A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03814408
• Date of registration: 14/01/2019
• Prospective Registration: No
• Primary sponsor: Rocket Pharmaceuticals Inc.
• Public title: A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
• Scientific title: A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A
• Date of first enrolment: January 11, 2019
• Target sample size: 2
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03814408
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Agnieszka Czechowicz, MD
• Affiliation: Stanford University, Stem Cell Transplantation and Regenerative Medicine

Key inclusion & exclusion criteria

Inclusion Criteria:

- Fanconi anemia, as diagnosed by chromosomal fragility assay of cultured T-lymphocytes
in the presence of DEB or a similar DNA-crosslinking agent.

- Subjects of Fanconi Anemia complementation group A.

- Minimum age: 1 year and a minimum of 8 kg.

- Maximum age: 12 years.

- At least one of the following hematologic parameters below lower limits of normal:

- Hemoglobin

- Absolute neutrophils

- Platelets

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

- If the number of C34+ cells/ µL in BM is in the range of 10-29, PB parameters should
meet two of the three following criteria:

- Hemoglobin: =11g/dL

- Neutrophils: =900 cells/µL

- Platelets: =60,000 cells/µL

- 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 human leukocyte antigen
(HLA)-identical sibling donor.

- Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities
predictive of these conditions 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 in at least one blood lineage over time
must be documented to enable eligibility).

- 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 NCI CTCAE v5.0
criteria.

- Pregnant or breastfeeding women.

- Hepatic dysfunction as defined by either:

- Bilirubin >3.0 × upper limit of normal (ULN) or

- Alanine aminotransferase (ALT) > 5.0 × ULN or

- Aspartate aminotransferase (AST) > 5.0 × ULN

- 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.

- Oxygen saturation by pulse oximetry <90%.

- Evidence of active metastatic or locoregionally advanced malignancy for which survival
is anticipated to be less than 3 years.

- Subject is receiving androgens (i.e. danazol, oxymethalone).



Age minimum: 1 Year
Age maximum: 12 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fanconi Anemia Complementation Group A

Intervention(s)

Biological: RP-L102

Primary Outcome(s)

Number of participants with treatment-related adverse events as assessed by CTCAE v.5.0 [Time Frame: 3 years]

Secondary Outcome(s)

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

Phenotypic correction of hematopoietic cells in bone marrow after infusion of RP-L102 [Time Frame: 3 years]

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

Prevention or rescue of bone marrow failure after infusion of RP-L102 [Time Frame: 3 years]

Secondary ID(s)

RP-L102-0418

Source(s) of Monetary Support

Please refer to primary and secondary sponsors