Long-Term Follow-Up (LTFU) for Gene Therapy of Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2022-501526-38-00

Acronym

RP-L301-0222-LTFU

Enrollment

Registered

2023-01-09

Start date

2023-06-06

Completion date

Unknown

Last updated

2025-04-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pyruvate kinase deficiency

Brief summary

Overall survival, Peripheral blood genetic correction, as demonstrated by vector copy number (VCN), Any red blood cell transfusion, Sustained and significant decrease in hemoglobin unrelated to infection or surgery, Adverse events related to worsening iron overload (for example, cirrhosis, cardiomyopathy, etc.), Any adverse event considered at least possibly related to RP-L301, Development of any malignancy

Interventions

DRUGMerilen

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Overall survival, Peripheral blood genetic correction, as demonstrated by vector copy number (VCN), Any red blood cell transfusion, Sustained and significant decrease in hemoglobin unrelated to infection or surgery, Adverse events related to worsening iron overload (for example, cirrhosis, cardiomyopathy, etc.), Any adverse event considered at least possibly related to RP-L301, Development of any malignancy	—

Measure

Time frame

—

Countries

Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026