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

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ClinicalTrials.gov Identifier: NCT04561518
Recruitment Status : Recruiting
First Posted : September 23, 2020
Last Update Posted : September 16, 2021
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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 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) mutation

Condition or disease
Transthyretin-Mediated Amyloidosis ATTR Amyloidosis

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Study Type : Observational
Estimated Enrollment : 1500 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: ConTTRibute: A Global Observational Multicenter Long-Term Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
Actual Study Start Date : November 23, 2020
Estimated Primary Completion Date : September 2030
Estimated Study Completion Date : September 2030

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis

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 mutation will be eligible for the study and will follow routine clinical care.



Primary Outcome Measures :
  1. Incidence of Adverse Events [ Time Frame: From time of enrollment for up to 10 years ]
  2. 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 in patients diagnosed with ATTRv amyloidosis.

  3. 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.

  4. 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.

  5. HCP-Assessed Neuropathy Impairment Score of the Lower Limb (NIS-LL) Score [ Time Frame: Up to 11 years ]
    The NIS-LL assesses muscle weakness, reflexes and sensation, scored separately for the left and right limbs. Components of muscle weakness (hip and knee flexion, hip and knee extension, ankle dorsiflexors, ankle plantar flexors, toe extensors, toe flexors) are scored on 0 to 4 scale (0=normal, 4=paralysis). Components of reflexes (quadriceps femoris, triceps surae) and sensation (touch pressure, pin-prick, vibration, joint position) are scored 0=normal, 1=decreased, or 2=absent. Total possible NIS-LL score ranges 0-88 with higher score=greater impairment.

  6. 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.

  7. 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).

  8. 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).

  9. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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 mutation will be eligible for the study.
Criteria

Inclusion Criteria:

  • Diagnosis of ATTR amyloidosis or documented known disease-causing TTR mutation 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04561518


Contacts
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Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526 clinicaltrials@alnylam.com

Locations
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United States, Iowa
Clinical Trial Site Recruiting
Iowa City, Iowa, United States, 52242
United States, Maryland
Clinical Trial Site Recruiting
Baltimore, Maryland, United States, 21224
United States, Ohio
Clinical Trial Site Recruiting
Columbus, Ohio, United States, 43210
United States, Texas
Clinical Trial Site Recruiting
Austin, Texas, United States, 78756
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04561518    
Other Study ID Numbers: ALN-TTR02-013
First Posted: September 23, 2020    Key Record Dates
Last Update Posted: September 16, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alnylam Pharmaceuticals:
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
Additional relevant MeSH terms:
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Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases