RESILIENT/Taldefgrobep Alfa in Spinal Muscular Atrophy

• Research type

  Research Study

• Full title

  A Randomized, Double-Blind, PlaceboControlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)

• IRAS ID

  1006287

• Contact name

  Jackie Marin

• Contact email

  jackie.marin@biohavenpharma.com

• Sponsor organisation

  Biohaven Pharmaceuticals

• Eudract number

  2022-000193-25

• Clinicaltrials.gov Identifier

  NCT05337553

• Research summary

  Spinal Muscular Atrophy (SMA) is an autosomal recessive neurodegenerative neuromuscular disease that affects about 1 in 10,000 births and with an estimated prevalence of 1-3 per 100,0001,2 (up to ~25,000 individuals in the United States, European Union and UK combined). The clinical manifestation is progressive skeletal muscle weakness and atrophy ranging from mild proximal weakness to extreme weakness, paraplegia (paralysis of all or part of trunk, legs, and pelvic organs) of infancy, respiratory failure, and death.
  Taldefgrobep alfa is injected subcutaneously (under the skin) once a week. The main purpose of this study is to learn whether the study drug, taldefgrobep alfa, is well tolerated, safe and effective in the treatment of SMA in children and young adults between the ages of 4 and 21 years old. Some study participants (33%) will receive placebo instead of the study drug taldefgrobep alfa.
  Participants will be on the study for 2 years. There is initial placebo controlled part and then open label part of the study where participants will receive the study drug only (without placebo).
  As part of the study participants will have regular visits to clinic at day 1 (when they start treatment), 7, 42, 85, 169, 253, and 337. Participants who continue in the open label part of the study will have visits at day 85, day 169, day 253 and day 337.
  During the study participants will have physical examination and measurements, vital signs, ECG, lab tests, DEXA scan, questionnaires and PK samples (to study the plasma concentration of the study drug).
  Participants will be on standard of care treatment with risdiplam, nusinersen, and/or onasemnogene, abeparvovec-xioi during the duration of the trial treatment.

• REC name

  East of Scotland Research Ethics Service REC 2

• REC reference

  22/ES/0037

• Date of REC Opinion

  7 Feb 2023

• REC opinion

  Further Information Favourable Opinion