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