FIREFISH
E561096
FIREFISH is a pivotal clinical trial evaluating the efficacy and safety of risdiplam in infants with Type 1 spinal muscular atrophy.
Observed surface forms (2)
| Surface form | Occurrences |
|---|---|
| FIREFISH Part 1 | 0 |
| FIREFISH Part 2 | 0 |
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
NERFINISHED
ⓘ
Hammersmith Infant Neurological Examination Section 2 NERFINISHED ⓘ overall survival ⓘ ventilation-free survival ⓘ |
| developedWith |
PTC Therapeutics
NERFINISHED
ⓘ
SMA Foundation NERFINISHED ⓘ |
| 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 NERFINISHED ⓘ |
| 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
NERFINISHED
ⓘ
infantile-onset spinal muscular atrophy ⓘ |
| monitorsSafetyParameters |
adverse events
ⓘ
laboratory abnormalities ⓘ ophthalmologic findings ⓘ |
| relatedTo |
JEWELFISH clinical trial
ⓘ
RAINBOWFISH clinical trial ⓘ SUNFISH clinical trial NERFINISHED ⓘ |
| sponsor |
F. Hoffmann-La Roche Ltd
NERFINISHED
ⓘ
Roche NERFINISHED ⓘ |
| 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 ⓘ |
Referenced by (1)
Full triples — surface form annotated when it differs from this entity's canonical label.