Back to resultsA Study of FF-10501-01 in Combination With Azacitidine in Patients With Myelodysplastic Syndrome
Primary Purpose
Myelodysplastic Syndrome (MDS)
Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
FF-10501-01
Azacitidine
Sponsored by
About this trial
This is an interventional treatment trial for Myelodysplastic Syndrome (MDS) focused on measuring myelodysplastic syndrome (MDS)
Eligibility Criteria
Inclusion Criteria:
- Male or female patients ≥ 18 years of age
- 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%.
- 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
- ECOG Performance Score of 0 or 1
- Adequate hepatic function as evidenced by AST/ALT < 3X the ULN and total bilirubin < 2X the ULN for the reference lab
- Adequate renal function as evidenced by serum creatinine < 2 mg/dL or a calculated creatinine clearance of > 50 mL/min
Exclusion Criteria:
- A current infection requiring treatment with intravenous antibiotics, anti-fungal agents, or anti-viral agents
- 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.
- 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
- 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
- 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
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Run-In Phase, Regimen 1
Run-In Phase, Regimen 2
Arm Description
Patients will be treated with FF-10501-01 at a dose of 400 mg/m2 twice daily (BID) for 14 days plus azacitidine at a dose of 75 mg/m2 either subcutaneously (SC) or intravenously (IV) x 7 days every 28 days. One treatment cycle will be 28 days in duration.
Patients will be treated with FF-10501-01 at a dose of 400 mg/m2 BID for 21 days plus azacitidine at a dose of 75 mg/m2 either SC or IV x 7 days every 28 days.
Outcomes
Primary Outcome Measures
Response Rate
The primary efficacy assessment will be the objective response rate, composed of those patients who achieve a best response of CR, mCR, PR or HI based on the Modified IWG Response Criteria in MDS
Secondary Outcome Measures
Hematologic Improvement Rate
Determination of the hematologic improvement rate for erythroid, platelet and/or neutrophil response. The number of patients who achieve trilineage hematologic improvement will be reported, as will the number who achieve improvement in each of the cell series (i.e., erythroid (HI-E), platelet (HI-P), and/or neutrophil (HI-N).
Duration of Response
Duration of response will be measured from the date of initial response until failure (includes death from any cause), relapse (after CR or PR) or progression, as defined by the Modified IWG Response Criteria in MDS.
Event-Free Survival
Duration of event free survival will be measured from the start of treatment until failure (as defined in the Modified IWG Response Criteria) or death from any cause.
Progression-Free Survival
Duration of progression free survival will be measured from the start of treatment until progression (as defined in the Modified IWG Response Criteria) or death from MDS.
Full Information
NCT ID
NCT03486353
First Posted
March 27, 2018
Last Updated
July 20, 2021
Sponsor
Fujifilm Pharmaceuticals U.S.A., Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03486353
Brief Title
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
2. Study Status
Record Verification Date
July 2021
Overall Recruitment Status
Withdrawn
Why Stopped
Program terminated
Study Start Date
October 2019 (Anticipated)
Primary Completion Date
October 2019 (Anticipated)
Study Completion Date
October 2019 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Fujifilm Pharmaceuticals U.S.A., Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndrome (MDS)
Keywords
myelodysplastic syndrome (MDS)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
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.
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Run-In Phase, Regimen 1
Arm Type
Experimental
Arm Description
Patients will be treated with FF-10501-01 at a dose of 400 mg/m2 twice daily (BID) for 14 days plus azacitidine at a dose of 75 mg/m2 either subcutaneously (SC) or intravenously (IV) x 7 days every 28 days. One treatment cycle will be 28 days in duration.
Arm Title
Run-In Phase, Regimen 2
Arm Type
Experimental
Arm Description
Patients will be treated with FF-10501-01 at a dose of 400 mg/m2 BID for 21 days plus azacitidine at a dose of 75 mg/m2 either SC or IV x 7 days every 28 days.
Intervention Type
Drug
Intervention Name(s)
FF-10501-01
Intervention Description
FF-10501-01 round film-coated tablets are immediate release and come in 3 dosage strengths: 50 mg (tan), 100 mg tablets (red) and 200 mg (yellow). Each tablet contains the active ingredient (FF-10501-01 white crystalline powder) and other excipients. All dosage strengths are packaged 32 tablets to a bottle. FF-10501-01 should be stored at room temperature (20 - 25 °C).
Intervention Type
Drug
Intervention Name(s)
Azacitidine
Intervention Description
azacitidine at a dose of 75 mg/m2 either subcutaneously (SC) or intravenously (IV) x 7 days every 28 days
Primary Outcome Measure Information:
Title
Response Rate
Description
The primary efficacy assessment will be the objective response rate, composed of those patients who achieve a best response of CR, mCR, PR or HI based on the Modified IWG Response Criteria in MDS
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Hematologic Improvement Rate
Description
Determination of the hematologic improvement rate for erythroid, platelet and/or neutrophil response. The number of patients who achieve trilineage hematologic improvement will be reported, as will the number who achieve improvement in each of the cell series (i.e., erythroid (HI-E), platelet (HI-P), and/or neutrophil (HI-N).
Time Frame
24 months
Title
Duration of Response
Description
Duration of response will be measured from the date of initial response until failure (includes death from any cause), relapse (after CR or PR) or progression, as defined by the Modified IWG Response Criteria in MDS.
Time Frame
24 months
Title
Event-Free Survival
Description
Duration of event free survival will be measured from the start of treatment until failure (as defined in the Modified IWG Response Criteria) or death from any cause.
Time Frame
24 months
Title
Progression-Free Survival
Description
Duration of progression free survival will be measured from the start of treatment until progression (as defined in the Modified IWG Response Criteria) or death from MDS.
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female patients ≥ 18 years of age
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%.
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
ECOG Performance Score of 0 or 1
Adequate hepatic function as evidenced by AST/ALT < 3X the ULN and total bilirubin < 2X the ULN for the reference lab
Adequate renal function as evidenced by serum creatinine < 2 mg/dL or a calculated creatinine clearance of > 50 mL/min
Exclusion Criteria:
A current infection requiring treatment with intravenous antibiotics, anti-fungal agents, or anti-viral agents
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.
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
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
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
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Study of FF-10501-01 in Combination With Azacitidine in Patients With Myelodysplastic Syndrome
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