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MDM2 Study: How Can Polycythemia Vera Patients Get Involved?

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Published on November 15, 2019

Key Takeaways

  • Learn about a current trial for polycythemia vera patients.
  • Qualifying factors for current PV trial.
  • Number of trail sites and phases for PV clinical trial.

Patient Power Co-Founder, CLL patient and advocate, Andrew Schorr, talks with Dr. Jason Gotlib, from Stanford University Cancer Institute, about the current MDM2 clinical trial for polycythemia vera patients. Dr. Gotlib shares inclusion criteria for patients to discuss with their healthcare team and gives an overview of how many trial sites and patients will be accepted for each phase.  Watch now to hear from a myeloproliferative neoplasm (MPN) expert.

See below for a map of clinical trial sites.

This program is sponsored by Kartos Therapeutics, Inc. This organization has no editorial control. It is produced solely by Patient Power.

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Transcript |

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That's how you’ll get care that's most appropriate for you.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Andrew Schorr:

Dr. Gotlib, for our patients who are watching their PV, what should they ask their doctor?  What situation might they be in where they should say is there a trial, this trial perhaps, that could be right for me?  

Dr. Gotlib:

So this trial would be appropriate for patients who have PV that are on Hydrea and are showing themselves to be Hydrea resistant or intolerant.  Let me give you a little bit of flavor what that means.  So Hydrea resistance, for example, would mean someone who has been on Hydrea for at least 12 weeks who is at least on 2 grams per day or lower than 2 grams per day but are not achieving the goals of being phlebotomy free or have an elevated white blood cell count or platelet count, so that would be some examples of Hydrea resistance.  

Hydrea intolerance, for example, would be patients that are having, for example, oral ulcers or skin ulcers or GI intolerance or have low blood counts and are not able to necessarily achieve their phlebotomy goals with their doses, for example, 2 grams per day or lower than 2 grams per day.  So that would be a Hydrea resistant or intolerant population that would be well suited for this trial.  

 

Andrew Schorr:

Dr. Gotlib, how is this trial an example of the progress you're trying to make for patients with PV? 

Dr. Gotlib:

So it's a great question.  You know, historically patients with PV who have needed treatment either because of advanced age or prior thrombosis, which define high‑risk PV, or again a hydroxyurea (Hydrea) resistance or intolerance, there is an unmet need here.  We can do a lot better in terms of trying to achieve hematologic responses in these patients.  

So I see KR T232 with this new mechanism of action really try to fill that niche of trying to do better with regard to human toxic responses, that is, phlebotomy control, reduction of spleen volume.  And there are secondary end points too such as reducing symptom burden in these patients, which is a major issue.  So we really are trying to basically improve hematologic benefits, quality of life, and there's a lot that we can do better given the agents that we have which have really been around for decades at this point in time.

Andrew Schorr:

And, Dr. Gotlib, as far as patients accessing this trial, so obviously at Stanford, you're one of the centers, so there'll be trial sites proliferating where patients might be able to have this treatment?  

Dr. Gotlib:

So that's correct, Andrew.  So in part A, which is the three‑dose arms, I believe there will be about 50 sites, and in part there will be about 70 sites.  In part A we're looking to accrue about 75 patients, 25 patients per each of those three dosing arms or 75, and then in part B it will be 110 patients for the KR T232 arm and 110 patients in the ruxolitinib (Jakafi) arm.  So altogether there are 220 plus 75 or 295 patients that we plan to accrue at 50 to 70 sites.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

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