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.
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT04522375.
Dose escalation will begin with young adult/adolescent patients. The initial two patients
enrolled in the study will be > 15 years of age. These patients must complete the entire
28-day period of treatment at Dose Level 1 prior to additional young adult/adolescent
patients being enrolled. All 4 young adult/adolescent patients must complete 28 days of
treatment at Dose Level 1, and cumulative safety must be reviewed by the Safety Review
Committee (SRC), prior to the enrollment of pediatric patients. The initial two pediatric
patients enrolled will be > 6 years of age. These patients must complete the entire
28-day period of treatment at Dose Level 1 prior to additional pediatric patients being
enrolled. A minimum of 8 and maximum of 12 pediatric patients will be enrolled to allow
for at least 4 patients between the ages of 3-6.
Study assessments will be conducted at each visit. Patients will be observed closely for
Dose Limiting Toxicity (DLT) during each dosing period. Any patient experiencing a DLT
will have study drug interrupted and will not be allowed to escalate to the next higher
dose level. The patient may resume treatment at one dose level lower once the DLT has
resolved to baseline or to ≤ Grade 1 in severity. The MTD will be defined as the dose
level immediately below the dose level at which DLT occurred. Patients requiring an
interruption in treatment of > 3 weeks following a DLT will be withdrawn from the study.
The MTD will be assessed separately for each individual patient.
Following the completion of dose escalation, each patient will continue treatment at
either the highest dose or their individual MTD, and then transition to the OBD for their
age group (once defined), for a total of 3 months. Patients failing to receive 75% of
planned doses for reasons other than adverse effects may be replaced.
Lead OrganizationForesee Pharmaceuticals Co., Ltd.
