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NCT04523376 · ClinicalTrials.gov registry record · NA
Pilot Study PBSCT with TCRab Depletion for Hemoglobinopathies
A NA study of Sickle Cell Disease and Thalassemia Major, sponsored by Timothy Olson.
- Active
- Registry status
- NA
- Development phase
- 9
- Enrollment target
- 1
- Study location
NCT04523376: Active NA study of Sickle Cell Disease and Thalassemia Major, sponsored by Timothy Olson.
NCT04523376 is a NA study of Sickle Cell Disease and Thalassemia Major that is active but no longer recruiting, run by Timothy Olson. The registered enrollment target is 9 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (97% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04523376, a NA study of Sickle Cell Disease and Thalassemia Major, is active but no longer recruiting, sponsored by Timothy Olson.
- ACTIVE NOT RECRUITING
- Registry status
- NA
- Development phase
- 9 participants
- Enrollment target
- 1
- Study location
Study Summary
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM).
Primary Outcome
Number of patients with primary graft failure (defined as no evidence of neutrophil engraftment by day +30 after stem cell infusion) and secondary graft failure (defined as ANC \<500 for at least 7-10 days after initial engraftment occurs in the absence of known infection or drug-mediated suppression, and confirmed by hypocellular bone marrow biopsy and/or total donor chimerism percentage from blood or bone marrow \< 10 percent)
Conditions Studied
Interventions
- DEVICE CliniMACS
Study Locations (1)
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2020-05-14 |
| Est. Completion | 2027-06-01 |
| Phase | NA |
What the registry record for NCT04523376 still lists
NCT04523376 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (97% lower).
The record links to 2 conditions, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention - of which CliniMACS is the first listed.
NCT04523376 reports a single indexed study location in Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT04523376 about?
NCT04523376 is a clinical study titled "Pilot Study PBSCT with TCRab Depletion for Hemoglobinopathies". This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SC...
What is the current status of trial NCT04523376?
This trial is currently active not recruiting. It is a NA study. The enrollment target is 9 participants. The study started on 2020-05-14. Estimated completion is 2027-06-01.
What conditions does trial NCT04523376 study?
This clinical trial studies the following conditions: Sickle Cell Disease, Thalassemia Major.
What interventions are being tested in trial NCT04523376?
The interventions under investigation include: CliniMACS (DEVICE).
Who is sponsoring clinical trial NCT04523376?
This trial is sponsored by Timothy Olson, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04523376 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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Where NCT04523376's enrollment target sits among peer trials
9 199th of 213 higher than 14 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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