Evaluation of the effecacy and safety of Risdiplam and Nusinersen in patients with Spinal Muscular Atrophy ??(SMA)

Status

Recruiting

Phases

Unknown

Study type

Interventional

Source

IRCT

Registry ID

IRCT20131012014988N8

Enrollment

Registered

2023-10-22

Start date

2023-10-23

Completion date

Unknown

Last updated

2023-11-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Spinal muscular atrophy. Spinal muscular atrophy and related syndromes

Interventions

Intervention 1: Intervention group: spinraza group. Intervention 2: Intervention group: risdiplam group.

Eligibility

Sex/Gender

All

Age

No minimum to 10 Years

Inclusion 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

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)

Design outcomes

Primary

Measure	Time frame
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.	—

Measure

Time frame

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.

—

Countries

Iran (Islamic Republic of)

Contacts

Public ContactDr. Mohammad Barzegar

Tabriz University of Medical Sciences

mm_barzegar@yahoo.com+98 914 117 0492

Outcome results

None listed

Source: IRCT (via WHO ICTRP) · Data processed: Feb 4, 2026