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

ATTR Amyloidosis Clinical Trial

— ConTTRibute  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04561518
• Other study ID #: ALN-TTR02-013
• Status: Recruiting
• Start date: November 23, 2020
• Est. completion date: September 2030

Study information

• Verified date: April 2024
• Source: Alnylam Pharmaceuticals
• Contact: Alnylam Clinical Trial Information Line
• Phone: 1-877-ALNYLAM
• Email: clinicaltrials[@]alnylam.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1500
• Est. completion date: September 2030
• Est. primary completion date: September 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Amyloidosis
• ATTR Amyloidosis
• Transthyretin-Mediated Amyloidosis

Locations

Country	Name	City	State
Brazil	Clinical Trial Site	Salvador
Denmark	Clinical Trial Site	Arhus
Denmark	Clinical Trial Site	Copenhagen
France	Clinical Trial Site	Bordeaux
France	Clinical Trial Site	Bron
France	Clinical Trial Site	Le Kremlin-Bicêtre
France	Clinical Trial Site	Marseille
Germany	Clinical Trial Site	Hanover
Israel	Clinical Trial Site	Jerusalem
Israel	Clinical Trial Site	Ramat Gan
Italy	Clinical Trial Site	Milan
Italy	Clinical Trial Site	Naples
Italy	Clinical Trial Site	Palermo
Italy	Clinical Trial Site	Roma
Netherlands	Clinical Trial Site	Groningen
Portugal	Clinical Trial Site	Lisbon
Portugal	Clinical Trial Site	Porto
Spain	Clinical Trial Site	Barcelona
Spain	Clinical Trial Site	Madrid
Spain	Clinical Trial Site	Palma
Taiwan	Clinical Trial Site	New Taipei City
Taiwan	Clinical Trial Site	Taipei
United States	Clinical Trial Site	Austin	Texas
United States	Clinical Trial Site	Baltimore	Maryland
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Columbus	Ohio
United States	Clinical Trial Site	Durham	North Carolina
United States	Clinical Trial Site	Houston	Texas
United States	Clinical Trial Site	Iowa City	Iowa
United States	Clinical Trial Site	Jacksonville	Florida
United States	Clinical Trial Site	Kansas City	Kansas
United States	Clinical Trial Site	La Jolla	California
United States	Clinical Trial Site	Los Angeles	California
United States	Clinical Trial Site	New York	New York
United States	Clinical Trial Site	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

United States,  Brazil,  Denmark,  France,  Germany,  Israel,  Italy,  Netherlands,  Portugal,  Spain,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Adverse Events		From time of enrollment for up to 10 years
Primary	Selected Events of Interest in Patients with Hereditary Transthyretin-mediated (hATTR) Amyloidosis (ATTRv Amyloidosis)	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
Primary	Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability Score	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
Primary	HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) Score	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
Primary	HCP-Assessed Neuropathy Impairment Score (NIS)	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
Primary	HCP-Assessed Cardiomyopathy	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
Primary	HCP- Assessed Cardiopulmonary Exercise Testing (CPET) Performance		Up to 11 years
Primary	Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score	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
Primary	Kansas City Cardiomyopathy Questionnaire (KCCQ)	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
Primary	Rasch-built Overall Disability Scale (R-ODS)	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