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Active, Not Recruiting

NCT Number: NCT04437771

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Fanconi Anemia Complementation Group A Anemia Anemia, Aplastic Anemia, Hypoplastic, Congenital Bone Marrow Diseases Bone Marrow Failure Disorders Congenital Bone Marrow Failure Syndromes Congenital, Hereditary, and Neonatal Diseases and Abnormalities DNA Repair-Deficiency Disorders Fanconi Anemia Fanconi Syndrome Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Kidney Diseases Male Urogenital Diseases Metabolic Diseases Nutritional and Metabolic Diseases Renal Tubular Transport, Inborn Errors Urogenital Diseases Urologic Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital Infantil Universitario Niño Jesús (HIUNJ)

Madrid, 28009, Spain

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Enrolled in the FANCOLEN-I study
  • Treated with gene therapy in the FANCOLEN-I study
  • Able to adhere to the study visit schedule and protocol requirements
  • Provided written informed consent and, as applicable, assent to participate

Exclusion criteria

  • There are no exclusion criteria for this study

Treatment and study plan

Safety and efficacy assessments

Other

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

Primary outcomes

  1. Monitor long term safety of patients through blood laboratory evaluations and general health status

    Time frame: 15 years post-drug product infusion

    Evaluate long term safety following infusion of hematopoietic cells transduced with therapeutic lentiviral vector (LV)

  2. Long term genetic correction assessed in bone marrow and blood

    Time frame: 15 years post-drug product infusion

    Determine long term persistence of therapeutic LV in hematopoietic cells in bone marrow and blood

  3. Replication competent lentivirus (RCL)

    Time frame: 15 years post-drug product infusion

    Evaluate RCL in peripheral blood

  4. Insertion site analysis in blood

    Time frame: 15 years post-drug product infusion

    Determine long term clonality

  5. Phenotypic correction

    Time frame: 15 years post-drug product infusion

    Determine phenotypic correction of bone marrow and peripheral blood cells by resistance to DNA-damaging agents

  6. Assessment for Malignancies

    Time frame: 15 years post-drug product infusion

    Monitor for incidence of hematologic malignancies and solid organ tumors

  7. Hematologic stabilization

    Time frame: 15 years post-drug product infusion

    Monitor for long term stability and normalization of blood counts

Sponsors and collaborators

Lead sponsor

Rocket Pharmaceuticals Inc.

Industry

Registry information

Official study title

Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Patients With Fanconi Anaemia Subtype A: FANCOLEN-I

Important dates

Study start
2020
Primary completion
2034
Study completion
2034
First posted
Jun 18, 2020
Registry last updated
Apr 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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