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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

9 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-06-01

Study Completion Date

2035-01-30

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Fanconi Anemia Complementation Group A Fanconi Anemia

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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Subjects with Fanconi Anaemia Subtype A (FA-A)

Subjects treated with ex vivo lentiviral gene therapy product in FANCOLEN-I trial and agree to participate in this long-term follow-up (LTFU) study

Safety and efficacy assessments

Intervention Type OTHER

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

Interventions

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Safety and efficacy assessments

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

Intervention Type OTHER

Eligibility Criteria

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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

Eligible Sex

ALL

Accepts Healthy Volunteers

No