JEWELFISH
E561098
JEWELFISH is a clinical trial study evaluating the safety and efficacy of risdiplam in individuals with spinal muscular atrophy who were previously treated with other SMA therapies.
Statements (46)
| Predicate | Object |
|---|---|
| instanceOf |
clinical trial
ⓘ
interventional study ⓘ pharmacological trial ⓘ |
| abbreviation | NCT03032172 ⓘ |
| ageGroup |
adolescents
ⓘ
adults ⓘ children ⓘ |
| aimsTo | assess safety of switching to or adding risdiplam after prior SMA therapies ⓘ |
| collaborator |
PTC Therapeutics
NERFINISHED
ⓘ
SMA Foundation NERFINISHED ⓘ |
| conditionSubtype | 5q-associated spinal muscular atrophy ⓘ |
| dataTypeCollected |
efficacy data
ⓘ
pharmacodynamic data ⓘ pharmacokinetic data ⓘ safety data ⓘ |
| design |
multicenter
ⓘ
open-label ⓘ single-arm ⓘ |
| drugClass | SMN2 splicing modifier ⓘ |
| drugTarget | SMN2 pre-mRNA splicing ⓘ |
| endpoint |
incidence of adverse events
ⓘ
laboratory abnormalities ⓘ motor function measures ⓘ pharmacokinetic parameters of risdiplam ⓘ serious adverse events ⓘ |
| evaluates | risdiplam NERFINISHED ⓘ |
| focusesOn | previously treated SMA population ⓘ |
| geographicScope | international ⓘ |
| hasOralFormulation | risdiplam NERFINISHED ⓘ |
| includesPriorTherapy |
nusinersen
NERFINISHED
ⓘ
onasemnogene abeparvovec ⓘ other investigational SMA treatments ⓘ |
| intervention | risdiplam ⓘ |
| phase | Phase 2 ⓘ |
| population | individuals with spinal muscular atrophy previously treated with other SMA therapies ⓘ |
| primaryObjective | to evaluate the safety of risdiplam in previously treated SMA patients ⓘ |
| registrationAuthority | ClinicalTrials.gov NERFINISHED ⓘ |
| routeOfAdministration | oral ⓘ |
| secondaryObjective |
to evaluate the pharmacokinetics of risdiplam
ⓘ
to explore efficacy of risdiplam ⓘ |
| sponsor |
F. Hoffmann-La Roche Ltd
NERFINISHED
ⓘ
Roche NERFINISHED ⓘ |
| sponsorCountry | Switzerland NERFINISHED ⓘ |
| studiesCondition | spinal muscular atrophy ⓘ |
| therapeuticArea |
neuromuscular disease
ⓘ
rare disease ⓘ |
Referenced by (1)
Full triples — surface form annotated when it differs from this entity's canonical label.