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NCT06422351
A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Subjects With Pyruvate Kinase Deficiency
Phase 2 trial testing RP-L301 in Pyruvate Kinase Deficiency in 10 participants. Suspended.
1 January 2029
Quick facts
| Lead sponsor | Rocket Pharmaceuticals Inc. |
|---|---|
| Phase | Phase 2 |
| Status | Suspended |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 10 |
| Start date | 1 April 2026 |
| Primary completion | 1 January 2029 |
| Estimated completion | 1 January 2029 |
| Sites | 3 locations across United States, Spain |
Drugs / interventions tested
- RP-L301 — full drug profile →
Conditions studied
- Pyruvate Kinase Deficiency — all drugs for Pyruvate Kinase Deficiency →
Sponsor
Rocket Pharmaceuticals Inc. — full company profile →
Who can join
Adults 8 to 55, any sex, with Pyruvate Kinase Deficiency. Patients with the condition only — healthy volunteers not accepted.
What's being measured
Primary outcomes are the specific endpoints the trial is designed to prove or disprove.
-
Improvement in Anemia
Time frame: 12 months post-infusion
Hemoglobin (Hb) level increase of ≥1.5g/dL at 12 months post-infusion, compared to baseline.
Sponsor's own description
This is an open-label Phase II trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT06422351
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
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Related trials
Other trials of RP-L301
Trials testing the same drug.
- NCT04105166 — Gene Therapy for Pyruvate Kinase Deficiency (PKD) · Phase 1 · completed
Other recruiting trials for Pyruvate Kinase Deficiency
Currently open trials in the same condition.
- NCT04902833 — Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms · active not recruiting
- NCT03481738 — Pyruvate Kinase Deficiency Global Longitudinal Registry · active not recruiting
Other Rocket Pharmaceuticals Inc. trials
Trials by the same sponsor.
- NCT05548855 — Natural History of Danon Disease · completed
- NCT06282432 — Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) · active not recruiting
- NCT04525352 — A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis · Phase 1 · terminated
- NCT04248439 — Gene Therapy for Fanconi Anemia, Complementation Group A · Phase 2 · active not recruiting
- NCT04105166 — Gene Therapy for Pyruvate Kinase Deficiency (PKD) · Phase 1 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT06422351 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Rocket Pharmaceuticals Inc.
- Last refreshed: 15 April 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06422351.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing