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

May 29, 2026 updated by: Biohaven Pharmaceuticals, Inc.

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

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 and/or risdiplam and/or have a history of onasemnogene abeparvovec, compared to placebo.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
269

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Belgium
      • Edegem, Belgium, 02650
        • University Hospital Antwerp
      • Ghent, Belgium, 09000
        • University Hospital Ghent
      • Leuven, Belgium, 03000
        • University Hospital Leuven
  • Czechia
      • Brno, Czechia, 625 00
        • University Hospital Brno - Dept. of Pediatric Neurology
      • Prague, Czechia, 150 06
        • Motol University Hospital
  • Germany
      • Essen, Germany, 45147
        • University Hospital Essen (Public-Law Institution) - Dept. of Pediatrics I
      • Freiburg im Breisgau, Germany, 79106
        • University Hospital Freiburg, Center For Children and Adolescent Medicine, Dept. of Neuropediatrics and Muscle Disorders
      • Munich, Germany, 80337
        • Dr. Von Haunersches Children'S Hospital - Lmu Munich
  • Italy
      • Bologna, Italy, 40139
        • Irccs Institute of Neurological Sciences of Bologna - Bellaria Hospital
      • Gussago, Italy, 25064
        • Nemo-Brescia Clinical Center For Neuromuscular Diseases
      • Pavia, Italy, 27100
        • IRCCS NEUROLOGICAL INSTITUTE C. MONDINO CHILD and NEUROPSYCHIATRIC UNIT
      • Roma, Italy, 00165
        • Bambino Gesù Children'S Research Hospital Irccs - San Paolo Office Dept. of Neuroscience
  • Netherlands
      • Utrecht, Netherlands, 3584 CX
        • University Medical Center Utrecht
  • Poland
      • Gdansk, Poland, 80-925
        • University Clinical Centre in Gdansk - Dept. of Developmental Neurology
      • Poznan, Poland, 60-355
        • Heliodor Swiecicki Clinical Hospital At Medical University - Child and Adolescents Neurology Clinic
      • Warsaw, Poland, 04-730
        • The Children'S Memorial Health Institute - Dept. of Neurology and Epileptology
      • Wroclaw, Poland, 54-049
        • T. Marciniak Lower Silesian Specialist Hospital, Pediatric Neurology Dept.
  • Spain
      • Donostia / San Sebastian, Spain, 20014
        • Donostia University Hospital
      • Esplugues de Llobregat, Spain, 08950
        • Hospital Sant Joan de Déu
      • Málaga, Spain, 29011
        • Maternal-Child'S Hospital of Málaga, Regional University Hospital - Pediatric Neurology Unit
      • Valencia, Spain, 46026
        • La Fe University and Polytechnic Hospital
  • United Kingdom
      • Oxford, United Kingdom, OX3 9DU
        • John Radcliffe Hospital
    • Scotland
      • Glasgow, Scotland, United Kingdom, G51 4TF
        • Royal Hospital For Children
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Phoenix Children's
    • California
      • La Jolla, California, United States, 92037
        • UCSD & Rady Children's
      • Los Angeles, California, United States, 90027
        • Children's Hospital of Los Angeles
      • San Francisco, California, United States, 94158
        • UCSF Benioff Children's Hospital, Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Florida
      • Gainesville, Florida, United States, 32610
        • UF Health, Shands Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Rare Disease Research
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University -Riley Research
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Kansas
      • Fairway, Kansas, United States, 66205
        • University of Kansas Medical Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital - Harvard
    • Michigan
      • Grand Rapids, Michigan, United States, 49503
        • BSHS Office of Research
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University in St. Louis
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical Center
      • Stony Brook, New York, United States, 11794
        • Stony Brook University Hospital
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University Medicine
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Penn State College of Medicine
      • Philadelphia, Pennsylvania, United States, 19104
        • CHOP Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, United States, 15224
        • UPMC Children's Hospital of Pittsburgh
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center
    • Texas
      • Austin, Texas, United States, 78723
        • UT Pediatric Neurosciences/Dell Children's Medical Center
      • Denton, Texas, United States, 75208
        • Neurology Rare Disease Center
      • Fort Worth, Texas, United States, 76104
        • Cook Children's Hospital
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • University of Virginia Children's Hospital
      • Norfolk, Virginia, United States, 23507
        • Children's Hospital of The King's Daughters
    • Washington
      • Tacoma, Washington, United States, 98405
        • MultiCare Institute of Research and Innovation
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

4 years to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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 and/or 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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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 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 OLE phase.
Drug: Placebo
DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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 scores from the 32 items are summed and transformed to a 0-100 scale, with higher scores reflecting higher levels of functional abilities.
Baseline to Week 48

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Efficacy of taldefgrobep alfa compared to placebo in change in the Revised Upper Limb Module (RULM) score
Time Frame: Baseline to Week 48
The RULM includes 20 items, graded from 0 to 2, with a maximum score of 37. Higher scores indicate better function.
Baseline to Week 48
Efficacy of taldefgrobep alfa compared to placebo in change in the Revised Hammersmith Scale (RHS)
Time Frame: Baseline to Week 48
The RHS has a maximum score of 69 points (33 items are scored 0-2, and 3 items scored 0-1). Higher scores indicate better function.
Baseline to Week 48
Change from Baseline in lean body mass
Time Frame: Baseline, Week 48
Body mass will be measured as change in kg (greater change meaning more improvement)
Baseline, Week 48
Change from Baseline in bone mineral density
Time Frame: Baseline, Week 48
Bone mineral density will be measured by Z-score change (higher score indicates improvement)
Baseline, Week 48
Change from baseline in Tanner staging
Time Frame: Baseline, Week 48
Baseline, Week 48
Injection acceptability assessments
Time Frame: Week 48
If local injection site reactions (such as redness, itching, swelling, hardening or bruising) are experienced, the acceptability is scored on a scale of 1-5 with 1 being totally acceptable and 5 being not at all acceptable If pain is experienced at the injection site, pain will be scored on a scale of 1-5 with 1 being totally acceptable and 5 being not at all acceptable
Week 48
Number of Participants with new or worsening lab abnormalities, Adverse Events (AEs), Serious AEs (SAEs) and AEs Leading to Study Drug Discontinuation in the Double-blind Phase
Time Frame: Up to 48 Weeks
Up to 48 Weeks
Trough plasma concentration
Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48
Baseline, Week 12, Week 24, Week 36, Week 48

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Biohaven Pharmaceuticals, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Lindsey Lair, MD, Biohaven Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 6, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 25, 2024

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 14, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 14, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 20, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 1, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 29, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • BHV2000-301

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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