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ALN-TTRSC for administration

Phase 2

TTR-mediated Amyloidosis | Small molecule | Rare Disease |Alnylam Pharmaceuticals, Inc.|Last Updated: Mar 20, 2023

Target and mechanism

ModalitySmall molecule

Also known as ALN-TTRSC (revusiran) for subcutaneous administration

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment26

FDA Designations

No designations recorded

Clinical trial landscape

ALN-TTRSC for administration · 1 trial · 1 indication

Phase 2 1
NCT01981837Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac AmyloidosisTTR-mediated Amyloidosis
COMPLETED26 Analytics
PHASE2COMPLETED
Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
TTR-mediated Amyloidosis
NCT0198183726
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Study Endpoints

Primary Endpoints

The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Up to 63 days

Secondary Endpoints

Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC, CL, Vss, Vz)
Up to 90 days
Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels)
Up to 90 days
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ALN-TTRSC (revusiran)EXPERIMENTAL -

Interventions

NameTypeDescription
ALN-TTRSC (revusiran) for subcutaneous administrationDRUG -
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Eligibility Criteria

Age Range18 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * TTR cardiac amyloidosis; * Women of child-bearing potential (WOCBP) must have a negative pregnancy test, cannot be breast feeding, and must be willing to use 2 highly effective methods of contraception; * Male subjects agree to use appropriate contraception; * Adequate blood c...

Countries:United StatesUnited Kingdom
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