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Expanded Access Program With Pevonedistat (Given With Azacitidine) for Adults With Higher-risk Myelodysplastic Syndromes

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ClinicalTrials.gov Identifier: NCT04484363
Expanded Access Status : No longer available
First Posted : July 23, 2020
Last Update Posted : September 30, 2022
Information provided by (Responsible Party):

Tracking Information
First Submitted Date July 22, 2020
First Posted Date July 23, 2020
Last Update Posted Date September 30, 2022
Descriptive Information
Brief Title Expanded Access Program With Pevonedistat (Given With Azacitidine) for Adults With Higher-risk Myelodysplastic Syndromes
Brief Summary

Participants in the expanded access program are adults with higher-risk myelodysplastic syndromes who have no other treatment options available.

The main aim of this program is to allow participants to have access to pevonedistat before FDA approval.

This program will take place in the United States.

Detailed Description

This is an expanded access program in which the drug being tested is called pevonedistat, which is used in combination with azcitidine. This study will provide expanded access of pevonedistat (in combination with azacitidine) for the first-line treatment to participants with HR-MDS and option of real world data (RWD) collection for the benefit of future participants.

All participants will receive azacitidine via intravenous or subcutaneous route in combination with pevonedistat intravenous infusion.

This multi-center trial will be conducted in the United States. Participant will continue treatment until benefit is no longer derived from the treatment (that is, until disease progression, or treatment is no longer tolerable), the benefit-risk no longer favors the individual, an appropriate alternative therapy becomes available, the participant chooses to discontinue the treatment, or pevonedistat becomes commercially available.

Study Type Expanded Access
Expanded Access Type Intermediate-size Population
  • Drug: Pevonedistat
    Pevonedistat 20 milligram per square meter (mg/m^2), intravenous infusion, on Days 1, 3, and 5 of repeated 28-day cycles.
  • Drug: Azacitidine
    Azacitidine 75 mg/m^2, intravenous or subcutaneous infusion, on Days 1 to 5, Days 8 and 9 of repeated 28-day cycles or Days 1 through 7 of repeated 28-day cycles.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status No longer available
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Not Provided
Removed Location Countries  
Administrative Information
NCT Number NCT04484363
Current Responsible Party Takeda
Original Responsible Party Millennium Pharmaceuticals, Inc.
Current Study Sponsor Takeda
Original Study Sponsor Millennium Pharmaceuticals, Inc.
Collaborators Not Provided
Study Director: Study Director Takeda
PRS Account Takeda
Verification Date September 2022