NL-OMON49730
Recruiting
Phase 2
An open-label study to investigate the safety, tolerability, and Pharmacokinetics/Pharmacodynamics of risdiplam (RO7034067) in adult and pediatric patients with spinal muscular atrophy - JEWELFISH
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Spinal muscular athrophy
- Sponsor
- Hoffmann-La Roche
- Enrollment
- 10
- Status
- Recruiting
- Last Updated
- last year
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Males and females 6 months to 60 years of age inclusive (at screening)
- •2\. Confirmed diagnosis of 5q\-autosomal recessive SMA, including:
- •\- Genetic confirmation of homozygous deletion or heterozygosity predictive of
- •loss of function of the SMN1 gene.
- •\- Clinical history, signs, or symptoms attributable to SMA.
- •3\. Previous enrollment in Study BP29420 (Moonfish) with the splicing modifier
- •RO6885247 or previous treatment with any of the following:
- •\- 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
Exclusion Criteria
- •1\. Inability to meet study requirements.
- •2\. Concomitant participation in any investigational drug or device study.
- •3\. 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.
- •4\. Any history of gene or cell therapy, with the exception of AVXS\-101\.
- •5\. Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular
- •system diseases as considered to be clinically significant by the Investigator.
- •6\. Inadequate venous or capillary blood access for the study procedures, in the
- •opinion of the Investigator.
Outcomes
Primary Outcomes
Not specified
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