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ALN-TTR01

Phase 1

Transthyretin Mediated Amyloidosis (ATTR) | Small molecule | Other |Alnylam Pharmaceuticals, Inc.|Last Updated: May 24, 2012

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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01148953Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) AmyloidosisPHASE1 COMPLETED 32Jun 1, 2010Feb 1, 2012May 24, 20124 France, Portugal +2
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Study Endpoints
Primary Endpoints
The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.
Up to 28 Days
Secondary Endpoints
Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL)
Up to 70 days
Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level)
up to 70 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-TTR01ACTIVE_COMPARATOR -
Sterile Normal Saline (0.9% NaCl)PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ALN-TTR01DRUGDose levels between 0.01 and 1.0 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 — N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Diagnosis of TTR amyloidosis * Adequate blood counts, liver and renal function * Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control * Males agree to use appropriate contraception * Wi...

Countries:FrancePortugalSwedenUnited Kingdom
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