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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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Biohaven

Status and phase

Active, not recruiting
Phase 3

Conditions

Neuromuscular Diseases
SMA
Spinal Muscular Atrophy

Treatments

Drug: Placebo
Drug: taldefgrobep alfa

Study type

Interventional

Funder types

Industry

Identifiers

NCT05337553
BHV2000-301

Details and patient eligibility

About

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.

Full 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.

Enrollment

269 patients

Sex

All

Ages

4 to 21 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Quadruple Blind

269 participants in 2 patient groups, including a placebo group

taldefgrobep alfa
Experimental group
Description:
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.
Treatment:
Drug: taldefgrobep alfa
Drug: taldefgrobep alfa
Placebo
Placebo Comparator group
Description:
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.
Treatment:
Drug: Placebo
Drug: taldefgrobep alfa
Drug: taldefgrobep alfa

Trial contacts and locations

53

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Central trial contact

Chief Medical Officer

Data sourced from clinicaltrials.gov

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