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ALN-TTR02

Phase 1

TTR-mediated Amyloidosis | Small molecule | Other |Alnylam Pharmaceuticals, Inc.|Last Updated: Jan 15, 2013

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
RandomizedPLACEBO_CONTROLLED
Total Trials1
Total Enrollment17
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01559077Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer SubjectsPHASE1 COMPLETED 17 — — Mar 1, 2012Nov 1, 2012Jan 15, 20132 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 28 days
Secondary Endpoints
Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL).
Up to 180 days
Effect of ALN-TTR02 on transthyretin (TTR), vitamin A, and retinol binding protein (RBP) (Determination of % Lowering of TTR, vitamin A and RBP to pretreatment/Baseline Levels)
Up to 56 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-TTR02ACTIVE_COMPARATOR -
Sterile Normal Saline (0.9% NaCl)PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ALN-TTR02DRUGDose levels between 0.01 and 0.5 mg/kg by intravenous (IV) infusion
Sterile Normal Saline (0.9% NaCl)DRUGCalculated volume to match active comparator
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Eligibility Criteria
Age Range18 Years — 45 Years
SexALL
Healthy VolunteersYes
Study Sites2

Inclusion Criteria: * Body mass index must be between 18.0 kg/m2 and ≤ 31.5 kg/m2; * Non-smokers for at least 3 months preceding screening; * Females subjects must be of non-childbearing potential e.g., post-menopausal or pre-menopausal with surgical sterilization; * Males agree to use appropriate ...

Countries:United Kingdom
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