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NCT Number: NCT05337553

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases SMA Spinal Cord Diseases

Age range

4 year–21 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

University Hospital Antwerp, Edegem, Belgium

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Taldefgrobep Alfa

Drug

DB Phase: 35 mg/50 mg weekly subcutaneous injection

Other names: BHV-2000, BMS-986089

Placebo

Drug

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

Primary outcomes

  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 scores from the 32 items are summed and transformed to a 0-100 scale, with higher scores reflecting higher levels of functional abilities.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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)

  4. 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)

  5. Change from baseline in Tanner staging

    Time frame: Baseline, Week 48

  6. 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

  7. 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

  8. Trough plasma concentration

    Time frame: Baseline, Week 12, Week 24, Week 36, Week 48

Sponsors and collaborators

Lead sponsor

Biohaven Pharmaceuticals, Inc.

Industry

Registry information

Official study 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

Acronym: RESILIENT

Important dates

Study start
2022
Primary completion
2024
Study completion
2027
First posted
Apr 20, 2022
Registry last updated
Jun 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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