Clinical trial NCT03486353: A Study of FF-10501-01 in Combination with Azacitidine in Patients with Myelodysplastic Syndrome
Official title: A Phase 2 Study of FF-10501-01 in Combination with Azacitidine in Patients with Myelodysplastic Syndrome
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Myelodysplastic Syndrome (MDS)
Keywords: myelodysplastic syndrome (MDS)
Lead sponsor: Fujifilm Pharmaceuticals U.S.A., Inc.
Sponsor class: INDUSTRY
First posted: 2018-04-03
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: open-label, Simon 2-stage clinical study of the combination of FF-10501-01 and azacitidine in MDS patients. The Phase 2 portion of the trial will be preceded by a Phase 1 "run-in" to evaluate the safety of the combination of FF-10501-01 plus azacitidine.
Primary purpose: TREATMENT

Brief summary: The primary objective of the study is to determine the response rate according to the International Working Group Response criteria for the combination of FF-10501-01 and azacitidine in patients previously untreated with hypomethylating agents, with Int2/High risk MDS according to the IPSS, and Intermediate/High/Very-High risk MDS according to the IPSS-R, or who are otherwise candidates for treatment with azacitidine.

Detailed description: This is an open-label, Simon 2-stage clinical study of the combination of FF-10501-01 and azacitidine in previously untreated patients with high-risk MDS, or in patients with myelodysplastic syndrome (MDS) who are otherwise candidates for treatment with azacitidine in the judgment of the Investigator. The Phase 2 portion of the trial will be preceded by a Phase 1 "run-in" to evaluate the safety of the combination of FF-10501-01 plus azacitidine.

Eligibility criteria: Inclusion Criteria:

1. Male or female patients ≥ 18 years of age
2. MDS as determined by bone marrow aspirate and/or biopsy according to the WHO Classification within 6 weeks of the first dose of study medication, with bone marrow blast counts of \< 20%.
3. Int2/High-risk MDS according to the IPSS, Intermediate/high/very high-risk MDS according to the IPSS-R, or otherwise eligible for treatment with azacitidine in the judgment of the Investigator
4. ECOG Performance Score of 0 or 1
5. Adequate hepatic function as evidenced by AST/ALT \< 3X the ULN and total bilirubin \< 2X the ULN for the reference lab
6. Adequate renal function as evidenced by serum creatinine \< 2 mg/dL or a calculated creatinine clearance of \> 50 mL/min

Exclusion Criteria:

1. A current infection requiring treatment with intravenous antibiotics, anti-fungal agents, or anti-viral agents
2. Previous treatment with a hypomethylating agent, an HDAC inhibitor, or any other drug intended for the treatment of MDS other than hematopoietic growth factors, immunosuppressive therapy or hydroxyurea. Patients with 5q deletions may have received prior lenalidomide.
3. Use of hematopoietic growth factors (erythropoietin, G-CSF, GM-CSF, thrombopoietin receptor agonists) or immunosuppressive treatments within 7 days of first dose of study medication
4. Administration of any investigational agent within 5 half-lives of the agent; if the half-life is not known, use of such an agent within 2 weeks of the first dose of study medication
5. Active infection with HIV or hepatitis B or C; patients with a history of such disorders should undergo serological testing to evaluate the activity of the infection

Primary outcomes:
measure: Response Rate
timeFrame: 24 months
Secondary outcomes:
measure: Hematologic Improvement Rate
timeFrame: 24 months
measure: Duration of Response
timeFrame: 24 months
measure: Event-Free Survival
timeFrame: 24 months
measure: Progression-Free Survival
timeFrame: 24 months
Other outcomes:
Locations:
Study officials:
References: