A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia

Summary

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 4 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 12-16 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.

Timeline

Start

2023-06-30

Primary completion

2025-06

Completion

2025-12

Outcome

Outcome not reported

Stopped: “Twenty months after investigative site initiations, and best efforts to identify study participants, no participants were identified.”

Drugs

Indications