1-Comparison of the efficacy of risdiplam and nusinersen in SMA patients
2-Comparison of side effects of risdiplam and nusinersen in SMA patients
3-Comparison of the amount of NCS changes in these two groups
Design
Clinical trial without control group, with parallel groups, not blinded, not randomized, phase 1 on 40 patients.
Settings and conduct
Before starting the medication, full clinical examinations of the patients will be performed and the growth indices, laboratory findings and scoring of the patients' motor abilities based on the criteria of CHOP INTEND, RULM, HFMSE and SMAIS will be used. The mentioned performance will be re-evaluated and recorded during the study period with intervals of 0, 1, 3 and 6 months and if possible 12 months. Also, unwanted side effects of drugs will be investigated. The initial NCV is performed at the time of entering the study and is repeated for patients with SMA type 1 every 3 months during the study period and for type 2 and 3 patients with 6 month intervals. Also, laboratory tests is repeated at the end of the 4th week and the 6th month.
Participants/Inclusion and exclusion criteria
Genetically confirmed SMA T 1, 2 and 3 patients with SMN2 copy number between 2 and 4 and age less than 10 years
Intervention groups
Spinraza Group and Risdiplam Group
Main outcome variables
Assessment of Motor Function, Amplitude changes in NCS and drugs side effects
General information
Reason for update
Acronym
Spinal Muscular Atrophy(SMA), Survival motor neuron2 (SMN2), Central nervous system (CNS), Cerebrospinal Fluid (CSF), Homozygous Gene Mutation or Deletion (5q SMA), Children's hospital of philadephia infant test of neuromuscular disorders (CHOP INT
IRCT registration information
IRCT registration number:IRCT20131012014988N8
Registration date:2023-10-22, 1402/07/30
Registration timing:prospective
Last update:2023-10-22, 1402/07/30
Update count:0
Registration date
2023-10-22, 1402/07/30
Registrant information
Name
Mohammad Barzegar
Name of organization / entity
Pediatric Health Research Center,Tabriz University of Medical Sciences
Country
Iran (Islamic Republic of)
Phone
+98 41 3526 2265
Email address
phrc_mb@tbzmed.ac.ir
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2023-10-23, 1402/08/01
Expected recruitment end date
2024-10-22, 1403/08/01
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Evaluation of the effecacy and safety of Risdiplam and Nusinersen in patients with Spinal Muscular Atrophy (SMA)
Public title
Evaluation of the effecacy and safety of Risdiplam and Nusinersen in patients with Spinal Muscular Atrophy (SMA)
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Definitive genetic diagnosis of homozygous gene mutation or deletion(5q SMA)
Patients with equal or less than 10 years old
SMN2 copy number is 2 to 4
Exclusion criteria:
Active infection requiring systemic antibiotic or antiviral treatment up to 2 weeks after recovery
Existence of concomitant incurable diseases (history of asphyxia, seizures, severe liver, cardiovascular, and endocrine envolvement)
Liver failure (due to the possibility of increased drug levels) (Child Pugh class C)
receiving metformin (for risdiplam)
Lactation and pregnancy
History of recent eye diseases (uncontrolled glaucoma) (for Risdiplam)
Existence of severe kyphoscoliosis or corrective surgery (for Nocinersen)
Having a CNS shunt and CSF catheter (for Nocinersen)
History of bacterial meningitis (for nosinersen)
Age
To 10 years old
Gender
Both
Phase
N/A
Groups that have been masked
No information
Sample size
Target sample size:
40
Randomization (investigator's opinion)
N/A
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 Tabriz University of Medical Sciences
Street address
Golgasht Ave, medicine faculty
City
Tabriz
Province
East Azarbaijan
Postal code
5157610144
Approval date
2023-08-13, 1402/05/22
Ethics committee reference number
IR.TBZMED.REC.1402.402
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, three months, and six months, and if possible, one year after starting the drug
Method of measurement
At the end of the 1st, 3rd, and 6th months from the start of treatment, a pediatric neurologist or a pediatric neurology fellowship will visit and perform CHOP INTENT, HFMSE, RULM, and SMAIS tests for patients.
2
Description
Amplitude changes in NCS
Timepoint
Every three months for SMA-1 and every six months for SMA-2,3
Method of measurement
A physical medicine and rehabilitation specialist (a permanent member of the research group) will evaluate the CMAP amplitude changes of a single nerve.
3
Description
Drug side effects evaluation
Timepoint
Every 2 weeks in the first month and then monthly by asking the patients themselves or their parents
Method of measurement
by asking the patients themselves or their parents
Secondary outcomes
empty
Intervention groups
1
Description
Intervention group: spinraza group
Category
Treatment - Drugs
2
Description
Intervention group: risdiplam group
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
zahra mardani azari children hospital
Full name of responsible person
Dr mohammad baezegar
Street address
Mashhouri st. Mardani Azari St. Khavaran Town
City
tabriz
Province
East Azarbaijan
Postal code
5143377505
Phone
+98 41 3159 2105
Email
mardaniazar@tbzmed.ac.ir
Web page address
https://mardaniazari.tbzmed.ac.ir/
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Tabriz University of Medical Sciences
Full name of responsible person
Dr. Parviz Shahabi
Street address
Golgasht Street
City
Tabriz
Province
East Azarbaijan
Postal code
5143377505
Phone
+98 41 3335 7310
Email
dabirkhanecent@tbzmed.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
No
Title of funding source
Board of Trustees of the Ministry of Health
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
Tabriz University of Medical Sciences
Full name of responsible person
Dr. Mohammad Barzegar
Position
professor
Latest degree
Subspecialist
Other areas of specialty/work
Pediatrics
Street address
No 3, maryam street, Golbad street, tabriz
City
tabriz
Province
East Azarbaijan
Postal code
5143377505
Phone
+98 914 117 0492
Fax
+98 41 3159 2105
Email
mm_barzegar@yahoo.com
Person responsible for scientific inquiries
Contact
Name of organization / entity
Tabriz University of Medical Sciences
Full name of responsible person
Dr. Mohammad Barzegar
Position
professor
Latest degree
Subspecialist
Other areas of specialty/work
Pediatrics
Street address
No 3, maryam street, Golbad street, tabriz
City
tabriz
Province
East Azarbaijan
Postal code
5143377505
Phone
+98 914 117 0492
Email
mm_barzegar@yahoo.com
Person responsible for updating data
Contact
Name of organization / entity
Tabriz University of Medical Sciences
Full name of responsible person
Dr. Bita poorshiri
Position
assistant professor
Latest degree
Subspecialist
Other areas of specialty/work
Pediatrics
Street address
aras building, tavanir street, tabriz
City
tabriz
Province
East Azarbaijan
Postal code
5138933171
Phone
+98 914 301 3061
Email
bpskam@yahoo.com
Sharing plan
Deidentified Individual Participant Data Set (IPD)
Undecided - It is not yet known if there will be a plan to make this available
Study Protocol
Undecided - It is not yet known if there will be a plan to make this available
Statistical Analysis Plan
Undecided - It is not yet known if there will be a plan to make this available
Informed Consent Form
Undecided - It is not yet known if there will be a plan to make this available
Clinical Study Report
Undecided - It is not yet known if there will be a plan to make this available
Analytic Code
Undecided - It is not yet known if there will be a plan to make this available
Data Dictionary
Undecided - It is not yet known if there will be a plan to make this available