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

April 17, 2024 updated by: Alnylam Pharmaceuticals

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

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

Study Overview

Status

Recruiting

Conditions

Study Type

Observational

Enrollment (Estimated)

1500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Brazil
      • Salvador, Brazil
        • Recruiting
        • Clinical Trial Site
  • Denmark
      • Arhus, Denmark
        • Recruiting
        • Clinical Trial Site
      • Copenhagen, Denmark
        • Recruiting
        • Clinical Trial Site
  • France
      • Bordeaux, France
        • Recruiting
        • Clinical Trial Site
      • Bron, France
        • Recruiting
        • Clinical Trial Site
      • Le Kremlin-Bicêtre, France
        • Recruiting
        • Clinical Trial Site
      • Marseille, France
        • Recruiting
        • Clinical Trial Site
  • Germany
      • Hanover, Germany
        • Recruiting
        • Clinical Trial Site
  • Israel
      • Jerusalem, Israel
        • Recruiting
        • Clinical Trial Site
      • Ramat Gan, Israel
        • Recruiting
        • Clinical Trial Site
  • Italy
      • Milan, Italy
        • Recruiting
        • Clinical Trial Site
      • Naples, Italy
        • Recruiting
        • Clinical Trial Site
      • Palermo, Italy
        • Recruiting
        • Clinical Trial Site
      • Roma, Italy
        • Recruiting
        • Clinical Trial Site
  • Netherlands
      • Groningen, Netherlands
        • Recruiting
        • Clinical Trial Site
  • Portugal
      • Lisbon, Portugal
        • Recruiting
        • Clinical Trial Site
      • Porto, Portugal
        • Recruiting
        • Clinical Trial Site
  • Spain
      • Barcelona, Spain
        • Recruiting
        • Clinical Trial Site
      • Madrid, Spain
        • Not yet recruiting
        • Clinical Trial Site
      • Palma, Spain
        • Recruiting
        • Clinical Trial Site
  • Taiwan
      • New Taipei City, Taiwan
        • Recruiting
        • Clinical Trial Site
      • Taipei, Taiwan
        • Recruiting
        • Clinical Trial Site
  • United States
    • California
      • La Jolla, California, United States, 92037
        • Recruiting
        • Clinical Trial Site
      • Los Angeles, California, United States, 90095
        • Recruiting
        • Clinical Trial Site
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Recruiting
        • Clinical Trial Site
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • Clinical Trial Site
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • Clinical Trial Site
    • Maryland
      • Baltimore, Maryland, United States, 21224
        • Recruiting
        • Clinical Trial Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02127
        • Recruiting
        • Clinical Trial Site
    • New York
      • New York, New York, United States, 10034
        • Recruiting
        • Clinical Trial Site
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Clinical Trial Site
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Recruiting
        • Clinical Trial Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Clinical Trial Site
    • Texas
      • Austin, Texas, United States, 78756
        • Recruiting
        • Clinical Trial Site
      • Houston, Texas, United States, 77030
        • Recruiting
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients with a diagnosis of ATTR amyloidosis, hereditary or wild type, and pre-symptomatic carriers with a known disease-causing TTR variant will be eligible for the study.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of Adverse Events
Time Frame: From time of enrollment for up to 10 years
From time of enrollment for up to 10 years
Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability Score
Time Frame: Up to 11 years
PND Scores: Stage 0=No symptoms; Stage I=Sensory disturbances but preserved walking capabilities; Stage II=Impaired walking capacity, but ability to walk without a stick or crutches; Stage IIIA=Walking with help of 1 stick or crutch; Stage IIIB=Walking with the help of 2 sticks or crutches; Stage IV=confined to wheel chair or bedridden.
Up to 11 years
HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) Score
Time Frame: Up to 11 years
FAP Scores: Stage 0=No symptoms; Stage I=Unimpaired ambulation; mostly mild sensory, motor and autonomic neuropathy in the lower limbs; Stage II=Assistance with ambulation required, mostly moderate impairment progression to the lower limbs, upper limbs, and trunk; Stage III=Wheelchair-bound or bedridden; severe sensory, motor, and autonomic involvement of all limbs.
Up to 11 years
HCP-Assessed Cardiomyopathy
Time Frame: Up to 11 years
Cardiomyopathy will be assessed using New York Heart Association (NYHA) Class: I=No symptoms; II=Symptoms with ordinary physical activity; III=Symptoms with less than ordinary physical activity; IV=Symptoms at rest.
Up to 11 years
Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score
Time Frame: Up to 11 years
Norfolk-QoL-DN: The Norfolk QOL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that assesses 6 domains: physical function, large-fiber neuropathy, activities of daily living, symptoms, small-fiber neuropathy, and autonomic neuropathy. The total score ranges from -4 points (best possible quality of life) to 136 points (worst possible quality of life).
Up to 11 years
Kansas City Cardiomyopathy Questionnaire (KCCQ)
Time Frame: Up to 11 years
The KCCQ is a 23-item self-administered questionnaire developed to independently measure the patient's perception of health status, which includes heart failure symptoms, impact on physical and social function, and how their heart failure impacts their quality of life within a 2-week recall period. The KCCQ quantifies 6 domains (symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary [OS] scores).
Up to 11 years
Rasch-built Overall Disability Scale (R-ODS)
Time Frame: Up to 11 years
The R-ODS is a 24-item self-administered questionnaire for assessment of the disability a patient experiences. It uses a linearly weighted categorical rating scale that specifically captures domains of activity and social participation limitations in patients.
Up to 11 years
Selected Events of Interest in Patients with Hereditary Transthyretin-mediated (hATTR) Amyloidosis (ATTRv Amyloidosis)
Time Frame: From 1 year prior to enrollment for up to 10 years
Selected events of interest are defined as hepatic events, cardiovascular events, renal events, ocular events and infusion-related reactions, hypersensitivity, and other events in patients diagnosed with hATTR amyloidosis.
From 1 year prior to enrollment for up to 10 years
HCP-Assessed Neuropathy Impairment Score (NIS)
Time Frame: Up to 11 years
NIS : 74 items, assess muscle weakness, reflexes and sensation; scored separately for left, right limbs (37 items for each side). Components of muscle weakness (hip and knee flexion, hip and knee extension, ankle dorsiflexors, ankle plantar flexors, toe extensors, toe flexors) scored on scale 0 (normal) to 4 (paralysis), higher score=greater weakness. Components of reflexes (quadriceps femoris, triceps surae) and sensation (touch pressure, pin-prick, vibration, joint position) scored 0 = normal, 1= decreased, or 2 = absent. Total possible NIS score range 0-244, higher score=greater impairment.
Up to 11 years
HCP- Assessed Cardiopulmonary Exercise Testing (CPET) Performance
Time Frame: Up to 11 years
Up to 11 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Medical Director, Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 23, 2020

Primary Completion (Estimated)

September 1, 2030

Study Completion (Estimated)

September 1, 2030

Study Registration Dates

First Submitted

September 18, 2020

First Submitted That Met QC Criteria

September 18, 2020

First Posted (Actual)

September 23, 2020

Study Record Updates

Last Update Posted (Actual)

April 19, 2024

Last Update Submitted That Met QC Criteria

April 17, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTR02-013

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Transthyretin-Mediated Amyloidosis

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Lebanon

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe