ong-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

Phase 1

Recruiting

• Conditions: Pyruvate kinase deficiency; MedDRA version: 21.1Level: PTClassification code: 10037682Term: Pyruvate kinase deficiency anaemia Class: 100000004850; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

• Registration Number: CTIS2022-501526-38-00
• Lead Sponsor: Rocket Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-09-20
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

Patient was enrolled in the parent Study RP-L301-0119, Patient received an autologous infusion of CD34+ enriched cells transduced ex vivo with a lentiviral vector carrying the coRPK gene, Patient is willing and able to adhere to the study visit schedule and other protocol requirements, Patient provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements

Exclusion Criteria

There are no exclusion criteria for the current study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified