FIREFISH

E561096

FIREFISH is a pivotal clinical trial evaluating the efficacy and safety of risdiplam in infants with Type 1 spinal muscular atrophy.

All labels observed (2)

Label Occurrences
FIREFISH canonical 1
FIREFISH Part 2 1

How this entity was disambiguated

Statements (47)

Predicate Object
instanceOf clinical trial ⓘ
confirmatory efficacy and safety study ⓘ
dose-finding study ⓘ
pivotal trial ⓘ
assessesOutcomeMeasure Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders ⓘ
Hammersmith Infant Neurological Examination Section 2 ⓘ
overall survival ⓘ
ventilation-free survival ⓘ
developedWith PTC Therapeutics ⓘ
SMA Foundation ⓘ
enrollsAgeGroup infants aged 1 to 7 months at first dose ⓘ
evaluates risdiplam ⓘ
hasCondition 5q-associated spinal muscular atrophy Type 1 ⓘ
hasDesign multicenter ⓘ
open-label ⓘ
two-part study ⓘ
hasGeographicScope international ⓘ
hasInterventionModel single-group assignment ⓘ
hasObjective determine the dose of risdiplam for Part 2 ⓘ
hasOutcome demonstrated clinically meaningful motor function improvement ⓘ
demonstrated survival benefit compared with natural history ⓘ
hasPart FIREFISH Part 1 ⓘ
FIREFISH Part 2 ⓘ
linked to: FIREFISH
hasPhase Phase 2 ⓘ
hasPopulation infants with Type 1 spinal muscular atrophy ⓘ
hasPrimaryEndpoint proportion of infants sitting without support ⓘ
survival without permanent ventilation ⓘ
hasPrimaryObjective evaluate efficacy of risdiplam in infants with Type 1 spinal muscular atrophy ⓘ
hasPublicationType peer-reviewed clinical trial report ⓘ
hasSecondaryObjective evaluate safety of risdiplam in infants with Type 1 spinal muscular atrophy ⓘ
hasTherapeuticArea neuromuscular disease ⓘ
rare disease ⓘ
investigatesIndication Type 1 spinal muscular atrophy ⓘ
infantile-onset spinal muscular atrophy ⓘ
monitorsSafetyParameters adverse events ⓘ
laboratory abnormalities ⓘ
ophthalmologic findings ⓘ
relatedTo JEWELFISH clinical trial ⓘ
RAINBOWFISH clinical trial ⓘ
SUNFISH clinical trial ⓘ
sponsor F. Hoffmann-La Roche Ltd ⓘ
linked to: Roche

Roche ⓘ
studiesDrug risdiplam ⓘ
supportsRegulatoryDecision approval of risdiplam for treatment of spinal muscular atrophy ⓘ
targetsPathology survival motor neuron protein deficiency ⓘ
usesDrugMechanism SMN2 pre-mRNA splicing modification by risdiplam ⓘ
usesRouteOfAdministration oral administration of risdiplam ⓘ

How these facts were elicited

Referenced by (2)

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

FIREFISH → hasPart → FIREFISH Part 2 ⓘ
linked to: FIREFISH