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ALN-TTRSC04

Phase 1

Transthyretin-Mediated Amyloidosis | Small molecule | Rare Disease |Alnylam Pharmaceuticals, Inc.|Last Updated: Feb 17, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment96

FDA Designations

No designations recorded

Clinical trial landscape

ALN-TTRSC04 · 1 trial · 1 indication

Phase 1 1
NCT05661916A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy SubjectsTransthyretin-Mediated Amyloidosis
COMPLETED96 Analytics
PHASE1COMPLETED
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
Transthyretin-Mediated AmyloidosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Frequency of Adverse Events
Up to 20 months

Secondary Endpoints

Change from Baseline in Serum Transthyretin (TTR) Levels Over Time
Up to 18 months
Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04
Day 1 up to Day 4
Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04
Day 1 up to Day 4
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ALN-TTRSC04EXPERIMENTALParticipants will be administered a single dose of ALN-TTRSC04.
PlaceboPLACEBO_COMPARATORParticipants will be administered a single dose of placebo.

Interventions

NameTypeDescription
ALN-TTRSC04DRUGALN-TTRSC04 will be administered by subcutaneous (SC) injection.
PlaceboDRUGPlacebo will be administered by SC injection.
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Body mass index (BMI) ≥18.0 kg/m\^2 and ≤30 kg/m\^2 assessed at Screening. Exclusion Criteria: * Has an estimated glomerular filtration rate (eGFR) \<90 mL/min/1.73m\^2 at screening. * Has alanine aminotransferase (ALT), aspartate aminotransferase (AST) or direct bilirubin a...

Countries:United Kingdom
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Frequently asked questions about ALN-TTRSC04

What is ALN-TTRSC used for?

ALN-TTRSC is an investigational drug being developed for transthyretin-mediated amyloidosis (ATTR amyloidosis), a rare disease. It is currently in Phase 1 clinical development. The drug is being studied in healthy volunteers to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics.

Who makes ALN-TTRSC?

ALN-TTRSC is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company. The company's stock ticker is ALNY. The drug is currently in Phase 1 clinical trials for transthyretin-mediated amyloidosis.

What phase is ALN-TTRSC in?

ALN-TTRSC is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The drug is being studied in healthy volunteers to assess its safety and tolerability.

What clinical trials is ALN-TTRSC in?

ALN-TTRSC has been studied in three completed Phase 1 clinical trials. These include NCT01814839, NCT02797847, and NCT05661916. All trials were conducted in the United Kingdom and enrolled healthy volunteers aged 18 years and older.

Is ALN-TTRSC the same as revusiran?

ALN-TTRSC is also known as revusiran. In clinical trial NCT01814839, the drug is referred to as ALN-TTRSC (Revusiran). The drug is being developed for transthyretin-mediated amyloidosis.