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ong-term follow-up of patients with spinal muscular atrophy Treated with OAV101 in Clinical Trials

Phase 1
Active, not recruiting
Conditions
Spinal Muscular Atrophy
MedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Registration Number
EUCTR2021-006781-21-FR
Lead Sponsor
ovartis Pharma AG
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Not Recruiting
Sex
All
Target Recruitment
260
Inclusion Criteria

Participants eligible for inclusion in this study must meet all of the following criteria:
1. Received OAV101 IT or OAV101 IV in a Phase I to IIIb clinical trial sponsored by AveXis, Novartis or Novartis Gene Therapies.
2. Patient/Parent/legal guardian willing and able to complete the informed consent process and comply with study procedures.

Are the trial subjects under 18? yes
Number of subjects for this age range: 260
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Score yes” on item 4 or item 5 of the suicidal ideation section of the C-SSRS, if this ideation occurred in the past 6 months, or yes” on any item of the suicidal behavior section, except for the Non-Suicidal Self-Injurious Behavior” (item also included in the suicidal behavior section) if this behavior occurred in the past 2 years.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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