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.

All labels observed (1)

Label Occurrences
JEWELFISH canonical 1

How this entity was disambiguated

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 ⓘ
SMA Foundation ⓘ
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 ⓘ
focusesOn previously treated SMA population ⓘ
geographicScope international ⓘ
hasOralFormulation risdiplam ⓘ
includesPriorTherapy nusinersen ⓘ
linked to: Spinraza

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 ⓘ
routeOfAdministration oral ⓘ
secondaryObjective to evaluate the pharmacokinetics of risdiplam ⓘ
to explore efficacy of risdiplam ⓘ
sponsor F. Hoffmann-La Roche Ltd ⓘ
linked to: Roche

Roche ⓘ
sponsorCountry Switzerland ⓘ
studiesCondition spinal muscular atrophy ⓘ
therapeuticArea neuromuscular disease ⓘ
rare disease ⓘ

How these facts were elicited

Referenced by (1)

Full triples — surface form annotated when it differs from this entity's canonical label.

risdiplam → clinicalTrialProgram → JEWELFISH ⓘ