COAV101A12306 Paediatric patients with Spinal Muscular Atropy

• Research type

  Research Study

• Full title

  A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA)

• IRAS ID

  289134

• Contact name

  Volker Straub

• Contact email

  volker.straub@newcastle.ac.uk

• Sponsor organisation

  Novartis Pharma AG

• Eudract number

  2020-005995-37

• Clinicaltrials.gov Identifier

  NCT04851873

• Duration of Study in the UK

  1 years, 9 months, 7 days

• Research summary

  Spinal muscular atrophy (SMA) is a rare, genetic, early childhood disease caused by a lack of functional Survival Motor Neuron 1 (SMN1) gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. SMA is the leading genetic cause of infant death.

  There are four types of SMA (type 0 to 4) and the severity of SMA corresponds to the copy number of the Survival Motor Neuron 2 (SMN2) gene, the “back-up” gene that produces a small fraction of functional SMN protein compared to SMN1.

  Zolgensma® also known as OAV101 has been approved for use in countries including the US and regions in Europe for the treatment of children with SMA.
  OAV101 consists of a modified virus called adeno-associated virus (AAV), which transports a healthy copy of the SMN1 gene to motor neurons in the spinal cord to replace SMN protein. Doctors call the gene and the modified virus together a “vector.”

  This study is to find out if OAV101 is safe and beneficial to use in children with SMA who weigh between 8.5 kg and 21 kg inclusive. This study will also build on the established safety profile and how well OAV101 works, and aims to gather additional data in a group of heavier children not previously studied in previous OAV101 studies.

  The study will last approximately 13 months (45 day screening period, 1 day of treatment and 12 month follow-up) and approximately 3 participants in the UK, across 2 sites, will be included in the study. On Day 1 eligible participants will receive a single administration of OAV101 at 1.1e14 vg/kg.

• REC name

  North East - York Research Ethics Committee

• REC reference

  21/NE/0128

• Date of REC Opinion

  25 Aug 2021

• REC opinion

  Further Information Favourable Opinion