to AnPV]

won’t that hide the potential longer term side effects of this intervention? Giving the whole cohort the same drug ( assuming it randomised controlled) in just over 9 months has the potential to miss more serious longer term side effects?

[AnPV in reply to AnBee]

Hi AnBee, phase 3 of the study takes 3 years in total. I started recently but others might have started a few years ago. It's only in the first 32 weeks that one can get a placebo (50% change - blind controlled). After 32 weeks every patient gets Rusfertide. So side effects over a period of 2,5 to 3 years will be noticed, I guess. I really have no idea when to speak of "long term" in cases like this. Kind regards, An

[EPguy in reply to AnBee]

Often studies allow cross over to the trial drug for all as an inducement for participants. Esp for MPNs, there are various good treatments that are competing with the study drug.

This is the study design:

clinicaltrials.gov/study/NC...

They require pts to be having phlbs and allow stable condition with hydroxyurea, interferon and/or ruxolitinib. "Response is defined as absence of phlebotomy eligibility." So it looks like they intend Rusf to specifically replace phlb rather than any of the other therapies. Since Rusf and phlb both act on just RBCs (HCT) this makes sense.

The post placebo stage (after week 32) has no measured criteria, including no safety goals during the trial period. This is unusual. The only late objective is "Approximately 6 and 12 months after their last dose of rusfertide, subjects will have a post-study contact (e.g. by phone) for safety"

Two implications:

-They really want to encourage pts to join.

- They are confident in Rusf safety.

[AnBee in reply to AnPV]

That’s just it. Side effects may show up in 2-3 years but will be lost in the noise

[Bobadog]

Hi

Thanks for all the above.

Is Rusefertide as an effective Jak 2 inhibitor as Rux and Momelitinib,which is soon to be available 🤞

Bobbie

[EPguy in reply to Bobadog]

Rusf address a different area by a different method. It does not inhibit Jak. Rather simulates "hepcidin", relating to iron regulation. It's for "Control of Erythrocytosis in Polycythemia Vera". In practical terms it means Rusf is supposed to reduce HCT but won't affect (help or hurt) other MPN issues like WBC, PLT etc.

This is the phase 2 results indicating it worked:

nejm.org/doi/full/10.1056/N...

From the phase 3 trial design it seems intended for those who can't get HCT down enough by or tolerate any of Rux, IFN, HU, Phleb. Maybe like a back up system. But for PV pts with only Hb/HCT troubles Rusf might be good by itself. Seems unlikely to reduce allele burden, but I don't think that is being tested.

[Bobadog]

Hi

Many thanks for this helpful explanation.

I’ve just found the video of the MPN Forum (ASH March 8th).

Dr Jonathan Lambert explained the recent work /trials on RUSF too.

It was a very informative video.

Thanks very much again,

Bobbie