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NCT07186179 · ClinicalTrials.gov registry record
Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)
A clinical trial of Diamond-Blackfan Anemia, sponsored by Northwell Health.
- Recruiting
- Registry status
- 10
- Enrollment target
- 1
- Study location
NCT07186179 is a study of Diamond-Blackfan Anemia that is actively recruiting participants, run by Northwell Health. The registered enrollment target is 10 participants, below the 708-participant average among 11 other Diamond-Blackfan Anemia trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state.
The verdict
NCT07186179, a study of Diamond-Blackfan Anemia, is actively recruiting participants, sponsored by Northwell Health.
- RECRUITING
- Registry status
- 10 participants
- Enrollment target
- 1
- Study location
Study Summary
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.
Conditions Studied
Interventions
- DRUG Mobilization Regimen
Study Locations (1)
New York
- Cohen Children's Medical Center - New Hyde Park
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2025-10-01 |
| Est. Completion | 2027-04-01 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07186179
The ClinicalTrials.gov registry entry for NCT07186179 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 708-participant average among 11 other Diamond-Blackfan Anemia trials with a reported enrollment target (99% lower). The listed sponsor is Northwell Health, which has 370 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Diamond-Blackfan Anemia appearing as the primary indexed condition, and to 1 intervention - of which Mobilization Regimen is the first listed.
NCT07186179 reports 1 study location spanning 1 distinct geographic area - top geographies include New York.
Frequently Asked Questions
What is clinical trial NCT07186179 about?
NCT07186179 is a clinical study titled "Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)". 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...
What is the current status of trial NCT07186179?
This trial is currently recruiting. The enrollment target is 10 participants. The study started on 2025-10-01. Estimated completion is 2027-04-01.
What conditions does trial NCT07186179 study?
This clinical trial studies the following conditions: Diamond-Blackfan Anemia.
What interventions are being tested in trial NCT07186179?
The interventions under investigation include: Mobilization Regimen (DRUG).
Who is sponsoring clinical trial NCT07186179?
This trial is sponsored by Northwell Health, which has 370 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07186179 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Related
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