Stopped: Twenty months after investigative site initiations, and best efforts to identify study participants, no participants were identified.
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.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
The Optimal Biologic Dose (OBP) of FP-045
Timeframe: 28 days x up to 3 doses
stabilizing or improving cytopenia in FA
Timeframe: 3 months