The Study of an Investigational Drug, ALN-TTR02, for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02.

A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTR02 in Patients With Familial Amyloidotic Polyneuropathy Who Have Previously Received ALN-TTR02

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-001644-65-PT

Enrollment

Registered

2013-05-30

Start date

2013-07-19

Completion date

Unknown

Last updated

2017-04-18

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

Conditions

Transthyretin mediated amyloidosis (ATTR) MedDRA version: 14.1 Level: PT Classification code 10007509 Term: Cardiac amyloidosis System Organ Class: 10007541 - Cardiac disorders MedDRA version: 14.1 Level: PT Classification code 10019889 Term: Hereditary neuropathic amyloidosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: ALN-TTR02 Product Code: ALN-TTR02 Pharmaceutical Form: Solution for injection INN or Proposed INN: ALN-18328 Current Sponsor code: ALN-18328 Concentration unit: mg/ml milligram(s)/millil

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Previously received and tolerated ALN-TTR02 in Study ALN-TTR02-002. 2. Adequate Karnofsky performance status, liver function, and renal function. Additional inclusion criteria are utilized to assess entry in PK subgroup and for the cardiac subgroup. 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 14 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 14

Exclusion criteria

Exclusion criteria: 1. Pregnant or nursing. 2. Has had a liver transplant. 3. Has a New York Heart Association heart failure classification >2. 4. Has unstable angina. 5. Has uncontrolled clinically significant cardiac arrhythmia.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the safety and tolerability of long-term dosing with ALN-TTR02.;Secondary Objective: To assess the PD effect associated with long-term dosing of ALN-TTR02 on serum TTR. To assess changes from baseline in: • Neurologic impairment, quality of life and disability, motor function impacting activities of daily living, and nutritional status.;Primary end point(s): To evaluate the safety and tolerability of long-term dosing with ALN-TTR02: •assessment of adverse events (AEs), •12 lead electrocardiograms (ECGs), •vital signs (blood pressure, pulse rate, oral body temperature and respiratory rate), •clinical laboratory safety tests (hematology, serum chemistry, thyroid function, coagulation, and urinalysis), •ophthalmology examinations, and •physical examinations.;Timepoint(s) of evaluation of this end point: The review of AEs will be done at each visit from Day 0 through through 56 days post last dose. The other assessments will be carried out at specific timepoints between screening and 56 days post last dose, as descibed in the study schedule.	—

Measure

Time frame

Main Objective: To evaluate the safety and tolerability of long-term dosing with ALN-TTR02.;Secondary Objective: To assess the PD effect associated with long-term dosing of ALN-TTR02 on serum TTR. To assess changes from baseline in: • Neurologic impairment, quality of life and disability, motor function impacting activities of daily living, and nutritional status.;Primary end point(s): To evaluate the safety and tolerability of long-term dosing with ALN-TTR02: •assessment of adverse events (AEs), •12 lead electrocardiograms (ECGs), •vital signs (blood pressure, pulse rate, oral body temperature and respiratory rate), •clinical laboratory safety tests (hematology, serum chemistry, thyroid function, coagulation, and urinalysis), •ophthalmology examinations, and •physical examinations.;Timepoint(s) of evaluation of this end point: The review of AEs will be done at each visit from Day 0 through through 56 days post last dose. The other assessments will be carried out at specific timepoints between screening and 56 days post last dose, as descibed in the study schedule.

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Secondary

Measure	Time frame
Secondary end point(s): To assess the PD effect associated with long-term dosing of ALN-TTR02 on serum TTR. To assess changes from baseline in: • Neurologic impairment, quality of life and disability, motor function impacting activities of daily living, and nutritional status.;Timepoint(s) of evaluation of this end point: An assessment of the concentrations of serum TTR, RBP, and Vitamin A will be determined at specified time points between screening and 56 days post last dose, as described in the study schedule. Other assessments of neurologic impairment and QOL will be performed approximately every six months for approximately two years.	—

Measure

Time frame

Secondary end point(s): To assess the PD effect associated with long-term dosing of ALN-TTR02 on serum TTR. To assess changes from baseline in: • Neurologic impairment, quality of life and disability, motor function impacting activities of daily living, and nutritional status.;Timepoint(s) of evaluation of this end point: An assessment of the concentrations of serum TTR, RBP, and Vitamin A will be determined at specified time points between screening and 56 days post last dose, as described in the study schedule. Other assessments of neurologic impairment and QOL will be performed approximately every six months for approximately two years.

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Countries

Brazil, France, Germany, Portugal, Spain, Sweden, United States

Contacts

Public ContactClinical Trials Information

Medpace Finland OY

regsubmissions@medpace.com49896737916

Outcome results

None listed

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