Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy
Launched by ROCKET PHARMACEUTICALS INC. · Jun 16, 2020
Trial Information
Current as of June 26, 2025
Enrolling by invitation
Keywords
ClinConnect Summary
This long-term follow-up protocol will evaluate the long term safety and efficacy of the infusion of autologous CD34+ cells transduced with lentiviral vector (LV) carrying the FANCA gene.
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • 1. Enrolled in the FANCOLEN-I study
- • 2. Treated with gene therapy in the FANCOLEN-I study
- • 3. Able to adhere to the study visit schedule and protocol requirements
- • 4. Provided written informed consent and, as applicable, assent to participate
- Exclusion Criteria:
- • There are no exclusion criteria for this study
About Rocket Pharmaceuticals Inc.
Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company focused on advancing transformative gene therapies for rare and devastating diseases. With a robust pipeline centered on innovative, potentially curative treatments, Rocket Pharmaceuticals employs cutting-edge technology and a patient-centric approach to address unmet medical needs. The company is committed to leveraging its expertise in genetic medicine to develop therapies that can significantly improve the quality of life for patients and their families. Through rigorous research and clinical trials, Rocket Pharmaceuticals aims to pioneer breakthroughs in the field of gene therapy, establishing itself as a leader in the biopharmaceutical industry.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Madrid, , Spain
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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