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NCT07186179
Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)
trial testing Mobilization Regimen in Diamond Blackfan Anemia in 10 participants. Currently enrolling.
1 October 2026
Quick facts
| Lead sponsor | Northwell Health |
|---|---|
| Status | Recruiting now |
| Study type | OBSERVATIONAL |
| Enrollment | 10 |
| Start date | 1 October 2025 |
| Primary completion | 1 October 2026 |
| Estimated completion | 1 April 2027 |
| Sites | 1 location across United States |
Drugs / interventions tested
- Mobilization Regimen — full drug profile →
Conditions studied
- Diamond Blackfan Anemia — all drugs for Diamond Blackfan Anemia →
Sponsor
Northwell Health — full company profile →
Who can join
Adults 3 to 30, any sex, with Diamond Blackfan Anemia. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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 move) from the bone marrow to the peripheral blood and then collected (also called 'harvested'). It is not known if patients with DBAS can mobilize enough stem cells into the peripheral blood to allow for the harvesting of sufficient numbers to permit genetic manipulation. It is important to demonstrate the ability to harvest an adequate number of stem cells before gene therapy can be tried in patients with DBAS. The purpose of this study is to determine if mobilization of stem cells from the bone marrow in patients with DBAS is enough to obtain the numbers of peripheral blood stem cells necessary for effective gene therapy. An actual harvest will not be done.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
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Related trials
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Other Northwell Health trials
Trials by the same sponsor.
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT07186179 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Northwell Health
- Last refreshed: 22 September 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT07186179.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing