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ALN-TTRSC

Phase 1

TTR-mediated Amyloidosis | Small molecule | Other |Alnylam Pharmaceuticals, Inc.|Last Updated: Feb 5, 2016

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment85
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01814839A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy VolunteersPHASE1 COMPLETED 85 — — Mar 1, 2013May 1, 2015Feb 5, 20162 United Kingdom
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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, AUC0-last, CL).
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
Effect of ALN-TTRSC (revusiran) on vitamin A (Determination of % Lowering of vitamin A to pretreatment/Baseline Levels).
Up to 90 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-TTRSC (revusiran)ACTIVE_COMPARATOR -
Sterile Normal Saline (0.9% NaCl)PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ALN-TTRSC (revusiran)DRUGAscending doses of ALN-TTRSC (revusiran) by subcutaneous (SC) injection
Sterile Normal Saline (0.9% NaCl)DRUGCalculated volume to match active comparator
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Eligibility Criteria
Age Range18 Years — 55 Years
SexALL
Healthy VolunteersYes
Study Sites2

Inclusion Criteria: * Body mass index (BMI) must be ≥ 16.5 kg/m2 and ≤ 35.0 kg/m2; * Female subjects must be of non-childbearing potential; e.g., postmenopausal or pre-menopausal with surgical sterilization; * Male subjects agree to use appropriate contraception; * Adequate blood counts, liver and ...

Countries:United Kingdom
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