Gene Therapy for Pyruvate Kinase Deficiency (PKD)
← catalyst calendarSponsored by Rocket Pharmaceuticals Inc. (industry) · RCKT — their whole pipeline →.
Phase
Phase 1
Status
Completed
Study type
Interventional
Design
Na · None · Treatment
Enrollment
4actual
Sites
3
Countries
Spain, United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2020-07-06 | actual | When the study began enrolling |
| Primary completion | 2025-06-09 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2025-06-09 | actual | The whole study's end, after follow-up |
| First posted | 2019-09-26 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-09-05 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Pyruvate Kinase Deficiency
Intervention
- Biological: RP-L301
Primary outcome
what the primary-completion date above is the date OFEvaluation of the safety and toxicity of RP-L301: number of participants with treatment-related adverse events
measured 2 years
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