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A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy (RESILIENT)

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ClinicalTrials.gov Identifier: NCT05337553
Recruitment Status : Recruiting
First Posted : April 20, 2022
Last Update Posted : September 21, 2023
Sponsor:
Information provided by (Responsible Party):
Biohaven Pharmaceuticals, Inc.

Study Description
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Brief Summary:
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo.

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy Neuromuscular Diseases SMA Drug: taldefgrobep alfa Drug: Placebo Phase 3

Detailed Description:
Myostatin is a negative regulator of muscle growth. Blocking myostatin activity has been shown to increase muscle size and function. Taldefgrobep alfa directly blocks myostatin activity and was well tolerated in other clinical studies. In combination with medications that increase the amount of SMN protein in the body, taldefgrobep alfa has the potential to further improve motor function and clinical measures for people living with SMA.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 225 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension
Actual Study Start Date : July 6, 2022
Estimated Primary Completion Date : January 2025
Estimated Study Completion Date : January 2025

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Arms and Interventions
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Arm Intervention/treatment
Experimental: taldefgrobep alfa

taldefgrobep alfa - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase.

taldefgrobep alfa/taldefgrobep alfa - Extension Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week Open label Extension (OLE) phase.

 Drug: taldefgrobep alfa
DB Phase: 35 mg/50 mg weekly subcutaneous injection
Other Names:
  • BHV-2000
  • BMS-986089

Drug: taldefgrobep alfa
Extension Phase: 35 mg/50 mg weekly subcutaneous injection
Other Names:
  • BHV-2000
  • BMS-986089
Placebo Comparator: Placebo

Placebo - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase.

Placebo/taldefgrobep alfa - Extension Phase: Participants who receive placebo during DB phase, receive weight based 35 mg/50 mg weekly subcutaneous taldefgrobep alfa injection for 48-week OLE phase.

 Drug: Placebo
DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection

Drug: taldefgrobep alfa
Extension Phase: 35 mg/50 mg weekly subcutaneous injection
Other Names:
  • BHV-2000
  • BMS-986089


Outcome Measures
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Primary Outcome Measures :
  1. Efficacy of taldefgrobep alfa compared to placebo in change in the 32 item Motor Function Measure (MFM-32) total score [ Time Frame: Baseline to Week 48 ]
    Change in MFM-32 total score from baseline to Week 48. Scores range from 0-3 on each item. The higher the score, indicates higher functioning.


Eligibility Criteria
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Ages Eligible for Study:   4 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number
  • Ambulant or Non-Ambulant
  • Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the trial including nusinersen, risdiplam, and/or a history of onasemnogene abeparvovec

Key Exclusion Criteria:

  • Cannot have previously taken anti-myostatin therapies
  • Must weigh at least 15kg
  • Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable)
  • History of Spinal Fusion within 6 months of Screening. MAGEC rod nonsurgical adjustments are allowed during the study
  • Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05337553


Contacts
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Contact: Chief Medical Officer 203-404-0410 clinicaltrials@biohavenpharma.com

Locations
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Sponsors and Collaborators
Biohaven Pharmaceuticals, Inc.
Investigators
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Study Director: Lindsey Lair, MD Biohaven Pharmaceuticals, Inc.
More Information
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Responsible Party: Biohaven Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT05337553    
Other Study ID Numbers: BHV2000-301
First Posted: April 20, 2022    Key Record Dates
Last Update Posted: September 21, 2023
Last Verified: September 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Neuromuscular Diseases
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
 Neurologic Manifestations
Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases