Fanconi Anemia Clinical Trial
Official title:
A Multinational, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity of FP 045 in Patients With Fanconi Anemia (FusuciA Study)
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 6 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 14-18 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.
Status | Recruiting |
Enrollment | 18 |
Est. completion date | December 2025 |
Est. primary completion date | June 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 3 Years to 25 Years |
Eligibility | Inclusion Criteria: - male or female aged 3-25 - 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 |
Country | Name | City | State |
---|---|---|---|
United States | St. Jude Childrens Research Hospital | Memphis | Tennessee |
United States | Masonic Cancer Center, University of Minnesota | Minneapolis | Minnesota |
United States | David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center | New York | New York |
United States | Lucille Packard Children's Hospital, Stanford University | Palo Alto | California |
Lead Sponsor | Collaborator |
---|---|
Foresee Pharmaceuticals Co., Ltd. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | |
Primary | stabilizing or improving cytopenia in FA | Change from baseline in hemoglobin | 3 months | |
Secondary | Safety and tolerability | Frequency of adverse events and serious adverse events | 3-6 months | |
Secondary | pharmacokinetic profile | Mean AUC of FP-045 by dose level | 3- 6 months |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT02931071 -
Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1
|
Phase 2 | |
Terminated |
NCT01319851 -
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
|
N/A | |
Recruiting |
NCT00084695 -
Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases
|
Phase 2 | |
Completed |
NCT00000603 -
Cord Blood Stem Cell Transplantation Study (COBLT)
|
Phase 2 | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Recruiting |
NCT01146210 -
Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia
|
N/A | |
Completed |
NCT01082133 -
Multicenter Transplant Study for Fanconi Anemia
|
Phase 2 | |
Completed |
NCT00965666 -
Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia
|
Early Phase 1 | |
Terminated |
NCT00290628 -
Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer
|
N/A | |
Recruiting |
NCT00027274 -
Cancer in Inherited Bone Marrow Failure Syndromes
|
||
Terminated |
NCT01001598 -
Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
|
Phase 1/Phase 2 | |
Recruiting |
NCT03206086 -
Eltrombopag for People With Fanconi Anemia
|
Phase 2 | |
Recruiting |
NCT03579875 -
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
|
Phase 2 | |
Recruiting |
NCT05598515 -
Time-restricted Feeding to Reduce Inflammation in Fanconi Anemia
|
N/A | |
Active, not recruiting |
NCT03476330 -
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
|
Phase 2 | |
Terminated |
NCT05910853 -
Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
|
||
Completed |
NCT03609840 -
Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
|
||
Completed |
NCT00479115 -
Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100
|
Phase 1/Phase 2 | |
Completed |
NCT00352976 -
TBI Dose De-escalation for Fanconi Anemia
|
Phase 2/Phase 3 | |
Terminated |
NCT03600909 -
A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia
|
Phase 2 |