Clinical trial • Phase IV • Musculoskeletal

Risdiplam for Spinal muscular atrophy

Phase IV trial of Risdiplam for Spinal muscular atrophy. open-label. 31 participants.

Overview

open-label Phase IV trial across 4 sites in Germany, Poland.

Recruits 31 paediatric patients.

Vulnerable Population: Vulnerable population selected: pediatric patients. Subject information and informed consent forms are listed in the application (documents L1_SIS_ICF Main with language variants including EN, PL, AR, TR, UK and country-of-treatment variants). Specific consent/assent procedures or age-specific consent handling are not detailed in the provided record.

{"criterion_text":"- Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the survival of motor neuron 1 (SMN1) telomeric gene"}
{"criterion_text":"- Confirmed presence of two survival of motor neuron 2 (SMN2) centromeric gene copies as documented through laboratory testing"}
{"criterion_text":"- Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically (<=3 months of symptoms)"}
{"criterion_text":"- Has received onasemnogene abeparvovec for SMA no less than 13 weeks (- 2 weeks) prior to enrollment"}
{"criterion_text":"- If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration"}
{"criterion_text":"- In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 6 months or less) documented by 2 individual time points in the functions as follows: - Swallowing AND - One (1) additional function/ability (respiratory, motor function, other) per appropriate expectation"}

{"criterion_text":"- Previous or current enrollment in investigational study prior to initiation of study treatment"}
{"criterion_text":"- Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information"}
{"criterion_text":"- Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide"}
{"criterion_text":"- Patients requiring invasive ventilation or tracheostomy"}
{"criterion_text":"- Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration"}
{"criterion_text":"- Concomitant or previous use of an anti-myostatin agent"}

{"endpoint_text":"- Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment in this population of SMA patients","definition_or_measurement_approach":"Change from baseline in the raw score of BSID-III Gross Motor Score measured at 72 weeks of risdiplam treatment"}

{"endpoint_text":"- 1. Incidence and severity of adverse events, with severity determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE v5.0)","definition_or_measurement_approach":"Incidence and severity assessed and graded per NCI CTCAE v5.0"}
{"endpoint_text":"- 2. Incidence and severity of serious adverse events","definition_or_measurement_approach":"Recording of incidence and severity of serious adverse events (SAEs) as per standard SAE reporting procedures"}
{"endpoint_text":"- 3. Incidence of treatment discontinuation due to adverse events","definition_or_measurement_approach":"Number and proportion of participants discontinuing treatment due to adverse events"}

Planned Sample Size: 31
Recruitment Window Months: 45
Consent Approach: Subject information and informed consent forms are available in multiple language versions (documents L1_SIS_ICF Main: EN, PL, AR, TR, UK and country-of-treatment variants). Specific details on who provides consent or assent and age-specific consent/assent procedures are not provided in the available record.

{"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"Drug Management","organisation_type":"Pharmaceutical company"}
{"country":"Netherlands","full_name":"Syneos Health Netherlands B.V.","duties_or_roles":"Global CRO","organisation_type":"Pharmaceutical company"}

Investigational Product Name: Evrysdi 0.75 mg/mL powder for oral solution
Active Substance: Risdiplam
Modality: Small molecule
Routes Of Administration: ORAL, NASOGASTRIC TUBE OR PERCUTANEOUS ENDOSCOPIC GASTROSTOMY TUBE USE
Route: ORAL, NASOGASTRIC TUBE OR PERCUTANEOUS ENDOSCOPIC GASTROSTOMY TUBE USE
Authorisation Status: Marketing authorisation (EU MA: EU/1/21/1531/001)
Maximum Dose: 5 mg per day (max daily dose amount: 5 mg)

Investigational Product Name: RO7034067
Active Substance: Risdiplam
Modality: Small molecule
Routes Of Administration: ORAL, NASOGASTRIC TUBE OR PERCUTANEOUS ENDOSCOPIC GASTROSTOMY TUBE USE
Route: ORAL, NASOGASTRIC TUBE OR PERCUTANEOUS ENDOSCOPIC GASTROSTOMY TUBE USE
Authorisation Status: Investigational / Sponsor product (RO 703-4067/F13-02)
Maximum Dose: 5 mg per day (max daily dose amount: 5 mg)

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