A Multinational, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity of FP 045 in Patients With Fanconi Anemia (FusuciA Study)

FP045C-19-001

This is a multi-center, Phase 1/2 study to determine the Optimal Biologic Dose (OBD) and to evaluate the safety, tolerability, PK, and preliminary activity of FP 045 when administered orally in young adult/adolescent and pediatric patients with Fanconi anemia. The study will enroll a total of 6 young adult/adolescent patients and a minimum of 8 and up to 12 pediatric patients with mild-moderate bone marrow failure who have not undergone hematopoietic cell transplant. This makes the total patient number between 14-18 total. Dose escalation will occur individually for each patient, within each age group. Each patient will receive each of 3 dose levels of FP 045 (intra-patient dose escalation), beginning with Dose Level 1, followed by Dose Levels 2 and 3. Each dose level will be administered for 28 days prior to escalation to the next higher dose level for that patient.

2023-06-30

2025-06-01

2025-12-01

Recruiting

Early phase I

18

Inclusion Criteria: * male or female aged 3-25 * documented Fanconi anemia by chromosome breakage analysis * females of child-bearing potential and males required to use highly effective birth control * mild to moderate bone marrow failure with at least one cytopenia of \> grade 1 severity Exclusion Criteria: * history of any malignancy except focal squamous cell or basal cell carcinoma of the skin or carcinoma in situ of cervix * has myelodysplastic syndrome or acute leukemia per world health organization (WHO) criteria * has history of any significant medical conditions * has aspartate aminotransferase (AST)/alanine aminotransferase (ALT) \> 5x upper limit of normal (ULN) or calculated creatinine clearance (Clcr) of \< 50 mL/min * has active Hepatitis B or C * has an ongoing systemic infection * requires a strong CYP3A4 inhibitor * has had major surgery within 30 days * Active graft versus host disease requiring systemic treatment * Has a history of bone marrow or stem cell transplant

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Phase of dose escalation to OBD followed by 3 months of treatment', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 18, 'type': 'ESTIMATED'}}

2023-11-13