Grants and Contracts Details
Description
Task Oder 7: Regimen B: Verdiperstat OLE - Platform Trial for the Treatment of Amyotrophic Lateral Sclerosis
Regimen-Specific Appendix [B]
For verdiperstat (also known as BHV-3241).
Rationale and RSA Design
The proposed study is based on cumulative preclinical and clinical studies that implicate
myeloperoxidase (MPO) activity in the onset and progression of neurodegenerative diseases and
suggest treatment with verdiperstat at a dosage of 600 mg twice daily (BID) has the potential to
slow neurodegeneration in Amyotrophic Lateral Sclerosis (ALS).
Allocation to Treatment Regimens
Participants must first be screened under the Master Protocol before they are randomized to an RSA.
As soon as pre-defined criteria for futility for the RSA are met, or the target number of
randomized participants for the RSA has been reached, enrollment will stop in the RSA.
Number of Planned Participants and Treatment Groups
The number of planned participants for this regimen is 160.
There are 2 treatment groups for this regimen, active and placebo. Participants will be
randomized in a 3:1 ratio to active treatment or placebo (i.e., 120 active: 40 placebo).
Planned Number of Sites
Research participants will be enrolled from approximately 60 centers in the US.
Treatment Duration
The maximum duration of the placebo-controlled portion is 24 weeks.
Follow-up Duration
At the conclusion of the 24-week placebo-controlled period of the study, all participants will
either schedule a 28-day follow up phone call and end their participation in the regimen or have
the option to receive verdiperstat in the open-label extension (OLE) phase of the study. The
duration of the OLE phase is planned for 52 weeks. At the completion of the OLE, a Follow-up Safety
Call should be conducted approximately 28 days after the last dose of study drug.
For participants who withdraw early from the placebo-controlled phase, an in-person Early
Termination Visit and a Follow-up Safety Call should be conducted. At the Early Termination Visit
the same procedures as described for the Week 24 visit should be conducted. At the Follow-up Safety
Call, information on clinical status should be collected.
For participants who withdraw early from the OLE, an in-person Early Termination Visit and a
Follow-up Safety Call should be conducted. At the Early Termination Visit the same procedures as
described for the Week 52 visit should be conducted. At the Follow-up Safety Call,
information on clinical status should be collected.
Status | Finished |
---|---|
Effective start/end date | 9/4/20 → 2/22/23 |
Funding
- Massachusetts General Hospital: $7,266.00
Fingerprint
Explore the research topics touched on by this project. These labels are generated based on the underlying awards/grants. Together they form a unique fingerprint.