Last reviewed · How we verify
NCT02797847
A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
Phase 1 trial testing ALN-TTRSC02 in Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) in 80 participants. Completed in 12 January 2018.
12 January 2018
Quick facts
| Lead sponsor | Alnylam Pharmaceuticals |
|---|---|
| Phase | Phase 1 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | single |
| Primary purpose | treatment |
| Enrollment | 80 |
| Start date | 7 June 2016 |
| Primary completion | 12 January 2018 |
| Estimated completion | 12 January 2018 |
| Sites | 1 location across United Kingdom |
Drugs / interventions tested
- ALN-TTRSC02 — full drug profile →
- Sterile Normal Saline (0.9% NaCl) — full drug profile →
Conditions studied
- Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) — all drugs for Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) →
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):
-
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 -
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 -
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 -
Pathophysiology and Therapeutic Approaches to Cardiac Amyloidosis.
Griffin JM, Rosenblum H, Maurer MS. · · 2021 · cited 130× · PMID 33983835 · DOI 10.1161/circresaha.121.318187 -
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 -
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 -
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 -
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
Verify or expand the search:
- PubMed search for NCT02797847
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other Alnylam Pharmaceuticals trials
Trials by the same sponsor.
- NCT07535606 — A Study to Evaluate ALN-4915 in Adult Healthy Volunteers · Phase 1 · recruiting
- NCT07463846 — A Study to Evaluate ALN-2232 in Participants With Obesity · Phase 1, PHASE2 · recruiting
- NCT07223203 — TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidos · Phase 3 · recruiting
- NCT07465224 — A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus · Phase 2 · recruiting
- NCT07358078 — DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT02797847 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Alnylam Pharmaceuticals
- Last refreshed: 21 September 2018
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02797847.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing