NCT02797847

Brief Summary

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
80

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Jun 2016

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 18, 2016

Completed
20 days until next milestone

Study Start

First participant enrolled

June 7, 2016

Completed
7 days until next milestone

First Posted

Study publicly available on registry

June 14, 2016

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 12, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 12, 2018

Completed
Last Updated

September 21, 2018

Status Verified

September 1, 2018

Enrollment Period

1.6 years

First QC Date

May 18, 2016

Last Update Submit

September 20, 2018

Conditions

Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)

Keywords

TTR-mediated AmyloidosisAmyloidosis, HereditaryAmyloid Neuropathies, FamilialFamilial Amyloid PolyneuropathiesAmyloid NeuropathiesAmyloidosis, Hereditary, Transthyretin-RelatedFamilial AmyloidosisRNAi therapeutic

Outcome Measures

Primary Outcomes (1)

  • Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs)

    Day 1 through to Day 314

Secondary Outcomes (11)

  • Profile of pharmacokinetics (PK) of ALN-TTRSC02

    Day 1 through to Day 3

  • Profile of pharmacokinetics (PK) of ALN-TTRSC02

    Day 1 through to Day 3

  • Profile of pharmacokinetics (PK) of ALN-TTRSC02

    Day 1 through to Day 3

  • Profile of pharmacokinetics (PK) of ALN-TTRSC02

    Day 1 through to Day 3

  • Profile of pharmacokinetics (PK) of ALN-TTRSC02

    Day 1 through to Day 3

  • +6 more secondary outcomes

Study Arms (2)

ALN-TTRSC02

ACTIVE COMPARATOR
Drug: ALN-TTRSC02

Sterile normal saline 0.9% for SC administration

PLACEBO COMPARATOR
Drug: Sterile Normal Saline (0.9% NaCl)

Interventions

ALN-TTRSC02DRUG

Ascending doses of ALN-TTRSC02 by subcutaneous (SC) injection

ALN-TTRSC02
Sterile Normal Saline (0.9% NaCl)DRUG

Calculated volume to match active comparator

Sterile normal saline 0.9% for SC administration

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male and female subjects, age 18 to 65 years, inclusive.
  • Body mass index (BMI) ≥18.0 kg/m2 and ≤30 kg/m2 assessed at Screening.
  • No clinically significant health concerns, as determined by medical history and physical examination, in the opinion of the Investigator.
  • Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception, willing and able to comply with the study requirements and to provide written informed consent.
  • For Japanese cohorts, subjects of Japanese descent are defined as people carrying a Japanese passport, descendants of 4 Japanese grandparents, and have not been outside Japan for more than 5 years.

You may not qualify if:

  • Clinically relevant history or presence of respiratory, gastrointestinal, renal, cardiovascular, hepatic, hematological, lymphatic, neurological, psychiatric, musculoskeletal, genitourinary, immunological, and other inflammatory diseases, or dermatological or connective tissue diseases or disorders.
  • Active serious mental illness or psychiatric disorder.
  • Clinically significant ECG abnormalities. Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant.
  • Known history of allergic reaction to an oligonucleotide or GalNAc.
  • History of intolerance to subcutaneous injection.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Clinical Trial Site

London, United Kingdom

Location

Related Publications (1)

  • Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ. Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. Clin Pharmacokinet. 2025 Dec 12. doi: 10.1007/s40262-025-01606-0. Online ahead of print.

    PMID: 41388232DERIVED

MeSH Terms

Conditions

Amyloidosis, FamilialAmyloid Neuropathies, FamilialAmyloid NeuropathiesAmyloidosis, Hereditary, Transthyretin-Related

Condition Hierarchy (Ancestors)

Metabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesAmyloidosisProteostasis DeficienciesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesPeripheral Nervous System DiseasesNeuromuscular Diseases

Study Officials

  • John Vest, MD, PhD

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 18, 2016

First Posted

June 14, 2016

Study Start

June 7, 2016

Primary Completion

January 12, 2018

Study Completion

January 12, 2018

Last Updated

September 21, 2018

Record last verified: 2018-09

Locations

GB(1)