SuspendedPhase 2NCT06422351

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

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: Ami Shah, MD
Stanford University
Intervention: RP-L301(biological)
Enrollment: 10 enrolled
Eligibility: 8-55 years · All sexes
Timeline: 2024 – 2029

Study locations (3)

Stanford University, Palo Alto, 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 NCT06422351 on ClinicalTrials.gov

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