Clinical trial NCT04561518: ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
Official title: ConTTRibute: A Global Observational Multicenter Long-Term Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Transthyretin-Mediated Amyloidosis
ATTR Amyloidosis
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
Lead sponsor: Alnylam Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2020-09-23
Enrollment: 1500
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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


Eligibility criteria: 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

Primary outcomes:
measure: Incidence of Adverse Events
timeFrame: From time of enrollment for up to 10 years
measure: Selected Events of Interest in Patients with Hereditary Transthyretin-mediated (hATTR) Amyloidosis (ATTRv Amyloidosis)
timeFrame: From 1 year prior to enrollment for up to 10 years
measure: Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability Score
timeFrame: Up to 11 years
measure: HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) Score
timeFrame: Up to 11 years
measure: HCP-Assessed Neuropathy Impairment Score (NIS)
timeFrame: Up to 11 years
measure: HCP-Assessed Cardiomyopathy
timeFrame: Up to 11 years
measure: HCP- Assessed Cardiopulmonary Exercise Testing (CPET) Performance
timeFrame: Up to 11 years
measure: Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score
timeFrame: Up to 11 years
measure: Kansas City Cardiomyopathy Questionnaire (KCCQ)
timeFrame: Up to 11 years
measure: Rasch-built Overall Disability Scale (R-ODS)
timeFrame: Up to 11 years
Secondary outcomes:
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: Clinical Trial Site
state: California
city: Los Angeles
country: United States
facility: Clinical Trial Site
state: California
city: Jacksonville
country: United States
facility: Clinical Trial Site
state: Florida
city: Iowa City
country: United States
facility: Clinical Trial Site
state: Iowa
city: Kansas City
country: United States
facility: Clinical Trial Site
state: Kansas
city: Baltimore
country: United States
facility: Clinical Trial Site
state: Maryland
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: New York
country: United States
facility: Clinical Trial Site
state: New York
city: Durham
country: United States
facility: Clinical Trial Site
state: North Carolina
city: Columbus
country: United States
facility: Clinical Trial Site
state: Ohio
city: Philadelphia
country: United States
facility: Clinical Trial Site
state: Pennsylvania
city: Austin
country: United States
facility: Clinical Trial Site
state: Texas
city: Houston
country: United States
facility: Clinical Trial Site
state: Texas
city: Salvador
country: Brazil
facility: Clinical Trial Site
city: Sao Paulo
country: Brazil
facility: Clinical Trial Site
city: Sofia
country: Bulgaria
facility: Clinical Trial Site
city: Arhus
country: Denmark
facility: Clinical Trial Site
city: Copenhagen
country: Denmark
facility: Clinical Trial Site
city: Bordeaux
country: France
facility: Clinical Trial Site
city: Bron
country: France
facility: Clinical Trial Site
city: Créteil
country: France
facility: Clinical Trial Site
city: Le Kremlin-Bicêtre
country: France
facility: Clinical Trial Site
city: Marseille
country: France
facility: Clinical Trial Site
city: Berlin
country: Germany
facility: Clinical Trial Site
city: Giessen
country: Germany
facility: Clinical Trial Site
city: Hanover
country: Germany
facility: Clinical Trial Site
city: Jerusalem
country: Israel
facility: Clinical Trial Site
city: Ramat Gan
country: Israel
facility: Clinical Trial Site
city: Milan
country: Italy
facility: Clinical Trial Site
city: Naples
country: Italy
facility: Clinical Trial Site
city: Palermo
country: Italy
facility: Clinical Trial Site
city: Roma
country: Italy
facility: Clinical Trial Site
city: Groningen
country: Netherlands
facility: Clinical Trial Site
city: Lisbon
country: Portugal
facility: Clinical Trial Site
city: Porto
country: Portugal
facility: Clinical Trial Site
city: Barcelona
country: Spain
facility: Clinical Trial Site
city: Madrid
country: Spain
facility: Clinical Trial Site
city: Palma
country: Spain
facility: Clinical Trial Site
city: New Taipei City
country: Taiwan
facility: Clinical Trial Site
city: Taipei
country: Taiwan
facility: Clinical Trial Site
Study officials:
affiliation: Alnylam Pharmaceuticals
name: Medical Director
role: STUDY_DIRECTOR
References: