Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)
Recruiting
Observational Study
Diamond Blackfan Anemia
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 3 – 30
- Sex
- Any
- Study type
- Observational
- Participants needed
- 10 (estimated)
- Sponsor
- Northwell Health · Other
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About This Trial
Gene therapy is a new possible treatment for the anemia of DBAS. Gene therapy will soon be available for patients with RPS19-mutated DBAS. This involves inserting the corrected RPS19 gene into the cells, leading to correction of the anemia. The application of gene therapy requires sufficient numbers of stem cells on which the correction can be performed. Stem cells must be mobilized (stimulated to…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. Diamond Blackfan anemia syndrome as defined by the known criteria with a known gene mutation
2. Male or female patients of all ethnic background, greater than or equal to 3 years of age and weighing at least 10 kg, and less than or equal to 30 years of age
3. Enrolled in Diam…
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