Investigating the effectiveness and side effects of Spinaraza and Risidiplam on the motor performance of patients with spinal muscular atrophy: a non-controlled trial
Investigating the effectiveness and side effects of Spinaraza and Risidiplam on the motor performance of patients with spinal muscular atrophy
Design
An interventional Study to investigate the effectiveness and side effects of 2 FDA-approved drugs for SMA patients, without a control group, non-randomized, no blinding
Settings and conduct
Suspected cases of SMA are called from Hamadan and Kermanshah, and the confirmed cases with informed consent are entered
Participants/Inclusion and exclusion criteria
Patients with a final diagnosis of SMA by a pediatric neurologist chosen for the research project in Hamadan and Kermanshah provinces, Patients who themselves or their legal guardians have informed consent to inject drugs and participate in the study
Intervention groups
Patients who receive Spinraza
Patients who receive Risdiplam
Main outcome variables
The score of motor function tests (Hammersmith and HINE)
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20120821010634N4
Registration date:2023-05-27, 1402/03/06
Registration timing:registered_while_recruiting
Last update:2023-05-27, 1402/03/06
Update count:0
Registration date
2023-05-27, 1402/03/06
Registrant information
Name
Afshin Fayazi
Name of organization / entity
Hamedan University of Medical Sciences
Country
Iran (Islamic Republic of)
Phone
+98 81 1264 0075
Email address
afshin_fay@yahoo.com
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2023-04-21, 1402/02/01
Expected recruitment end date
2024-09-19, 1403/06/29
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Investigating the effectiveness and side effects of Spinaraza and Risidiplam on the motor performance of patients with spinal muscular atrophy: a non-controlled trial
Public title
effectiveness and side effects of Spinaraza and Risidiplam on the motor performance of patients with spinal muscular atrophy
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
SMA clinical diagnosis by pediatric neurologist
SMA diagnosis according to genetic test
Exclusion criteria:
The patient do not have a consent
Age
No age limit
Gender
Both
Phase
N/A
Groups that have been masked
No information
Sample size
Target sample size:
30
Randomization (investigator's opinion)
Not randomized
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Parallel
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics committee of Hamadan University of Medical Sciences
Street address
Deputy of research, Hamadan University of Medical Sciences, Shahid Fahmideh Street
City
Hamadan
Province
Hamadan
Postal code
6517838678
Approval date
2023-03-04, 1401/12/13
Ethics committee reference number
IR.UMSHA.REC.1401.1046
Health conditions studied
1
Description of health condition studied
Spinal Muscular Atrophy
ICD-10 code
G12
ICD-10 code description
Spinal muscular atrophy and related syndromes
Primary outcomes
1
Description
Assessment of Motor Function
Timepoint
one month, 3 months, and 6 months after drug initiation
Method of measurement
Assessment of motor function based on Hammersmith and HINE scales
2
Description
Clinical and laboratory side effects of drugs
Timepoint
one month, 3 months, and 6 month after drug initiation
Method of measurement
fulfilling check list according to patient reports, requesting CBC, liver and renal function tests
Secondary outcomes
empty
Intervention groups
1
Description
Intervention group: patients that receive Spinraza. intrathecal injection of 5 ml (12mg) of the drug after drainage of 5 ml from CSF. Injections are planned for days 0, 14,28, 63, and every 4 months after that. (manufactured by Biogen and Alcyone Therapeutics)
Category
Treatment - Drugs
2
Description
Intervention group: patients that receive Risdiplam, this drug is prescribed as an oral daily dosage according to age and weight. (under 2 months: 0.15 mg/kg, two months to two years: 0.2 mg/kg, more than two years and less than 20 kg: 0.25 mg/kg, more than two years and more than 20 kg: 5 mg)
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Besat Hospital (Hamadan)
Full name of responsible person
Afshin Fayazi
Street address
Motahari Blvd
City
Hamadan
Province
Hamadan
Postal code
6514845411
Phone
+98 81 3264 0030
Email
Besat@umsha.ac.ir
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Hamedan University of Medical Sciences
Full name of responsible person
Reza Shokouhi
Street address
Shaid Fahmideh street
City
Hamadan
Province
Hamadan
Postal code
6517838678
Phone
+98 81 3131 0000
Email
info.research@umsha.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Hamedan University of Medical Sciences
Proportion provided by this source
100
Public or private sector
Public
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Academic
Person responsible for general inquiries
Contact
Name of organization / entity
Hamedan University of Medical Sciences
Full name of responsible person
Afshin Fayazi
Position
Associate professor
Latest degree
Subspecialist
Other areas of specialty/work
Pediatrics
Street address
Besat Hospital, Motahari Blvd
City
Hamadan
Province
Hamadan
Postal code
6514845411
Phone
+98 81 3264 0030
Email
afshin_fay@yahoo.com
Person responsible for scientific inquiries
Contact
Name of organization / entity
Hamedan University of Medical Sciences
Full name of responsible person
Afshin Fayazi
Position
Associate professor
Latest degree
Subspecialist
Other areas of specialty/work
Pediatrics
Street address
Besat hospital, Motahari Blvd
City
Hamadan
Province
Hamadan
Postal code
6514845411
Phone
+98 81 3264 0030
Email
Afshin_fay@yahoo.com
Person responsible for updating data
Contact
Name of organization / entity
Hamedan University of Medical Sciences
Full name of responsible person
Afshin Fayazi
Position
Associate professor
Latest degree
Subspecialist
Other areas of specialty/work
Pediatrics
Street address
Besat hospital, Motahari Blvd
City
Hamadan
Province
Hamadan
Postal code
6514845411
Phone
+98 81 3264 0030
Email
Afshin_fay@yahoo.com
Sharing plan
Deidentified Individual Participant Data Set (IPD)
No - There is not a plan to make this available
Justification/reason for indecision/not sharing IPD
Respecting the privacy and personal data of participants