A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam in adult and pediatric patients with spinal muscular atrophy

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 167

Inclusion Criteria

1. Males and females 6 months to 60 years of age inclusive (at screening), 2. Confirmed diagnosis of 5q-autosomal recessive SMA, 3. Previous enrollment in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previous treatment with nusinersen (defined as having received = 4 doses of nusinersen, provided that the last dose was received = 90 days prior to screening),olesoxime (provided that the last dose was received = 18 months and = 90 days prior to screening) or onasemnogene abeparvovec (AVXS-101) (provided that the time of treatment was = 12 months prior to screening), 4. Adequately recovered from any acute illness at the time of screening and considered well enough to participate in the opinion of the Investigator, 5. For patients aged 2 years or younger at screening: •Receiving adequate nutrition and hydration (with or without gastrostomy) at the time of screening, in the opinion of the Investigator •Medical care meets local accepted standard of care, in the opinion of the Investigator •Would be able to complete all study procedures, measurements and visits, and the parent or caregiver of the patient has adequately supportive psychosocial circumstances, in the opinion of the Investigator •Parent or caregiver of patient is willing to consider nasogastric, nasojejunal or gastrostomy tube placement, as recommended by the Investigator, during the study to maintain safe hydration, nutrition and treatment delivery •Parent or caregiver of patient is willing to consider the use of noninvasive ventilation, as recommended by the Investigator during the study

Exclusion Criteria

1. With the exception of studies of olesoxime, AVXS-101, or nusinersen: Previous participation in any investigational drug or device study within 90 days prior to screening, or 5 half-lives of the drug, whichever is longer, 2. Any history of gene or cell therapy, with the exception of AVXS-101, 3. Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases as considered to be clinically significant by the Investigator, 4. Inadequate venous or capillary blood access for the study procedures, in the opinion of the Investigator, 5. For patients aged <2 years, hospitalization for a pulmonary event within 2months prior to screening and pulmonary function not fully recovered at the time of screening, 6. Lactating women

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Related Trials