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CTRI Number  CTRI/2021/10/037251 [Registered on: 11/10/2021] Trial Registered Prospectively
Last Modified On: 31/07/2024
Post Graduate Thesis  No 
Type of Trial  PMS 
Type of Study   Drug 
Study Design  Single Arm Study 
Public Title of Study   A study to observe if Risdiplam treatment is safe and effective in Spinal Muscular Atrophy Patients in India.  
Scientific Title of Study   Observational, Non-Interventional, Post-marketing surveillance study of Risdiplam in Spinal Muscular Atrophy patients in India. 
Trial Acronym   
Secondary IDs if Any  
Secondary ID  Identifier 
ML43557_Version 1.0 Dated 04 Aug 2021  Protocol Number 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)  
Name   
Designation   
Affiliation   
Address 




 
Phone    
Fax    
Email    
 
Details of Contact Person
Scientific Query  
Name  Milan Choksey 
Designation  Scientific Responsible  
Affiliation  Roche Products India Private Limited  
Address  146B, 166 A, Unit No. 7, 8, 9 8th Floor, R City Office, R City Mall Lal Bahadur Shastri Marg Ghatkopar, Mumbai

Mumbai
MAHARASHTRA
400086
India 
Phone    
Fax    
Email  milan.choksey@roche.com  
 
Details of Contact Person
Public Query
Modification(s)  
Name  Sharad Junnare 
Designation  Manager -Clinical Operations  
Affiliation  Roche Products India Private Limited 
Address  146B, 166 A, Unit No. 7, 8, 9 8th Floor, R City Office, R City Mall Lal Bahadur Shastri Marg Ghatkopar, Mumbai

Mumbai
MAHARASHTRA
400086
India 
Phone  9920171414  
Fax    
Email  sharad.junnare@roche.com  
 
Source of Monetary or Material Support  
Roche Products India Pvt Ltd 146-B, 166 A, Unit No. 7, 8, 98th Floor, R City Office, R City Mall, Lal Bahadur Shastri Marg, Ghatkopar, Mumbai - 400 086, Maharashtra  
 
Primary Sponsor  
Name  Roche Products India Pvt Ltd  
Address  146-B, 166 A, Unit No. 7, 8, 9 8th Floor, R City Office, R City Mall Lal Bahadur Shastri Marg Ghatkopar, Mumbai  
Type of Sponsor  Pharmaceutical industry-Global 
 
Details of Secondary Sponsor  
Name  Address 
NIL  NIL 
 
Countries of Recruitment     India  
Sites of Study
Modification(s)  
No of Sites = 7  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Siddharth Shah  Royal Institute of Child Neuro Sciences  Opposite to Drive In Cinema
Ahmadabad
GUJARAT 		 9909960555

sidh909@hotmail.com 
Dr Smilu Mohanlal  Aster MIMS Hospital  Aster Malaber Institute of Medical Sciences , Mini Bypass road, Govindapuram, Kozhikode 673016
Kozhikode
KERALA 		 9892071925

smilu.mohanlal@asterhospital.com 
Dr Anaita Udwadia Hegde  Jaslok Hospital and Research Centre  15 Dr Deshmukh Marg Pedder Road
Mumbai (Suburban)
MAHARASHTRA 		 9820186155

anaitaheqde@gmail.com 
Dr Alpana Kondekar  Nair  Hospital  Department of Pediartrics, College Bulding, First Floor, Topiwala National Medical College, A L Nair Road, Mumbai Central Mumbai 400008
Mumbai
MAHARASHTRA 		 8097952226

dralpanakondekar@gmail.com 
Dr Neelu Krishnadas Shah  P D Hinduja Hospital and Medical Research Centre  Veer Savarkar Marg Mahim Mumbai-400016
Mumbai (Suburban)
MAHARASHTRA 		 9920614333

neelushahdesai@gmail.com 
Dr Sanjukta Dey  Peerless Hospital  360, Pancha Sayar Rd, Sahid Smirity Colony, Pancha Sayar, Kolkata, West Bengal 700094
Kolkata
WEST BENGAL 		 9836363642

dey.sanjukta@gmail.com 
Dr Ratna Puri  Sir Gangaram Hospital  Sir Ganga Ram Hospital Marg, Rajinder Nagar, New Delhi, Delhi 110060
New Delhi
DELHI 		 9811869192

ratnadpuri@yahoo.com 
 
Details of Ethics Committee
Modification(s)  
No of Ethics Committees= 7  
Name of Committee  Approval Status 
Clinical Research Ethics Committee Peerless Hospital and Research Centre Limited  Approved 
Institutional Ethics Committee Jaslok Hospital and Research Centre  Approved 
Institutional Ethics Committee Nair Hospital  Approved 
Institutional Ethics Committee P. D Hinduja Hospital and Medical Research Centre  Submittted/Under Review 
Malabar Institute of Medical Sciences(MIMS IEC)  Approved 
Sangini Hospital Ethics Committee  Submittted/Under Review 
Sir Ganga Ram Hospital  Approved 
 
Regulatory Clearance Status from DCGI  
Status 
Notified 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  (1) ICD-10 Condition: G129||Spinal muscular atrophy, unspecified,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Intervention  Evrysdi®Risdiplam  Evrysdi® risdiplam powder for oral solution 0.75 mg/mL. It is prescribed orally once daily after a meal or breastfeeding as per the routine clinical practice and Prescribing Information. The recommended dosage is determined by age and body weight as per Prescribing Information. For the patients of age ≥2 years weighing ≥20 kg, the recommended daily dose is 5 mg.  
Comparator Agent  Not applicable  It is a non interventional observational study and there is no comparator arm for the study 
 
Inclusion Criteria  
Age From  2.00 Month(s)
Age To  60.00 Year(s)
Gender  Both 
Details  1. Have signed the informed consent/assent (as applicable). In the case of minors, a parent or authorized adult will be required to sign the Informed Consent Form on behalf of the patient. The informed consent/assent will be obtained in accordance with local requirements
2. Patients aged ≥2 months at enrollment (for preterm infants this is the corrected age)
3. Confirmed diagnosis of 5q autosomal recessive SMA including genetic confirmation of homozygous deletion or compound heterozygosity that is predictive of loss of function of the survival motor neuron 1 (SMN1) gene and/or SMN2 gene
4. Clinical history, signs or symptoms attributable to SMA in patients of age 2 months and older
5. Patients who have been prescribed and received at least one dose of risdiplam for SMA.
 
 
ExclusionCriteria 
Details   
 
Method of Generating Random Sequence   Not Applicable 
Method of Concealment   Not Applicable 
Blinding/Masking   Not Applicable 
Primary Outcome  
Outcome  TimePoints 
The primary objective of the study is to assess the safety and effectiveness of risdiplam for the treatment of SMA in patients of age 2 months and older in India.  Three years 
 
Secondary Outcome  
Outcome  TimePoints 
To describe the profile of SMA patients (2 months of age and older) in India based on demographics and familial, clinical, and genetic characteristics.   Three years 
 
Target Sample Size   Total Sample Size="15"
Sample Size from India="15" 
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   Post Marketing Surveillance 
Date of First Enrollment (India)   15/10/2021 
Date of Study Completion (India) Applicable only for Completed/Terminated trials 
Date of First Enrollment (Global)  Date Missing 
Date of Study Completion (Global) Applicable only for Completed/Terminated trials 
Estimated Duration of Trial   Years="3"
Months="6"
Days="0" 
Recruitment Status of Trial (Global)
Modification(s)  		 Not Applicable 
Recruitment Status of Trial (India)  Open to Recruitment 
Publication Details   NIL 
Individual Participant Data (IPD) Sharing Statement

Will individual participant data (IPD) be shared publicly (including data dictionaries)?  

Response - NO
Brief Summary  

This is a non-interventional, single-arm, observational study with primary prospective data collection (NIS PDC) and retrospective collection of prior medical/treatment history data from medical records. Study is designed to evaluate the safety and effectiveness of risdiplam for the treatment of SMA patients in the routine clinical setting. All treatment choices in this study will be at the discretion of the treating physician according to local standard of care or best practice and are independent of participation in this study. No additional laboratory or other diagnostic tests will be performed on patients due to their participation in this study. No study-specific site visits are mandated by the study protocol.

 
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