Autologous CD34+ enriched cells from patients with Fanconi anemia subtype A transduced ex vivo with lentiviral vector carrying the FANCA gene for Fanconi anemia (subtype A)

Inclusion criteria

• {"criterion_text":"- Subjects must meet all the following criteria to be included in the study: 1. Was enrolled in the clinical phase 1/2 study FANCOLEN-I."}
• {"criterion_text":"- 2. Received infusion of autologous CD34+ enriched gene corrected hematopoietic cells in clinical phase 1/2 study FANCOLEN-I."}
• {"criterion_text":"- 3. Is willing and able to adhere to the study visit schedule and other protocol requirements."}
• {"criterion_text":"- 4. Provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements."}
• {"criterion_text":"- Patients who have undergone allogeneic HSCT (either because of bone marrow failure or leukemia/MDS) will also be followed in this protocol."}
• {"criterion_text":"- Evaluations for VCN in HSCT recipients will not be performed if 3 prior assessments did not indicate presence of provirus (transgene) in any evaluated cell population."}

Exclusion criteria

• {"criterion_text":"- There are no criteria for exclusion in this study."}

Primary endpoints

• {"endpoint_text":"- Safety Assessments: will include blood-based evaluation of integration site analysis (ISA) in peripheral blood mononuclear cells (including lineage-specific subsets as determined by flow cytometry), assessment of RCL (serum and blood cells) when relevant, and detailed history regarding adverse events including hospitalizations, administration of medications or therapies for bone marrow failure, and development of hematologic and non-hematologic malignancies.","definition_or_measurement_approach":"Blood-based evaluation of integration site analysis (ISA) in PBMCs including lineage-specific subsets (flow cytometry); assessment of replication competent lentivirus (RCL) in serum and blood cells when relevant; collection of detailed adverse event history including hospitalizations, medications/therapies for bone marrow failure, and development of hematologic and non-hematologic malignancies."}

Secondary endpoints

• {"endpoint_text":"- Importantly, in settings of hematologic malignancy development, a blood sample will be obtained to enable determination of whether the malignant clone developed from a gene-corrected lineage or an uncorrected FA hematopoietic population by means of PCR for the transgene within the malignant population. Efficacy Assessments: will include blood-based evaluation of peripheral blood counts, and ongoing assessment of phenotypic correction via peripheral blood T-lymphocyte DEB chromosomal fragility","definition_or_measurement_approach":"If hematologic malignancy develops, PCR for the transgene within malignant population to determine origin (gene-corrected vs uncorrected). Efficacy: blood-based evaluation of peripheral blood counts; assessment of phenotypic correction via peripheral blood T-lymphocyte DEB chromosomal fragility assay."}

Spain

Earliest CTIS Part Ii Submission Date

13-08-2024

Latest Decision Or Authorization Date

20-03-2026

Processing Time Days

584

Number Of Sites

1

Number Of Participants

9

Primary sponsor

Full Name

Rocket Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Premier Research Group S.L.

Responsibilities

Sponsor duties codes: 1,10,11,12,2,5,6,7,8,9 (as listed in CTIS record)

Third parties

• {"country":"Spain","full_name":"Hospital De La Santa Creu I Sant Pau","duties_or_roles":"Chromosomal Fragility Test","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Germany","full_name":"Medizinische Hochschule Hannover","duties_or_roles":"ISA Assessments","organisation_type":"Educational Institution"}
• {"country":"Spain","full_name":"Premier Research Group S.L.","duties_or_roles":"Sponsor duties codes: 1,10,11,12,2,5,6,7,8,9","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Precision For Medicine Inc.","duties_or_roles":"Provides Kits/ Collection Supplies","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Genezen Laboratories Inc.","duties_or_roles":"Gene Therapy Testing; other duties (code 4)","organisation_type":"Hospital/Clinic/Other health care facility"}