DBAR Clinical Trials

The purpose of this study is to maintain a comprehensive registry of patients with the rare inherited bone marrow failure syndrome Diamond Blackfan anemia (DBA). The objective of this study is to expand and update the DBAR in order to:

 

1.   Facilitate investigations into the epidemiology and biology of Diamond Blackfan anemia;

2.   Provide an accurate phenotype of DBA patients to facilitate genotype- phenotype correlations;

3.   Provide access of well characterized patients to treatment protocols;

4.   Provide patients to access to research studies;

5.   Provide patients with results of research studies;

6.   Serve as a resource to patients and their doctors to guide diagnostic, therapeutic, and reproductive decisions.

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
The Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance, using Bone Marrow Failure as a Model: A Pilot, Phase I/II Study of the Amino Acid Leucine in the Treatment of Patients with Transfusion-Dependent Diamond Blackfan Anemia
Phase I/II, Open-Label Study to Determine Safety of Trifluoperazine (TFP) in Adults with Red Blood Cell Transfusion-Dependent Diamond Blackfan Anemia