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

• Register: EUCTR
• Last refreshed on: 19 August 2024
• Main ID: EUCTR2021-006781-21-DK
• Date of registration: 20/12/2022
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma AG
• Public title: Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 in Clinical Trials
• Scientific title: Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials
• Date of first enrolment: 15/02/2023
• Target sample size: 260
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2021-006781-21
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: Long-term follow-up of patients with SMA Treated with OAV101 IT or IV administration. No study drug. If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Belgium, Brazil, Canada, China, Colombia, Denmark, Egypt, France, Germany, Greece, India, Italy, Japan, Malaysia, Mexico, Netherlands, Saudi Arabia, Singapore, South Africa, Spain, Taiwan, Thailand, United Kingdom, United States, Viet Nam

Contacts

• Name: Medical Information
• Affiliation: Novartis Healthcare A/S

• Name: Medical Information
• Affiliation: Novartis Healthcare A/S

Key inclusion & exclusion criteria

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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Spinal Muscular Atrophy
MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Trade Name: Zolgensma 2 x 1013 vg (vector genomes)/mL solution for infusion
Product Name: OAV101
Product Code: OAV101
Pharmaceutical Form: Solution for infusion

Trade Name: Zolgensma 2 x 1013 vg (vector genomes)/mL solution for infusion
Product Name: OAV101
Product Code: OAV101
Pharmaceutical Form: Solution for injection

Primary Outcome(s)

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).
? 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).

Secondary Objective: The secondary objectives of this study are;
(1) assess long-term efficacy of OAV101 treatment.
(2) assess long-term safety for measures other than adverse events .

Timepoint(s) of evaluation of this end point: up to 15 years

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)

Secondary Outcome(s)

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

Timepoint(s) of evaluation of this end point: 5 years

Secondary ID(s)

2021-006781-21-BE

COAV101A12308

NCT05335876

Source(s) of Monetary Support

Novartis Pharma AG

Ethics review

Status: Approved
Approval date: 15/02/2023
Contact: