AnemiaRare diseaseGenetics
What this study is
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).
Study details
- Sponsor
- Northwell Health
- Conditions
- Anemia · Blood Disease
- Enrolling
- 900 people
- Starts
- 2004-09
- Est. completion
- 2026-04
- Record
- View on ClinicalTrials.gov ↗
Who can join
Sex ALLHealthy volunteers Not acceptedYou may qualify if
- Normochromic, usually macrocytic and occasionally normocytic anemia developing early in childhood
- Reticulocytopenia
- Normocellular bone marrow with a selective deficiency of red cell precursors
- Normal or slightly decreased leukocyte count
- Normal or often increased platelet count
- Or, a confirmed mutation in one of the identified DBA genes
You can't join if
- Any subject identified as having another bone marrow failure syndrome (eg. Fanconi anemia, dyskeratosis congenita, Shwachman Diamond syndrome, etc.) will be excluded.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
Loading treatment details…
1
Study locations (1)
1 openCohen Children's Medical Center of NY
New Hyde Park, New York, United States
- Eva Atsidaftos, MA · Contact518-562-1504eatsidaf@northwell.edu
- Jeffey M Lipton, MD, PhD · Contact
- Adrianna Vlachos, MD · Contact
Recruiting
Study team
Sponsor
Northwell Health
Study ID 01.10.088G
Principal investigator
Adrianna Vlachos, MD
Cohen Children's Medical Center of NY/Feinstein Institutute for Medical Research
Study chair
Jeffrey M Lipton, MD, PhD
Cohen Children's Medical Center of NY/Feinstein Institute for Medical Research
Have questions? The contacts above can answer them before you apply.
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.