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Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

NCT04437771 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 9

Last updated 2026-04-13

No results posted yet for this study

Summary

This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial. After completion of the FANCOLEN-I study, eligible subjects will be followed for a total of 15 years post gene therapy treatment. No investigational drug product will be administered during this study.

Conditions

  • Fanconi Anemia Complementation Group A
  • Fanconi Anemia

Interventions

OTHER

Safety and efficacy assessments

Long term disease and gene therapy specific safety evaluations and efficacy assessments

Sponsors & Collaborators

  • Rocket Pharmaceuticals Inc.

    lead INDUSTRY

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-06-01
Primary Completion
2034-01-17
Completion
2034-01-17

Countries

  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04437771 on ClinicalTrials.gov