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

Phase 1

• Conditions: Transthyretin (TTR) Cardiac Amyloidosis; MedDRA version: 19.0 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2014-001229-34-GB
• Lead Sponsor: Alnylam Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-10-03
• Study Completion: 2017-02-22
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 25

Inclusion Criteria

1. Previously received and tolerated ALN-TTRSC in Study ALN-TTRSC-002; and completed Study ALN-TTRSC-002 through the Day 90 visit

2. Adequate liver function, demonstrated by an aspartate transaminase and alanine transaminase = 2.5 × the upper limit of normal, total bilirubin< 2 g/dL (34.2 µmol/L), and albumin > 3 g/dL (> 4.35 µmol/L)

3. Women of child-bearing potential (WOCBP) must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception prior to Screening/Baseline, throughout study participation, and for 1 month after last dose administration

4. Males who agree to use appropriate means of contraception throughout study participation until 1 month after last dose administration

5. Patient, or patient’s legal representative, is able and willing to provide written informed consent and the patient is willing to comply with the study requirements.
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 3
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 22

Exclusion Criteria

1. Estimated Glomerular Filtration Rate < 20 mL/min/1.73m2 (using the Modification of Diet in Renal Disease [MDRD] formula)

2. Uncontrolled hypertension

3. Uncontrolled ischemic heart disease

4. Uncontrolled clinically significant cardiac arrhythmia

5. Untreated hypo- or hyperthyroidism

6. Prior major organ transplant

7. Known or suspected systemic bacterial, viral, parasitic, or fungal infection

8. Seropositive for hepatitis B virus, hepatitis C virus (HCV) or known to be human immunodeficiency virus positive

9. 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

10. Discontinued ALN-TTRSC-002 study due to a treatment-related AE

11. Metastatic cancer within the past 5 years

12. Any conditions which, in the opinion of the Investigator, would make the patient unsuitable for enrollment or could interfere with the patient’s participation in, or completion of, the study

13. History of allergic reaction to an oligonucleotide or GalNAc

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified