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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: Completed Phase 1 study, sponsored by Rocket Pharmaceuticals.
NCT03812263 is a Phase 1 study that has completed, run by Rocket Pharmaceuticals. The registered enrollment target is 9 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Evaluation of safety associated with treatment with RP-L201
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 |
What the finished NCT03812263 record still lists
NCT03812263 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 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower).
The record links to 0 conditions, and to 1 intervention - of which RP-L201 is the first listed.
NCT03812263 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03812263's enrollment target sits among peer trials
9 1955th of 2000 higher than 42 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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