RecruitingNot Applicable

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

United States, Brazil, Bulgaria1,500 participantsStarted 2020-11-23

Plain-language 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

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Incidence of Adverse Events

Timeframe: 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)

Timeframe: From 1 year prior to enrollment for up to 10 years

Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability Score

Timeframe: Up to 11 years

HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) Score

Timeframe: Up to 11 years

HCP-Assessed Neuropathy Impairment Score (NIS)

Timeframe: Up to 11 years

HCP-Assessed Cardiomyopathy

Timeframe: Up to 11 years

HCP- Assessed Cardiopulmonary Exercise Testing (CPET) Performance

Timeframe: Up to 11 years

Trial details

NCT IDNCT04561518

SponsorAlnylam Pharmaceuticals

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2033-12-31

Contact for this trial

Alnylam Clinical Trial Information Line

1-877-ALNYLAM clinicaltrials@alnylam.com

View on ClinicalTrials.gov More Transthyretin-Mediated Amyloidosis trials

Plain-language summary

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Incidence of Adverse Events

Timeframe: 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)

Timeframe: From 1 year prior to enrollment for up to 10 years

Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability Score

Timeframe: Up to 11 years

HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) Score

Timeframe: Up to 11 years

HCP-Assessed Neuropathy Impairment Score (NIS)

Timeframe: Up to 11 years

HCP-Assessed Cardiomyopathy

Timeframe: Up to 11 years

HCP- Assessed Cardiopulmonary Exercise Testing (CPET) Performance

Timeframe: Up to 11 years