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NCT04105166 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
A Phase 1 study, sponsored by Rocket Pharmaceuticals.
- Completed
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
NCT04105166: Completed Phase 1 study, sponsored by Rocket Pharmaceuticals.
NCT04105166 is a Phase 1 study that has completed, run by Rocket Pharmaceuticals. The registered enrollment target is 4 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (93% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04105166, a Phase 1 study, has completed, sponsored by Rocket Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
Study Summary
This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
Primary Outcome
The number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute (NCI) v.5.0.
Interventions
- BIOLOGICAL RP-L301
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2020-07-06 |
| Est. Completion | 2025-06-09 |
| Phase | Phase 1 |
What the finished NCT04105166 record still lists
NCT04105166 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 4 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (93% lower).
The record links to 0 conditions, and to 1 intervention - of which RP-L301 is the first listed.
NCT04105166 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04105166 about?
NCT04105166 is a clinical study titled "Gene Therapy for Pyruvate Kinase Deficiency (PKD)". This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
What is the current status of trial NCT04105166?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2020-07-06. Estimated completion is 2025-06-09.
What interventions are being tested in trial NCT04105166?
The interventions under investigation include: RP-L301 (BIOLOGICAL).
Who is sponsoring clinical trial NCT04105166?
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 NCT04105166's enrollment target sits among peer trials
4 1995th of 2000 higher than 2 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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