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

Phase 1

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-BE

Lead Sponsor: ovartis Pharma AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 260

Inclusion Criteria

Participants eligible for inclusion in this study must meet all of the
following criteria:
1. Participated in an OAV101 clinical trial.
2. Written informed consent must be obtained before any assessment is
performed.
3. Patient/Parent/legal guardian willing and able to 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

There are no specific exclusion criteria for this study.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of this study is to assess long-term safety in terms of treatment-emergent serious adverse events (SAEs) and treatment-emergent adverse events of special interest (AESIs)<br>;Secondary Objective: The secondary objectives of this study are; <br>(1) assess long-term efficacy of OAV101 treatment.<br>(2) assess long-term safety for measures other than adverse events . ;Primary end point(s): ? Number and proportion of patients reporting treatment-emergent serious adverse events (SAEs) by Medical Dictionary for Regulatory Activities (MedDRA) System Organ Class (SOC) and Preferred Term (PT) for the entire duration of the study (i.e., up to 15 years)<br>? Number and proportion of patients reporting treatment-emergent adverse events of special interest (AESIs) by AESI category and MedDRA SOC and PT within each AESI category for the entire duration of the study (i.e., up to 15 years) <br>;Timepoint(s) of evaluation of this end point: up to 15 years

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): ? The number and proportion of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist <br>? The number and proportion of participants demonstrating maintenance of each developmental milestone<br>? Change from Baseline in the Hammersmith Functional Motor Scale – Expanded (HFMSE) total score <br>? Change from Baseline in the Revised Upper Limb Module (RULM) total score<br>? Number and proportion of patients with potentially clinically significant vital sign findings, summarized individually for each vital sign parameter <br>? Number and proportion of patients with potentially clinically significant laboratory values, summarized individually for each laboratory parameter<br>;Timepoint(s) of evaluation of this end point: 5 years