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ALN-TTRSC for administration

Phase 2

TTR-mediated Amyloidosis | RNA therapy | Rare Disease |Alnylam Pharmaceuticals, Inc.|Trials Updated: Mar 20, 2023

ALN-TTRSC for administration target and mechanism

ModalityRNA therapy

Also known as ALN-TTRSC (revusiran) for subcutaneous administration

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment26

FDA Designations

No designations recorded

ALN-TTRSC for administration clinical trials

ALN-TTRSC for administration · 1 trial · 1 indication

Phase 2 1
NCT01981837Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac AmyloidosisTTR-mediated Amyloidosis
COMPLETED26 Analytics
PHASE2COMPLETED
Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
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, AUC, CL, Vss, Vz)
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
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ALN-TTRSC (revusiran)EXPERIMENTAL -

Interventions

NameTypeDescription
ALN-TTRSC (revusiran) for subcutaneous administrationDRUG -
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Eligibility Criteria

Age Range18 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * TTR cardiac amyloidosis; * Women of child-bearing potential (WOCBP) must have a negative pregnancy test, cannot be breast feeding, and must be willing to use 2 highly effective methods of contraception; * Male subjects agree to use appropriate contraception; * Adequate blood c...

Countries:United StatesUnited Kingdom
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Frequently asked questions about ALN-TTRSC for administration

What is ALN-TTRSC used for?

ALN-TTRSC is an investigational RNA therapy being developed for TTR-mediated amyloidosis, a rare disease in which transthyretin protein deposits build up in tissues. The Phase 2 study of ALN-TTRSC enrolled patients with transthyretin cardiac amyloidosis, the form affecting the heart. It is not an approved treatment.

Who is developing ALN-TTRSC?

ALN-TTRSC is being developed by Alnylam Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker ALNY. The company sponsored the Phase 2 study of the drug in patients with transthyretin cardiac amyloidosis.

What phase is ALN-TTRSC in?

ALN-TTRSC is in Phase 2 development. One Phase 2 trial has been completed, and no trials of the drug are currently active. The completed study enrolled 26 patients with transthyretin cardiac amyloidosis and was conducted in the United States and the United Kingdom.

What clinical trial was conducted for ALN-TTRSC?

ALN-TTRSC was studied in NCT01981837, a Phase 2 trial titled "Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis." The trial enrolled 26 patients aged 18 years and older, was uncontrolled, and has been completed.

Is ALN-TTRSC the same as revusiran?

Yes. ALN-TTRSC is also known as revusiran, and the subcutaneous formulation is referred to as ALN-TTRSC (revusiran) for subcutaneous administration. The Phase 2 trial in transthyretin cardiac amyloidosis used the revusiran name in its title.

Is ALN-TTRSC FDA approved?

ALN-TTRSC is not approved. It is an investigational RNA therapy that has completed a single Phase 2 trial in TTR-mediated amyloidosis, specifically transthyretin cardiac amyloidosis, and no trials of the drug are currently active.