The Phase 2 Study of an Investigational Drug, ALN-TTRSC, in Patients with TTR Cardiac Amyloidosis

A Phase 2, Open-Label Trial to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Exploratory Clinical Activity of ALN-TTRSC in Patients with Transthyretin (TTR) Cardiac Amyloidosis

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-002856-33-GB

Enrollment

Registered

2013-09-03

Start date

2013-11-15

Completion date

Unknown

Last updated

2015-08-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transthyretin (TTR) Cardiac Amyloidosis MedDRA version: 16.1 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: ALN-TTRSC Product Code: ALN-TTRSC Pharmaceutical Form: Solution for injection INN or Proposed INN: ALN-51547 Current Sponsor code: ALN-51547 Other descriptive name: ALN-51547 Concentrati

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Males or females 18 to 80 years of age, inclusive; Biopsy-proven TTR cardiac amyloidosis NYHA Class =3 Stable congestive heart failure status and stable clinical treatment Able to walk =150 meters Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 5 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10

Exclusion criteria

Exclusion criteria: Uncontrolled hypertension; Uncontrolled ischemic heart disease; Uncontrolled clinically significant cardiac arrhythmia; Untreated hypo- or hyperthyroidism Prior major organ transplant; Known or suspected systemic bacterial, viral, parasitic, or fungal infection; Seropositive for hepatitis B virus (HBV), hepatitis C virus (HCV) or known to be human immunodeficiency virus (HIV) positive; Received an investigational agent other than tafamidis, diflunisal, doxycycline or tauroursodeoxycholic acid, or an investigational device within 30 days prior to first dose of study drug;

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the safety and tolerability of multiple doses of ALN-TTRSC ;Secondary Objective: To assess the pharmacodynamic (PD) effect of multiple dose of ALN-TTRSC on serum levels of transthyretin (TTR) To characterize the pharmacokinetics (PK) of multiple doses of ALN-TTRSC ;Primary end point(s): Safety and tolerability of multiple doses of ALN-TTRSC, including: •assessment of adverse events (AEs), •vital signs (blood pressure, pulse rate, oral body temperature and respiratory rate), •clinical laboratory safety tests (hematology, serum chemistry, thyroid function, coagulation, and urinalysis) •12-lead ECG •physical examinations ;Timepoint(s) of evaluation of this end point: •The review of AEs will be done at each visit through D63. •The other assessments will be carried out at specific timepoints between screening and D90, as described in the study schedule.	—

Measure

Time frame

Main Objective: To evaluate the safety and tolerability of multiple doses of ALN-TTRSC ;Secondary Objective: To assess the pharmacodynamic (PD) effect of multiple dose of ALN-TTRSC on serum levels of transthyretin (TTR) To characterize the pharmacokinetics (PK) of multiple doses of ALN-TTRSC ;Primary end point(s): Safety and tolerability of multiple doses of ALN-TTRSC, including: •assessment of adverse events (AEs), •vital signs (blood pressure, pulse rate, oral body temperature and respiratory rate), •clinical laboratory safety tests (hematology, serum chemistry, thyroid function, coagulation, and urinalysis) •12-lead ECG •physical examinations ;Timepoint(s) of evaluation of this end point: •The review of AEs will be done at each visit through D63. •The other assessments will be carried out at specific timepoints between screening and D90, as described in the study schedule.

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Secondary

Measure	Time frame
Secondary end point(s): •To assess the PD effect of multiple doses of ALN-TTRSC •To assess the PK profile of multiple doses of ALN-TTRSC ;Timepoint(s) of evaluation of this end point: •An assessment of the concentrations of serum TTR, will be determined at specified time points between screening and D90 , as described in the study schedule. •An assessment of the PK of ALN-TTRSC will be determined at specified time points between D0 and D90, as described in the study schedule.	—

Measure

Time frame

Secondary end point(s): •To assess the PD effect of multiple doses of ALN-TTRSC •To assess the PK profile of multiple doses of ALN-TTRSC ;Timepoint(s) of evaluation of this end point: •An assessment of the concentrations of serum TTR, will be determined at specified time points between screening and D90 , as described in the study schedule. •An assessment of the PK of ALN-TTRSC will be determined at specified time points between D0 and D90, as described in the study schedule.

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Countries

United Kingdom, United States

Contacts

Public ContactPatricia Armstrong-Project Director

PAREXEL International, Ltd.

Patricia.Armstrong@parexel.com1978495-4499

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026