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NCT02797847

A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects

Completed Phase 1 Last updated 21 September 2018
What this trial tests

Phase 1 trial testing ALN-TTRSC02 in Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) in 80 participants. Completed in 12 January 2018.

Timeline
7 June 2016
Primary endpoint
12 January 2018
12 January 2018

Quick facts

Lead sponsorAlnylam Pharmaceuticals
PhasePhase 1
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingsingle
Primary purposetreatment
Enrollment80
Start date7 June 2016
Primary completion12 January 2018
Estimated completion12 January 2018
Sites1 location across United Kingdom

Drugs / interventions tested

Conditions studied

Sponsor

Alnylam Pharmaceuticals — full company profile →

Who can join

Adults 18 to 65, any sex, with Transthyretin-mediated Amyloidosis (ATTR Amyloidosis). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Therapeutic siRNA: state of the art.
    Hu B, Zhong L, Weng Y, Peng L, et al · · 2020 · cited 991× · PMID 32561705 · DOI 10.1038/s41392-020-0207-x
  2. Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics.
    Huang Y. · · 2017 · cited 205× · PMID 28325278 · DOI 10.1016/j.omtn.2016.12.003
  3. Bioconjugated Oligonucleotides: Recent Developments and Therapeutic Applications.
    Benizri S, Gissot A, Martin A, Vialet B, et al · · 2019 · cited 161× · PMID 30608140 · DOI 10.1021/acs.bioconjchem.8b00761
  4. Pathophysiology and Therapeutic Approaches to Cardiac Amyloidosis.
    Griffin JM, Rosenblum H, Maurer MS. · · 2021 · cited 130× · PMID 33983835 · DOI 10.1161/circresaha.121.318187
  5. Improved Nucleic Acid Therapy with Advanced Nanoscale Biotechnology.
    Weng Y, Huang Q, Li C, Yang Y, et al · · 2020 · cited 72× · PMID 31927331 · DOI 10.1016/j.omtn.2019.12.004
  6. Inhaled siRNA Formulations for Respiratory Diseases: From Basic Research to Clinical Application.
    Fan Y, Yang Z. · · 2022 · cited 10× · PMID 35745766 · DOI 10.3390/pharmaceutics14061193
  7. RNA Interference Therapeutics for Hereditary Amyloidosis: A Narrative Review of Clinical Trial Outcomes and Future Directions.
    Dave P, Anand P, Kothawala A, Srikaram P, et al · · 2024 · cited 5× · PMID 39044869 · DOI 10.7759/cureus.62981
  8. C16-siRNAs in Focus: Development of ALN-APP, a Promising RNAi-Based Therapeutic for Alzheimer's Disease.
    Titze-de-Almeida R, Oliveira Gomes GM, Santos TCD, Titze-de-Almeida SS. · · 2025 · PMID 41599628 · DOI 10.3390/ph19010026

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