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ALN-TTR02 administered by infusion

Phase 2

TTR-mediated Amyloidosis | Small molecule | Rare Disease |Alnylam Pharmaceuticals, Inc.|Last Updated: Apr 19, 2024

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment27

FDA Designations

No designations recorded

Clinical trial landscape

ALN-TTR02 administered by infusion · 1 trial · 1 indication

Phase 2 1
NCT01961921The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)TTR-mediated Amyloidosis
COMPLETED27 Analytics
PHASE2COMPLETED
The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
TTR-mediated Amyloidosis
NCT0196192127
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Study Endpoints

Primary Endpoints

The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation
From Baseline up to 56 days post last dose

An AE is any untoward medical occurrence in a patient or clinical investigational patient administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.

Secondary Endpoints

Percentage Change From Baseline in Serum TTR Levels
From Baseline up to 56 days post last dose
Change From Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7)
Baseline, Month 24
Change From Baseline in Quality of Life and Disability as Assessed by the EuroQoL (Quality of Life)-5 Dimensions (EQ-5D), EuroQoL Visual Analog Scale (EQ-VAS) Questionnaires and Rasch-built Overall Disability Scale (R-ODS)
Baseline, Month 24
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ALN-TTR02 (patisiran)EXPERIMENTAL -

Interventions

NameTypeDescription
ALN-TTR02 (patisiran) administered by intravenous (IV) infusionDRUG -
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Eligibility Criteria

Age Range18 Years to 85 Years
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: * Previously received and tolerated ALN-TTR02 (patisiran) in Study ALN-TTR02-002. * Adequate Karnofsky performance status, liver function, and renal function. Exclusion Criteria: * Pregnant or nursing. * Has had a liver transplant. * Has a New York Heart Association heart fail...

Countries:United StatesBrazilFranceGermanyPortugalSpainSweden
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