Gene Therapy for Fanconi Anemia Complementation Group A

  • STATUS
    Recruiting
  • participants needed
    5
  • sponsor
    Rocket Pharmaceuticals Inc.
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Updated on 19 February 2024
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Summary

The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A).

Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.

Description

This is a pediatric open-label Phase II clinical trial to assess the efficacy of a hematopoietic gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the FANCA gene in subjects with FA-A.

Enriched CD34+ hematopoietic stem cells will be transduced ex vivo with the therapeutic lentiviral vector and infused via intravenous infusion following transduction without any prior conditioning.

Details
Condition Fanconi Anemia Complementation Group A
Age 1-100 years
Treatment RP-L102
Clinical Study IdentifierNCT04248439
SponsorRocket Pharmaceuticals Inc.
Last Modified on19 February 2024

Eligibility

 Yes No Not Sure

Inclusion Criteria

Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent
Patients of the complementation group FA-A
Minimum age: 1 year and a minimum weight of 8 kg
At least 30 CD34+ cells/L are determined in one bone marrow (BM) aspiration within 3 months prior to CD34+ cell collection OR (see subsequent criterion)
If the number of CD34+ cells/ L in BM is in the range of 10-29, peripheral blood (PB) parameters should meet two of the three following criteria
Hemoglobin: 11g/dL
Neutrophils: 900 cells/L
Platelets: 60,000 cells/L
Provide informed consent in accordance with current legislation
Women of childbearing age must have a negative urine pregnancy test at the baseline visit, and accept the use of an effective contraception method during participation in the trial

Exclusion Criteria

Subjects with an available and medically eligible HLA-identical sibling donor
Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities other than those reported as variant(s) of normal in BM aspirate analysis. This assessment should be made by valid studies conducted within the 3 months before the subject commences the stem cell mobilization/collection procedures of the clinical trial
Subjects with somatic mosaicism associated with stable or improved counts in all PB cell lineages. (If T-lymphocyte chromosomal fragility analysis indicates potential mosaicism, a medically significant decrease (1 NCI CTCAE grade) in at least one blood lineage over time must be documented to enable eligibility, as should <5% resistance of bone marrow colony forming cells (CFCs) to 10nM MMC; whenever possible potential mosaicism should also be evaluated by gene sequencing of MMC-resistant CFCs)
Lansky performance status 60%
Any concomitant disease or condition that, in the opinion of the Principal Investigator, renders the subject unfit to participate in the study
Pre-existing sensory or motor impairment grade 2 according to the criteria of the NCI
Pregnant or breastfeeding women
Hepatic dysfunction as defined by either
Bilirubin >3.0 the upper limit of normal (ULN) or
Alanine aminotransferase (ALT) > 5.0 ULN or
Aspartate aminotransferase (AST) > 5.0 ULN
For subjects with bilirubin, ALT or AST above ULN, a workup to identify the
etiology of liver abnormality should be conducted prior to confirmation of
eligibility as stipulated in exclusion criterion 5, including evaluation of
viral hepatitis, iron overload, drug injury or other causes
\. Renal dysfunction requiring either hemodialysis or peritoneal dialysis
\. Pulmonary dysfunction as defined by either
Need for supplemental oxygen during the prior 2 weeks in absence of acute infection or
Oxygen saturation by pulse oximetry <90%. 11\. Evidence of active metastatic or locoregionally advanced malignancy for which survival is anticipated to be less than 3 years. 12\. Subject is receiving androgens (i.e. danazol, oxymetholone). 13\. Subject is receiving other investigational therapy for treatment/prevention of FA-associated bone marrow failure
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