A Study of FF-10501-01 in Combination with Azacitidine in Patients with Myelodysplastic Syndrome

NCT03486353 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: FF1050101US201
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Myelodysplastic Syndrome (MDS)

Keywords

myelodysplastic syndrome (MDS)

Outcome Measures

Primary Outcomes (1)

• 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

  24 months

Secondary Outcomes (4)

• Hematologic Improvement Rate

  24 months

• Duration of Response

  24 months

• Event-Free Survival

  24 months

• Progression-Free Survival

  24 months

Study Arms (2)

Run-In Phase, Regimen 1

EXPERIMENTAL

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.

Drug: FF-10501-01; Drug: Azacitidine

Run-In Phase, Regimen 2

EXPERIMENTAL

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.

Drug: FF-10501-01; Drug: Azacitidine

Interventions

FF-10501-01 DRUG

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).

Run-In Phase, Regimen 1; Run-In Phase, Regimen 2

Azacitidine DRUG

azacitidine at a dose of 75 mg/m2 either subcutaneously (SC) or intravenously (IV) x 7 days every 28 days

Run-In Phase, Regimen 1; Run-In Phase, Regimen 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Fujifilm Pharmaceuticals U.S.A., Inc.: lead

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: March 27, 2018
• First Posted: April 3, 2018
• Study Start: October 1, 2019
• Primary Completion: October 1, 2019
• Study Completion: October 1, 2019
• Last Updated: December 5, 2024

Record last verified: 2021-07

Data Sharing

• IPD Sharing: Will not share