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IRCT20131012014988N8 IRCT 2023-10-22 Tabriz University of Medical Sciences Evaluation of the effecacy and safety of Risdiplam and Nusinersen in patients with Spinal Muscular Atrophy ‎‎(SMA) 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 Evaluation of the effecacy and safety of Risdiplam and Nusinersen in patients with Spinal Muscular Atrophy ‎‎(SMA) 2023-10-23 anticipated 40 Complete https://irct.ir/trial/72852 interventional Randomization: N/A, Blinding: Not blinded, Placebo: Not used, Assignment: Parallel, Purpose: Treatment. N/A Spinal muscular atrophy. Intervention 1: Intervention group: spinraza group. Intervention 2: Intervention group: risdiplam group. Undecided - It is not yet known if there will be a plan to make this available Justification or reason for indecision in sharing IPD is Respecting privacy and personal data of participants
public Dr. Mohammad Barzegar
No 3, maryam street, Golbad street, tabriz
tabriz Iran (Islamic Republic of) 5143377505 +98 914 117 0492 mm_barzegar@yahoo.com Tabriz University of Medical Sciences scientific Dr. Mohammad Barzegar
No 3, maryam street, Golbad street, tabriz
tabriz Iran (Islamic Republic of) 5143377505 +98 914 117 0492 mm_barzegar@yahoo.com Tabriz University of Medical Sciences Iran (Islamic Republic of) 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 no limit 10 years Both 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) G12 Spinal muscular atrophy and related syndromes Treatment - Drugs Treatment - Drugs Intervention group: spinraza group Intervention group: risdiplam group 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. 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. 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. Board of Trustees of the Ministry of Health Approved 2023-08-13 Ethics Committee of Tabriz University of Medical Sciences Golgasht Ave, medicine faculty Tabriz East Azarbaijan Iran (Islamic Republic of)