RISDIPLAM for Spinal muscular atrophy

Inclusion criteria

• {"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 than13 weeks (- 2 weeks),, but not more than, 30 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":"- Parent or caregiver of patient willing to consider the use of non-invasive ventilation during the study, as recommended by the investigator"}

Exclusion criteria

• {"criterion_text":"- Previous or current enrolment 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 and patients requiring awake non-invasive ventilation or with awake hypoxemia (SaO2 < 95%) with or without ventilator support"}
• {"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"}

Primary endpoints

• {"endpoint_text":"- 1.Change from baseline in the raw score of Bayley Scales of Infant and Toddler DevelopmentTM, 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 raw BSID-III Gross Motor Score measured at 72 weeks of risdiplam treatment"}

Secondary endpoints

• {"endpoint_text":"- Incidence and severity of adverse events, with severity determined according to the NCI CTCAE v5.0","definition_or_measurement_approach":"Incidence and severity of AEs assessed and graded according to NCI CTCAE v5.0"}
• {"endpoint_text":"- Incidence and severity of serious adverse events","definition_or_measurement_approach":"Incidence and severity counts of serious adverse events"}
• {"endpoint_text":"- Incidence of treatment discontinuation due to adverse events","definition_or_measurement_approach":"Incidence (number/proportion) of subjects discontinuing treatment because of AEs"}

Germany

Earliest CTIS Part Ii Submission Date

11-01-2024

Latest Decision Or Authorization Date

20-03-2026

Processing Time Days

799

Number Of Sites

2

Number Of Participants

8

Poland

Earliest CTIS Part Ii Submission Date

19-01-2024

Latest Decision Or Authorization Date

10-06-2024

Processing Time Days

143

Number Of Sites

2

Number Of Participants

7

Primary sponsor

Full Name

F. Hoffmann-La Roche AG

Organisation Type

Pharmaceutical company

Country Of Registered Address

Switzerland

Contract research organisations

Name

Syneos Health Netherlands B.V.

Responsibilities

Global CRO

Name

Almac Clinical Technologies LLC

Responsibilities

Drug Management

Name

Almac Clinical Technologies LLC

Responsibilities

3

Third parties

• {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"3","organisation_type":"Pharmaceutical company"}
• {"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"}