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ALN-TTRSC

Phase 1

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

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment85

FDA Designations

No designations recorded

Clinical trial landscape

ALN-TTRSC · 1 trial · 1 indication

Phase 1 1
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 VolunteersTTR-mediated Amyloidosis
COMPLETED85 Analytics
PHASE1COMPLETED
A 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 Volunteers
TTR-mediated AmyloidosisUnlock trial analytics

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 to 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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Frequently asked questions about ALN-TTRSC

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.