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NCT03812263 · ClinicalTrials.gov registry record · Phase 1
A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
A Phase 1 study, sponsored by Rocket Pharmaceuticals.
- Completed
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
NCT03812263 is a Phase 1 study that has completed, run by Rocket Pharmaceuticals. The registered enrollment target is 9 participants.
The verdict
NCT03812263, a Phase 1 study, has completed, sponsored by Rocket Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
Study Summary
The primary purpose of the Phase I portion of the study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with the therapeutic lentiviral vector, Chim-CD18-WPRE, RP-L201. The primary objectives of the Phase II portion of the study are evaluation of survival, as determined by the proportion of subjects alive at age 2 (24 months) and at least 1-year post-infusion without allogeneic hematopoietic stem cell transplant (HSCT) and characterization of the safety and toxicity associated with the infusion.
Interventions
- BIOLOGICAL RP-L201
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2019-08-30 |
| Est. Completion | 2023-09-12 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03812263
The ClinicalTrials.gov registry entry for NCT03812263 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 9 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Rocket Pharmaceuticals, which has 10 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which RP-L201 is the first listed.
NCT03812263 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03812263 about?
NCT03812263 is a clinical study titled "A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I". The primary purpose of the Phase I portion of the study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with the therapeutic lentiviral vector, Chim-CD18-WPRE, RP-L201. The primary object...
What is the current status of trial NCT03812263?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2019-08-30. Estimated completion is 2023-09-12.
What interventions are being tested in trial NCT03812263?
The interventions under investigation include: RP-L201 (BIOLOGICAL).
Who is sponsoring clinical trial NCT03812263?
This trial is sponsored by Rocket Pharmaceuticals, which has 10 total clinical trials registered on ClinicalTrials.gov.
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