Status:
COMPLETED
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Lead Sponsor:
Novartis Gene Therapies
Conditions:
SMA
Eligibility:
All Genders
Up to 6 years
Phase:
PHASE3
Brief Summary
Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and a...
Detailed Description
Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and a...
Eligibility Criteria
Inclusion
- Patients 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 \[inclusive of the known SMN2 gene modifier mutation (c.859G\>C)\]
- Patients must be \< 6 months (\< 180 days) of age at the time of onasemnogene abeparvovec-xioi infusion
- Patients must have a swallowing evaluation test performed prior to administration of gene replacement therapy
Exclusion
- 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
- Patient with signs of aspiration based on a swallowing test or whose weight-for-age falls below the 3rd percentile based on World Health Organization (WHO) Child Growth Standards 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.
Key Trial Info
Start Date :
August 16 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 11 2020
Estimated Enrollment :
33 Patients enrolled
Trial Details
Trial ID
NCT03461289
Start Date
August 16 2018
End Date
September 11 2020
Last Update
August 16 2022
Active Locations (10)
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1
University Hospital Ghent Neuromuscular reference center
Ghent, Belgium
2
Neuropédiatrie - Centre de Référence des Maladies Neuromusculaires
Liège, Belgium
3
Hôpital Armand Trousseau
Paris, France
4
Istituto Gianninia Gaslini
Genova, Italy