Phase I/II Study of FP-045 in Patients With Fanconi Anemia

• Status: Not yet recruiting
• Trial end date: 2023-07-01
• Target enrollment: 0
• Participant gender: All

Summary

Phase 1 dose escalation in adolescents (ages 12-18 y.o.). Once safety established, dose escalation in pediatric subjects (3-11 y.o.) will follow. In expansion phase 20 adolescent or pediatric subjects will receive FP-045 at recommended phase 2 dose for 6 months.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Foresee Pharmaceuticals Co., Ltd.

Criteria

Inclusion Criteria:

• male or female aged 3-18

• documented Fanconi anemia by chromosome breakage analysis

• females of child-bearing potential and males required to use highly effective birth control

Phase 2 only:

• 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

Phase 1:

• Stem cell or bone marrow transplant within 3 months prior

• Active graft versus host disease requiring systemic treatment

Phase 2:

• Has a history of bone marrow or stem cell transplant