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NCT03653338 · ClinicalTrials.gov registry record · Phase 1
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
A Phase 1 study of Sickle Cell Anemia and Diamond-Blackfan Anemia, sponsored by Paul Szabolcs.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
- 1
- Study location
NCT03653338: Recruiting Phase 1 study of Sickle Cell Anemia and Diamond-Blackfan Anemia, sponsored by Paul Szabolcs.
NCT03653338 is a Phase 1 study of Sickle Cell Anemia and Diamond-Blackfan Anemia that is actively recruiting participants, run by Paul Szabolcs. The registered enrollment target is 5 participants, below the 100-participant average among 22 other Sickle Cell Anemia trials with a reported enrollment target (95% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03653338, a Phase 1 study of Sickle Cell Anemia and Diamond-Blackfan Anemia, is actively recruiting participants, sponsored by Paul Szabolcs.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
- 1
- Study location
Study Summary
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.
Primary Outcome
How frequent, if any, graft rejection occurs
Conditions Studied
Interventions
- DRUG Alemtuzumab
- DRUG Hydroxyurea
- DRUG Rituximab
- BIOLOGICAL CD3/CD19 depleted leukocytes
- BIOLOGICAL CD45RA depleted leukocytes
Study Locations (1)
Pennsylvania
- Children's Hospital of Pittsburgh of UPMC - Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2018-08-02 |
| Est. Completion | 2027-08-01 |
| Phase | Phase 1 |
What NCT03653338 shows while recruiting
NCT03653338 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 100-participant average among 22 other Sickle Cell Anemia trials with a reported enrollment target (95% lower).
The record links to 3 conditions, with Sickle Cell Anemia appearing as the primary indexed condition, and to 5 interventions - of which Alemtuzumab is the first listed.
NCT03653338 reports a single indexed study location in Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT03653338 about?
NCT03653338 is a clinical study titled "T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias". The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidenti...
What is the current status of trial NCT03653338?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2018-08-02. Estimated completion is 2027-08-01.
What conditions does trial NCT03653338 study?
This clinical trial studies the following conditions: Sickle Cell Anemia, Diamond-Blackfan Anemia, Beta-thalassemia Major.
What interventions are being tested in trial NCT03653338?
The interventions under investigation include: Alemtuzumab (DRUG), Hydroxyurea (DRUG), Rituximab (DRUG), CD3/CD19 depleted leukocytes (BIOLOGICAL), CD45RA depleted leukocytes (BIOLOGICAL).
Who is sponsoring clinical trial NCT03653338?
This trial is sponsored by Paul Szabolcs, which has 7 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03653338 being conducted?
This trial has 1 study location across Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03653338's enrollment target sits among peer trials
5 22nd of 22 higher than 1 of 22 other Sickle Cell Anemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Sickle Cell Anemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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