Recruiting

Observational Study

Sponsor:

Alnylam Pharmaceuticals

Code:

NCT04561518

Conditions

Transthyretin-Mediated Amyloidosis

ATTR Amyloidosis

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

The purpose of this study is to:

  • Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients
  • Characterize the safety and effectiveness of patisiran and vutrisiran as part of routine clinical practice in the real-world clinical setting
  • Describe disease emergence/progression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) variant

Conditions

Transthyretin-Mediated Amyloidosis

ATTR Amyloidosis

Study ID

NCT04561518

Start date

Nov 23, 2020

Status verified date

Sep, 2026

Completion date

Dec 31, 2033

Anticipated

Primary completion date

Dec 31, 2033

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Diagnosis of ATTR amyloidosis or documented known disease-causing TTR variant for the cohort of pre-symptomatic carriers
  • Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis

Exclusion Criteria:

  • Current enrollment in a clinical trial for any investigational agent

Study Design

Enrollment

1500 participants

Anticipated

Interventions and Outcome Measures

Arms

Patients with ATTR amyloidosis

Patients with a diagnosis of ATTR amyloidosis, hereditary or wild type, will be eligible for the study and will follow routine clinical care.

Pre-symptomatic Carriers

Pre-symptomatic carriers with a known disease-causing TTR variant will be eligible for the study and will follow routine clinical care.

Primary outcome measure

  • Incidence of Adverse Events [ Time Frame: From time of enrollment for up to 10 years ]
  • Selected Events of Interest in Patients with Hereditary Transthyretin-mediated (hATTR) or Wild-type Transthyretin-mediated (wtATTR) Amyloidosis (ATTRv Amyloidosis) [ Time Frame: From 1 year prior to enrollment for up to 10 years ]
  • Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability Score [ Time Frame: Up to 11 years ]
  • HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) Score [ Time Frame: Up to 11 years ]
  • HCP-Assessed Neuropathy Impairment Score (NIS) [ Time Frame: Up to 11 years ]
  • HCP-Assessed Cardiomyopathy [ Time Frame: Up to 11 years ]
  • HCP- Assessed Cardiopulmonary Exercise Testing (CPET) Performance [ Time Frame: Up to 11 years ]
  • Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score [ Time Frame: Up to 11 years ]
  • Kansas City Cardiomyopathy Questionnaire (KCCQ) [ Time Frame: Up to 11 years ]
  • Rasch-built Overall Disability Scale (R-ODS) [ Time Frame: Up to 11 years ]

Central Contacts and Locations

Central contacts

Alnylam Clinical Trial Information Line

1-877-ALNYLAMclinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

1-877-256-9526clinicaltrials@alnylam.com

Locations

Clinical Trial Site

Recruiting

La Jolla, California, United States, 92037

Clinical Trial Site

Recruiting

Los Angeles, California, United States, 90095

Clinical Trial Site

Recruiting

Jacksonville, Florida, United States, 32224

Clinical Trial Site

Recruiting

Iowa City, Iowa, United States, 52242

Clinical Trial Site

Recruiting

Kansas City, Kansas, United States, 66160

Clinical Trial Site

Recruiting

Baltimore, Maryland, United States, 21224

Clinical Trial Site

Recruiting

New York, New York, United States, 10034

Clinical Trial Site

Recruiting

Durham, North Carolina, United States, 27710

Clinical Trial Site

Recruiting

Columbus, Ohio, United States, 43210

Clinical Trial Site

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Clinical Trial Site

Recruiting

Austin, Texas, United States, 78756

Clinical Trial Site

Recruiting

Houston, Texas, United States, 77030

More Information

Sponsor

Alnylam Pharmaceuticals

Last update posted

Sep 8, 2026

Last verified

Sep, 2026

Keywords

  • RNAi therapeutic
  • Transthyretin
  • TTR
  • Amyloidosis
  • Hereditary Transthyretin-mediated (hATTR) Amyloidosis
  • hATTR amyloidosis
  • Hereditary ATTR amyloidosis
  • Wild-type amyloidosis
  • wtATTR amyloidosis
  • ATTRv amyloidosis
  • ATTRwt amyloidosis
  • Polyneuropathy
  • Familial amyloid polyneuropathies
  • ATTR
  • Transthyretin amyloidosis
  • TTR-mediated amyloidosis
  • Polyneuropathies
  • Amyloid neuropathies
  • Amyloid neuropathies, familial
  • Amyloidosis, familial

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-10. This information was provided to ClinicalTrials.gov by Alnylam Pharmaceuticals on 2026-09-08. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.