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NCT03263559 · ClinicalTrials.gov registry record · Phase 2
Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
A Phase 2 study of Sickle Cell Disease, sponsored by Medical College of Wisconsin.
- Completed
- Registry status
- Phase 2
- Development phase
- 95
- Enrollment target
- 20
- Study locations
NCT03263559: Completed Phase 2 study of Sickle Cell Disease, sponsored by Medical College of Wisconsin.
NCT03263559 is a Phase 2 study of Sickle Cell Disease that has completed, run by Medical College of Wisconsin. The registered enrollment target is 95 participants, below the 292-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (67% lower). The trial reports 20 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03263559, a Phase 2 study of Sickle Cell Disease, has completed, sponsored by Medical College of Wisconsin.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 95 participants
- Enrollment target
- 20
- Study locations
Study Summary
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with severe SCD; and (2) adults with severe SCD.
Primary Outcome
EFS is defined as survival without a qualifying event from transplant. Primary graft failure (PGF), secondary graft failure (SGF), second infusion of hematopoietic cells, or death from any cause will count as events for this endpoint. This endpoint was adjudicated by an Endpoint Review Committee. PGF is lack of engraftment on or before Day 42 post-transplant. Engraftment is defined as having greater than or equal to 5% donor cells post-transplant, from any molecular chimerism assessment (e.g.,
Conditions Studied
Interventions
- DRUG Fludarabine
- DRUG Hydroxyurea
- DRUG Thiotepa
- PROCEDURE Haploidentical Bone Marrow Transplantation
- DRUG Rabbit-ATG
Study Locations (20)
Florida
- University of Florida College of Medicine - Gainesville
- Nicklaus Children's Hospital/University of Miami Children's Hospital - Miami
- H. Lee Moffitt Cancer Center - Tampa
Indiana
- Riley Children's Hospital at IU Health - Indianapolis
- Indiana University Medical Center - Indianapolis
Michigan
- Children's Hospital of Michigan - Detroit
- Helen Devos Children's at Spectrum Health - Grand Rapids
Missouri
- Cardinal Glennon Children's Hospital - St Louis
- Washington University, St. Louis - St Louis
New York
- Roswell Park Cancer Center - Buffalo
- Northwell Health/Monter Cancer Center - Lake Success
North Carolina
- Levine Children's Hospital - Charlotte
- Duke University Medical Center - Durham
Alabama
- University of Alabama at Birmingham - Birmingham
California
- UCSF Benioff Children's Hospital Oakland - Oakland
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 95 participants |
| Start Date | 2017-10-03 |
| Est. Completion | 2025-01-29 |
| Phase | Phase 2 |
What the finished NCT03263559 record still lists
NCT03263559 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 95 participants, a relatively small participant target, below the 292-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (67% lower).
The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 5 interventions - of which Fludarabine is the first listed.
NCT03263559 names 20 study sites across 13 states, led by Florida, Indiana, Michigan.
Frequently Asked Questions
What is clinical trial NCT03263559 about?
NCT03263559 is a clinical study titled "Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)". This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with severe S...
What is the current status of trial NCT03263559?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 95 participants. The study started on 2017-10-03. Estimated completion is 2025-01-29.
What conditions does trial NCT03263559 study?
This clinical trial studies the following conditions: Sickle Cell Disease.
What interventions are being tested in trial NCT03263559?
The interventions under investigation include: Fludarabine (DRUG), Hydroxyurea (DRUG), Thiotepa (DRUG), Haploidentical Bone Marrow Transplantation (PROCEDURE), Rabbit-ATG (DRUG).
Who is sponsoring clinical trial NCT03263559?
This trial is sponsored by Medical College of Wisconsin, which has 447 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03263559 being conducted?
This trial has 20 study locations across Alabama, California, Colorado, District of Columbia, Florida. 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 NCT03263559's enrollment target sits among peer trials
95 69th of 213 higher than 145 of 213 other Sickle Cell Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Sickle Cell Disease 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.
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