Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

• Register: ClinicalTrials.gov
• Last refreshed on: 17 April 2023
• Main ID: NCT04069533
• Date of registration: 23/08/2019
• Prospective Registration: Yes
• Primary sponsor: Rocket Pharmaceuticals Inc.
• Public title: Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
• Scientific title: A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients With Fanconi Anemia Subtype A
• Date of first enrolment: November 28, 2019
• Target sample size: 7
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT04069533
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Spain

Contacts

• Name: Julián Sevilla Navarro, MD, PhD
• Affiliation: Hospital Infantil Universitario Niño Jesús (HIUNJ)

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in
the presence of diepoxybutane (DEB) or similar DNA-crosslinking agent

2. Patient of the complementation group FA-A

3. Minimum age: 1 year and minimum weight of 8 kg.

4. Maximum age: 17 years

5. At least 30 CD34+ cells/µL are determined in one BM aspiration within 3 months prior
to the CD34+ cell collection.

6. Provide informed consent in accordance with current legislation

7. 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:

1. Patients with an available and medically eligible human leukocyte antigen
(HLA)-identical sibling donor

2. Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities other
than those predictive of these conditions in bone marrow (BM) aspirate analysis. This
assessment should be made by valid studies conducted within the 3 months before the
patient enters the clinical trial

3. Patients 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 in at least one blood lineage over time
must be documented to enable eligibility)

4. Lansky performance index = 60%

5. Any concomitant disease or condition that, in the opinion of the Principal
Investigator, deems the patient unfit to participate in the trial

6. Pre-existing sensory or motor impairment >/= grade 2 according to the criteria of the
National Cancer Institute (NCI)

7. Pregnant or breastfeeding women

8. Hepatic dysfunction as defined by either:

- Bilirubin > 3 x the upper limit of normal (ULN)

- Alanine aminotransferase (ALT ) > 5 x ULN

- Aspartate aminotransferase (AST) > 5 x 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.

9. Renal dysfunction requiring either hemodialysis or peritoneal dialysis

10. Pulmonary dysfunction as defined by either:

- Need for supplemental oxygen during the prior 2 weeks (in absence of acute
infection)

- 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



Age minimum: 1 Year
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fanconi Anemia Complementation Group A

Intervention(s)

Biological: RP-L102

Primary Outcome(s)

Phenotypic correction of bone marrow colony forming units after infusion of RP-L102 [Time Frame: 3 years]

Secondary Outcome(s)

Phenotypic correction of T-lymphocytes in peripheral blood after infusion of RP-L102 [Time Frame: 3 years]

Engraftment of gene-corrected hematopoietic cells after infusion of RP-L102 [Time Frame: 3 years]

Short- and long-term Safety [Time Frame: 3 years]

Prevention or rescue of bone marrow failure [Time Frame: 3 years]

Secondary ID(s)

2018-002502-31

RP-L102-0118

Source(s) of Monetary Support

Please refer to primary and secondary sponsors