In the next few weeks we are expecting the AveXis AVXS-101 trial to start enrolling.
There will be two site centres here in the UK:
Great Ormond Street Hospital for Children
Contact : hinal.patel@ucl.ac.uk
The John Walton Muscular Dystrophy Research Centre MRC Centre for Neuromuscular Diseases.
Contact: Anna.peel1@newcastle.ac.uk
There will be strict inclusion and exclusion criteria which is included below.
Criteria
Inclusion Criteria
- Children with SMA Type 1 as determined by diagnosis of SMA based on gene mutation analysis with biallelic SMN1 mutations (deletion or point mutations) and one or two copies of SMN2 [inclusive of the known SMN2 gene modifier mutation (c.859G>C)]
- Children must be < 6 months (< 180 days) of age at the time of AVXS-101 infusion
- Children must have a swallowing evaluation test performed prior to administration of gene replacement therapy
Exclusion Criteria
- Previous, planned or expected scoliosis repair surgery/procedure prior to 18 months of age
- Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry < 95% saturation at screening
- Use or requirement of non-invasive ventilatory support for 12 or more hours daily in the two weeks prior to dosing
- Children with signs of aspiration based on a swallowing test or whose weight-for-age falls below the 3rd percentile based on World Health Organisation (WHO) Child Growth Standards [27] and unwilling to use an alternative method to oral feeding
- Participation in recent SMA treatment clinical trial (with the exception of observational cohort studies or non-interventional studies) or receipt of an investigational or commercial compound, product or therapy administered with the intent to treat SMA (eg, nusinersen, valproic acid,) at any time prior to screening for this trial.
Please contact Kelly at kelly.jones@treatsma.uk
