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

NCT04522375 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: FP045C-19-001
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Fanconi Anemia

Outcome Measures

Primary Outcomes (2)

• The Optimal Biologic Dose (OBP) of FP-045

  The OBP of FP-045 in adolescent and pediatric subjects

  28 days x up to 3 doses

• stabilizing or improving cytopenia in FA

  Change from baseline in hemoglobin

  3 months

Secondary Outcomes (2)

• Safety and tolerability

  3-6 months

• pharmacokinetic profile

  3- 6 months

Study Arms (1)

FP-045

EXPERIMENTAL

The study will enroll a total of 6 young adult/adolescent patients progressing through three dose levels, followed by a minimum of 8 and up to 12 pediatric patients progressing through up to three dose levels.

Drug: FP-045

Interventions

FP-045 DRUG

activator of aldehyde dehydrogenase

FP-045

Eligibility Criteria

• Age: 3 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Foresee Pharmaceuticals Co., Ltd.: lead

MeSH Terms

Conditions

Fanconi Anemia

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, Congenital; Anemia, Aplastic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Susan Whitaker

  Foresee Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Phase of dose escalation to OBD followed by 3 months of treatment

Study Record Dates

• First Submitted: August 3, 2020
• First Posted: August 21, 2020
• Study Start: June 30, 2023
• Primary Completion: June 1, 2025
• Study Completion: December 1, 2025
• Last Updated: March 6, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share