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NCT05337553 Clinical Trial

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

Neuromuscular Diseases Clinical Trial

— RESILIENT  

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT05337553
Other study ID # BHV2000-301
Secondary ID
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date July 6, 2022
Est. completion date January 2025

Study information
Verified date November 2023
Source Biohaven Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility
Status Active, not recruiting
Enrollment 269
Est. completion date January 2025
Est. primary completion date January 2025
Accepts healthy volunteers No
Gender All
Age group 4 Years to 21 Years
Eligibility 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

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• taldefgrobep alfa
DB Phase: 35 mg/50 mg weekly subcutaneous injection
• Placebo
DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection
• taldefgrobep alfa
Extension Phase: 35 mg/50 mg weekly subcutaneous injection

Locations
Country Name City State
Belgium University Hospital Antwerp Edegem
Belgium University Hospital Ghent Ghent
Belgium University Hospital Leuven Leuven
Czechia University Hospital Brno - Dept. of Pediatric Neurology Brno
Czechia Motol University Hospital Prague
Germany University Hospital Essen (Public-Law Institution) - Dept. of Pediatrics I Essen
Germany University Hospital Freiburg, Center For Children and Adolescent Medicine, Dept. of Neuropediatrics and Muscle Disorders Freiburg
Germany Dr. Von Haunersches Children'S Hospital - Lmu Munich Munich
Italy Irccs Institute of Neurological Sciences of Bologna - Bellaria Hospital Bologna
Italy Nemo-Brescia Clinical Center For Neuromuscular Diseases Gussago
Italy IRCCS NEUROLOGICAL INSTITUTE C. MONDINO CHILD and NEUROPSYCHIATRIC UNIT Pavia
Italy Bambino Gesù Children'S Research Hospital Irccs - San Paolo Office Dept. of Neuroscience Roma
Netherlands University Medical Center Utrecht Utrecht
Poland University Clinical Centre in Gdansk - Dept. of Developmental Neurology Gdansk
Poland Heliodor Swiecicki Clinical Hospital At Medical University - Child and Adolescents Neurology Clinic Poznan
Poland The Children'S Memorial Health Institute - Dept. of Neurology and Epileptology Warsaw
Poland T. Marciniak Lower Silesian Specialist Hospital, Pediatric Neurology Dept. Wroclaw
Spain Donostia University Hospital Donostia
Spain Hospital Sant Joan de Déu Esplugues De Llobregat
Spain Maternal-Child'S Hospital of Málaga, Regional University Hospital - Pediatric Neurology Unit Málaga
Spain La Fe University and Polytechnic Hospital Valencia
United Kingdom Royal Hospital For Children Glasgow Scotland
United Kingdom John Radcliffe Hospital Oxford
United States Rare Disease Research Atlanta Georgia
United States Children's Hospital Colorado Aurora Colorado
United States UT Pediatric Neurosciences/Dell Children's Medical Center Austin Texas
United States Boston Children's Hospital - Harvard Boston Massachusetts
United States University of Virginia Children's Hospital Charlottesville Virginia
United States Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
United States Cincinnati Children's Hospital Medical Center Cincinnati Ohio
United States Nationwide Children's Hospital Columbus Ohio
United States Neurology Rare Disease Center Denton Texas
United States Duke University Medicine Durham North Carolina
United States University of Kansas Medical Center Fairway Kansas
United States Cook Children's Hospital Fort Worth Texas
United States UF Health, Shands Hospital Gainesville Florida
United States BSHS Office of Research Grand Rapids Michigan
United States Penn State College of Medicine Hershey Pennsylvania
United States Indiana University -Riley Research Indianapolis Indiana
United States University of Iowa Iowa City Iowa
United States UCSD & Rady Children's La Jolla California
United States Children's Hospital of Los Angeles Los Angeles California
United States Medical College of Wisconsin Milwaukee Wisconsin
United States Vanderbilt University Medical Center Nashville Tennessee
United States Columbia University Medical Center New York New York
United States Children's Hospital of The King's Daughters Norfolk Virginia
United States CHOP Children's Hospital of Philadelphia Philadelphia Pennsylvania
United States Phoenix Children's Phoenix Arizona
United States UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania
United States Washington University in St. Louis Saint Louis Missouri
United States UCSF Benioff Children's Hospital, Medical Center San Francisco California
United States Stony Brook University Hospital Stony Brook New York
United States MultiCare Institute of Research and Innovation Tacoma Washington

Sponsors (1)
Lead Sponsor Collaborator
Biohaven Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Belgium,  Czechia,  Germany,  Italy,  Netherlands,  Poland,  Spain,  United Kingdom, 

Outcome
Type Measure Description Time frame Safety issue
Primary Efficacy of taldefgrobep alfa compared to placebo in change in the 32 item Motor Function Measure (MFM-32) total score 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. Baseline to Week 48
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