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Phase 3 Luspatercept Trial

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CrazyPharm:
Hi everyone! I didn't see any threads about the new Luspatercept trial so I figured let me pass some info. along and get a discussion going since I am so excited. Recently Acceleron and Celgene announced they are ready to progress their Luspatercept program into Phase 3 Clinical trials. This means once this trial is over and the results are good they can submit to the FDA for drug approval.

Recap: Luspatercept is a subcutaneous injection given once every 3 weeks to help reduce the blood transfusion burden in thalassemia patients. Phase 2 results have been excellent with minimal side effects and all evaluable patients showing greater than 40% reduction in blood transfusion requirement (transfusion dependant patients).

The phase 3 trial is titled the "BELIEVE" trial and is scheduled to start by the end of this year, 2015. Patients who are regularly transfused (8-20 units per 24 weeks) will be eligible to participate. 200 patients will receive luspatercept 1 mg/kg SubQ while 100 patients will receive placebo (non-active injection). Neither patient nor investigator will know who is receiving drug or placebo. The trial is therefore random and double-blinded.

No details yet on where the trial will be offered but I have been told it is a global trial. Stay alert for more updates. If any one has any further to add please do so :)

Andy Battaglia:
I can't see why a double blind trial is being used. It's not like a placebo effect is going to raise the Hb level.

CrazyPharm:

--- Quote from: Andy on November 07, 2015, 06:25:30 PM ---I can't see why a double blind trial is being used. It's not like a placebo effect is going to raise the Hb level.

--- End quote ---

I absolutely agree with you. I think it complicates things and adds undue harm to the placebo recipient since they will wait for hgb to drop to a specific level before transfusing most likely 1 unit at a time. There will be at least weekly cbc to monitor hgb. For 48 weeks for a patient to get placebo and undergo all this is tortourous (being a patient myself).

The only logic I can see in a placebo trial is to compare side effects. If placebe thalassemia patients complain of headache and bone pain more so than the general population it may help to discredit these side effects being attributed to the drug itself. I can't imagine why a patient would stick it out for 48 weeks when one would realize by week 5-6 that obviously the drug isn't being administered since blood requirement would remain the same and quality of life worse than before.

Andy Battaglia:
All good points. And something expressed to me is that not knowing if they're getting the drug is keeping patients from joining trials.

Sharmin:
I have been waiting to enrol lil A in this trial for a long time.  I understand why it may be helpful to make this trial double blind, but for my son - who has an antibody which is active some times and not others - his participation in the study may skew the results of the study is conducted double blind.  His results can only be compared to himself - how he did in a given year with and without luspatercept. 

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