A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia (NCT04522375) | Clinical Trial Compass
WithdrawnPhase 1/2
A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia
Stopped: Twenty months after investigative site initiations, and best efforts to identify study participants, no participants were identified.
0Started 2023-06-30
Plain-language 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.
Who can participate
Age range
3 Years – 35 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* male or female aged 3-35
* 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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.