CompletedPhase 1NCT04105166

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Studying Hemolytic anemia due to red cell pyruvate kinase deficiency

Last synced from ClinicalTrials.gov May 10, 2026

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Key facts

Sponsor: Rocket Pharmaceuticals Inc.
Principal Investigator: José Luis López Lorenzo, MD
Hospital Universitario Fundación Jiménez Díaz
Intervention: RP-L301(biological)
Enrollment: 4 enrolled
Eligibility: 8-50 years · All sexes
Timeline: 2020 – 2025

Study locations (3)

Stanford University, Stanford, California, United States
Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT04105166 on ClinicalTrials.gov

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