A Phase-2, Prospective, Open-Label Study to Determine the Safety and Efficacy of Sotatercept (ACE-011) in Subjects With Myeloproliferative Neoplasm (MPN) -Associated Myelofibrosis and Anemia
Study Groups:
If you are found to be eligible to take part in this study, you will be assigned to 1 of 2
study groups, that will receive different doses of the study drug, based on when you join
this study.
- Group 1 will receive sotatercept 1 time every 3 weeks (at a lower dose).
- Group 2 will receive will receive sotatercept 1 time every 3 weeks (at a higher dose).
Study Drug Administration:
Each cycle is 21 days.
You will receive sotatercept at the clinic as an injection under the skin on Day 1 of every
cycle. The injections will be given in the upper arm, abdomen, or thigh.
Study Visits:
On Day 1 of every cycle:
- You will have a physical exam, including measurement of your weight and vital signs
(blood pressure, breathing rate, temperature, and heart rate).
- Your spleen and/or liver will be measured by feeling your abdomen.
- You will be asked about any drugs you are taking and about recent blood transfusions
you may have had.
- You will also be asked about any new side effects or medical conditions you may have
experienced.
- Your performance status will be recorded.
- Blood (about 4 teaspoons) will be drawn for routine tests.
Weekly during Cycle 1-5 only:
- Blood (about 2 teaspoons) will be drawn for routine tests.
- You will have vital signs measured on Day 1 of Week 3 (around Day 15 of Cycle 1).
On Day 1 of Cycle 6, and then every 6-12 cycles after that if the disease responds to
treatment, a bone marrow biopsy will be performed to check the status of the disease.
Length of Study:
You may continue taking the study drug for as long as the doctor thinks it is in your best
interest. You will no longer be able to take the study drug if the disease gets worse, if
intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over after the follow-up visits.
End-of-Treatment Visit:
After your last dose of study drug, you will have an end-of-study visit and the following
tests and procedures will be performed:
- You will have a physical exam, including measurement of your weight and vital signs
(blood pressure, heart rate, breathing rate, and temperature) and your spleen and/or
liver will be measured by feeling your abdomen.
- Your performance status will be recorded.
- Blood (about 4 teaspoons) will be collected for routine tests.
- You will be asked about any drugs you are taking and about recent blood transfusions
you may have had.
- You will also be asked about any new side effects or medical conditions you may have
experienced.
Follow-up Visit:
About 1 month after your last dose of study drug, you will receive a telephone call from
someone on the study team. You will be asked about any drugs you are taking, any recent
blood transfusions you may have had, any new side effects or medical conditions you may have
experienced. This phone call should last about 10 minutes.
This is an investigational study. Sotatercept is not FDA approved or commercially
available. It is currently being used for research purposes only.
Up to 40 patients will take part in this study. All will be enrolled at MD Anderson.
Interventional
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Anemia Response
Primary analysis of response for each dose cohort performed once all 20 patients have been accrued to that dose cohort and treated for at least 5 cycles. Clinical efficacy assessed as anemia response with a target rate of greater than or equal to 30% at either dose level. Anemia response is a composite endpoint defined as an increase in hemoglobin in a subject with anemia or becoming RBC-transfusion-independent in a subject who is RBC-transfusion-dependent.
15 weeks
Yes
Srdan Verstovsek, MD
Principal Investigator
UT MD Anderson Cancer Center
United States: Food and Drug Administration
2012-0534
NCT01712308
February 2013
February 2017
Name | Location |
---|---|
UT MD Anderson Cancer Center | Houston, Texas 77030 |