A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia
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| ClinicalTrials.gov Identifier: NCT04522375 |
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Recruitment Status :
Recruiting
First Posted : August 21, 2020
Last Update Posted : November 13, 2023
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Fanconi Anemia | Drug: FP-045 | Phase 1 Phase 2 |
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 6 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 3 weeks following a DLT will be withdrawn from the study.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.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 18 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Intervention Model Description: | Phase of dose escalation to OBD followed by 3 months of treatment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| 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) |
| Actual Study Start Date : | June 30, 2023 |
| Estimated Primary Completion Date : | June 2025 |
| Estimated Study Completion Date : | December 2025 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: FP-045
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.
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Drug: FP-045
activator of aldehyde dehydrogenase |
- The Optimal Biologic Dose (OBP) of FP-045 [ Time Frame: 28 days x up to 3 doses ]The OBP of FP-045 in adolescent and pediatric subjects
- stabilizing or improving cytopenia in FA [ Time Frame: 3 months ]Change from baseline in hemoglobin
- Safety and tolerability [ Time Frame: 3-6 months ]Frequency of adverse events and serious adverse events
- pharmacokinetic profile [ Time Frame: 3- 6 months ]Mean AUC of FP-045 by dose level
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| Ages Eligible for Study: | 3 Years to 25 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04522375
| Contact: Bassem Elmankabadi, MD | 562 310-8718 | Bassem.elmankabadi@foreseepharma.com | |
| Contact: Yisheng Lee, MD, PhD | 408 823-4807 | yisheng.lee@foreseepharma.com |
| United States, California | |
| Lucille Packard Children's Hospital, Stanford University | Recruiting |
| Palo Alto, California, United States, 94305 | |
| Contact: Rajni Agarwal-Hashmi, M.D. 650-497-0753 | |
| United States, Minnesota | |
| Masonic Cancer Center, University of Minnesota | Recruiting |
| Minneapolis, Minnesota, United States, 55455 | |
| Principal Investigator: Meera Srikanthan, MD | |
| United States, New York | |
| David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center | Recruiting |
| New York, New York, United States, 10022 | |
| Contact: Maria Cancio, M.D. 212-639-8478 canciom@mskcc.org | |
| United States, Tennessee | |
| St. Jude Childrens Research Hospital | Not yet recruiting |
| Memphis, Tennessee, United States, 38105 | |
| Contact: Olivia McGregor Olivia.McGregor@STJUDE.ORG | |
| Principal Investigator: Clifford Takemoto | |
| Study Director: | Susan Whitaker | Foresee Pharmaceuticals |
| Responsible Party: | Foresee Pharmaceuticals Co., Ltd. |
| ClinicalTrials.gov Identifier: | NCT04522375 |
| Other Study ID Numbers: |
FP045C-19-001 |
| First Posted: | August 21, 2020 Key Record Dates |
| Last Update Posted: | November 13, 2023 |
| Last Verified: | May 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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Fanconi Syndrome Anemia Fanconi Anemia Hematologic Diseases Anemia, Hypoplastic, Congenital Anemia, Aplastic Congenital Bone Marrow Failure Syndromes Bone Marrow Failure Disorders Bone Marrow Diseases Genetic Diseases, Inborn |
DNA Repair-Deficiency Disorders Metabolic Diseases Renal Tubular Transport, Inborn Errors Kidney Diseases Urologic Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Urogenital Diseases Male Urogenital Diseases |