| CTRI Number |
CTRI/2025/02/080626 [Registered on: 14/02/2025] Trial Registered Prospectively |
| Last Modified On: |
24/04/2026 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Other |
|
Public Title of Study
|
To learn if the drug OAV101/ Zolgensma is safe and continues to work in children and young adults with Spinal Muscular Atrophy (SMA) who were previously treated with this drug in past trials |
|
Scientific Title of Study
|
Long-term Follow-up of Patients with Spinal Muscular Atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials |
| Trial Acronym |
NIL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| 2021-006781-21 |
EudraCT |
| COAV101A12308, Version 03, date:04-May-2023 |
Protocol Number |
| NCT05335876 |
ClinicalTrials.gov |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Murugananthan K |
| Designation |
SSO Country Monitoring Head |
| Affiliation |
Novartis Healthcare Private |
| Address |
Novartis Healthcare Private Limited 6 and 7 floor Inspire BKC G
Block BKC Main Road Bandra Kurla Complex Bandra (East) Mumbai
Mumbai
MAHARASHTRA
400051
India |
| Phone |
912250243544 |
| Fax |
|
| Email |
murugananthan.k@novartis.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Murugananthan K |
| Designation |
SSO Country Monitoring Head |
| Affiliation |
Novartis Healthcare Private |
| Address |
Novartis Healthcare Private Limited 6 and 7 floor Inspire BKC G
Block BKC Main Road Bandra Kurla Complex Bandra (East) Mumbai
MAHARASHTRA
400051
India |
| Phone |
912250243544 |
| Fax |
|
| Email |
murugananthan.k@novartis.com |
|
Details of Contact Person
Public Query
|
| Name |
Murugananthan K |
| Designation |
SSO Country Monitoring Head |
| Affiliation |
Novartis Healthcare Private |
| Address |
Novartis Healthcare Private Limited 6 and 7 floor Inspire BKC G
Block BKC Main Road Bandra Kurla Complex Bandra (East) Mumbai
MAHARASHTRA
400051
India |
| Phone |
912250243544 |
| Fax |
|
| Email |
murugananthan.k@novartis.com |
|
|
Source of Monetary or Material Support
|
| Novartis Pharma AG, Novartis Campus 4056 – Basel, Switzerland |
|
|
Primary Sponsor
|
| Name |
Novartis Healthcare Pvt Ltd |
| Address |
7 floor, Inspire BKC, G Block, BKC Main Road, Bandra Kurla
Complex, Bandra (East), Mumbai – 400051,India |
| Type of Sponsor |
Pharmaceutical industry-Global |
|
|
Details of Secondary Sponsor
|
|
|
Countries of Recruitment
|
Australia
Belgium
Brazil
Canada
China
Denmark
France
India
Italy
Japan
Malaysia
Mexico
Netherlands
Saudi Arabia
Singapore
South Africa
Spain
Taiwan
Thailand
United Kingdom
United States of America
Viet Nam |
Sites of Study
Modification(s)
|
| No of Sites = 4 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Sheffali Gulati |
All India Institute of Medical Sciences |
Child Neurology Division, Department of Pediatrics, A wing, Mother and Child Block, AIIMS, New Delhi, Deihi,110029
South
DELHI |
9810386847
Sheffaligulati@gmail.com |
| Dr Neelu Desai |
P D Hinduja Hospital & Medical Research Centre |
Department of Pediatrics, P D Hinduja Hospital & Medical Research Centre, Veer Savarkar Marg, Mahim, Mumbai,400016
Mumbai (Suburban)
MAHARASHTRA |
02224447193
neelushahdesai@gmail.com |
| Dr Sanjukta Dey |
Peerless Hospital |
Department of Pediatrics, Peerless Hospital, 360 Panchasayar, Kolkata, Westbengal, 700094
Kolkata
WEST BENGAL |
9836414848
dey.sanjukta@gmail.com |
| Dr Ratna Dua Puri |
Sir Ganga Ram Hospital |
Institute of Medical Genetics & Genomics, Sir Ganga Ram Hospital Marg, Rajinder Nagar, New Delhi, Deihi,110060
North
DELHI |
01142251412
ratnadpuri@yahoo.com |
|
Details of Ethics Committee
Modification(s)
|
| No of Ethics Committees= 4 |
| Name of Committee |
Approval Status |
| Clinical Research Ethics Committee Peerless Hospitex Hospital & Research Center Ltd. |
Approved |
| Institution Ethics Committee, P D Hinduja Hospital & Medical Research Centre |
Approved |
| Institutional Ethics Committee, AIIMS |
Approved |
| Sir Ganga Ram Hospital Ethics Committee |
Submittted/Under Review |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: G129||Spinal muscular atrophy, unspecified, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Comparator Agent |
NA |
NA |
| Intervention |
NA |
No study drug will be administered in this study as this is a long-term follow-up study |
|
|
Inclusion Criteria
|
| Age From |
2.00 Year(s) |
| Age To |
18.00 Year(s) |
| Gender |
Both |
| Details |
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. |
|
| ExclusionCriteria |
| Details |
There are no specific exclusion criteria for this study. |
|
|
Method of Generating Random Sequence
|
Not Applicable |
|
Method of Concealment
|
Not Applicable |
|
Blinding/Masking
|
Not Applicable |
|
Primary Outcome
|
| Outcome |
TimePoints |
| What is the long-term effect of OAV101/ Zolgensma on safety in patients with SMA who were treated with this drug in past trials? |
Every 6 months for 2 years and then every year till the 5th Year |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| To explore other potential effects of OAV101 on exploratory efficacy & safety biomarkers |
Every 6 months for 2 years & then every year till 5th Year |
| To describe the time-course of the elevation of anti-AAV9 antibodies after administration of OAV101 |
Every 6 months for 2 years & then every year till 5th Year |
| To assess long-term caregiver experience |
Every 6 months for 2 years & then every year till 5th Year |
|
|
Target Sample Size
|
Total Sample Size="175"
Sample Size from India="30"
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" |
|
Phase of Trial
|
Phase 3/ Phase 4 |
Date of First Enrollment (India)
Modification(s)
|
17/04/2025 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
19/12/2022 |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
Estimated Duration of Trial
Modification(s)
|
Years="5"
Months="0"
Days="0" |
|
Recruitment Status of Trial (Global)
|
Open to Recruitment |
| Recruitment Status of Trial (India) |
Open to Recruitment |
|
Publication Details
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
Brief Summary
Modification(s)
|
This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment. For Follow-up Periods 1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at the Investigational site. For the first 2 years (Follow-up Period 1), visits will occur every 6 months. For Years 3 to 5 (Follow-up Period 2) follow-up visits will be conducted annually. All patients will enter the study at the baseline visit and continue for 5 years. |