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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 is a Phase 1 study that has completed, run by Rocket Pharmaceuticals. The registered enrollment target is 4 participants.
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).
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04105166
The ClinicalTrials.gov registry entry for NCT04105166 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 4 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-L301 is the first listed.
NCT04105166 reports 0 study locations.
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.
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