Clinical Trial: Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Phase I/II, Open-Label Study to Determine Safety and Efficacy of Sotatercept (ACE-011) in Adults With Red Blood Cell Transfusion- Dependent Diamond Blackfan Anemia
Brief Summary: The purpose of this study is to determine the safety and dosing of drug Sotatercept, as a subcutaneous injection, to stimulate production of red blood cell production. To be given every 28 days for up to four doses.
Detailed Summary:
This study is for adult patients with Diamond Blackfan Anemia who are currently being transfused every 3- 4 weeks.
The drug will be given as a subcutaneous injection within one of three dose levels. The first dose level (0.1 mg/kg) will include 3 subjects who will receive the same dose of the drug monthly for a total of 4 doses, as long as there are no dose- limiting toxicities (DLTs). A Dose limiting toxicity is defined as inability to deliver the scheduled doses because of toxicity >/= Grade 3, according to NCI Toxicity Grading Scale.
Once all 3 subjects have received and tolerated the low dose level, the next level will open (0.3 mg/kg)to 3 new subjects. Subjects will be monitored closely for response and side effects.
If more than 2 subjects have a dose limiting toxicity in the first dose level, then dose level -1 (0.05 mg/kg)will open, enrolling 3 more subjects. If more than 2 subjects at dose level -1, have dose limiting toxicities, the study will be discontinued. If there are no additional dose limiting toxicities, dose level -1 will be the maximum tolerated dose.
There are a total of 3 dose levels, not including dose level -1. Once the maximum tolerated dose has been reached, up to a total of 10 additional subjects will be enrolled.
Protocol Amendment: The protocol has been amended to include an additional enrollment of 20 subjects with two additional dose levels of Sotatercept (0.075 mg/kg and 0.100 mg/kg ) to be given with or without a prednisone boost.
Efficacy will be measured by response. A complete response will be determined if the subject no longer requires transfusion, while on study drug. A partial respons
Sponsor: Northwell Health
Current Primary Outcome: Determine a safe and effective dose of sotatercept in adults with DBA and RBC transfusion dependence [ Time Frame: 9 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Safety [ Time Frame: 9 months ]Assess type, frequency, and severity of adverse events and relationship to sotatercept according to the currently active minor version of the NCI Common Terminology for Adverse Events version 4.0
- Time to response [ Time Frame: 9 months ]Length of time required to achieve a response
- Response duration [ Time Frame: 9 months ]Length of time between transfusions
- Exploratory outcome improvement of bone density [ Time Frame: 9 months ]Improvement as measured by bone densitometry (DEXA scan)
Original Secondary Outcome: Same as current
Information By: Northwell Health
Dates:
Date Received: October 31, 2011
Date Started: January 2012
Date Completion: December 2017
Last Updated: December 20, 2016
Last Verified: December 2016