effectiveness and side effects of Spinaraza and Risidiplam on the motor performance of patients with spinal muscular atrophy

Investigating the effectiveness and side effects of Spinaraza and Risidiplam on the motor performance of patients with spinal muscular atrophy: a non-controlled trial

Status

Recruiting

Phases

Unknown

Study type

Interventional

Source

IRCT

Registry ID

IRCT20120821010634N4

Enrollment

Registered

2023-05-27

Start date

2023-04-21

Completion date

Unknown

Last updated

2023-05-29

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: 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

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: SMA clinical diagnosis by pediatric neurologist SMA diagnosis according to genetic test

Exclusion criteria

Exclusion criteria: The patient do not have a consent

Design outcomes

Primary

Measure	Time frame
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.;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.	—

Measure

Time frame

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.;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.

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Countries

Iran (Islamic Republic of)

Contacts

Public ContactAfshin Fayazi

Hamedan University of Medical Sciences

afshin_fay@yahoo.com+98 81 3264 0030

Outcome results

None listed

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