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Trial record 1 of 1 for:    NCT04561518
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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

Tracking Information
First Submitted Date September 18, 2020
First Posted Date September 23, 2020
Last Update Posted Date September 16, 2021
Actual Study Start Date November 23, 2020
Estimated Primary Completion Date September 2030   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: March 5, 2021)
  • Incidence of Adverse Events [ Time Frame: From time of enrollment for up to 10 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 in patients diagnosed with ATTRv amyloidosis.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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).
  • 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).
  • 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.
Original Primary Outcome Measures
 (submitted: September 18, 2020)
  • Incidence of Adverse Events [ Time Frame: From time of enrollment for up to 10 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 in patients diagnosed with ATTRv amyloidosis.
  • Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability Score [ Time Frame: 1 year prior to enrollment and at routine clinical care visits for up to 10 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.
  • HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) Score [ Time Frame: 1 year prior to enrollment and at routine clinical care visits for up to 10 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.
  • HCP-Assessed Neuropathy Impairment Score of the Lower Limb (NIS-LL) Score [ Time Frame: 1 year prior to enrollment and at routine clinical care visits for up to 10 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.
  • HCP-Assessed Cardiomyopathy [ Time Frame: 1 year prior to enrollment and at routine clinical care visits for up to 10 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.
  • Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score [ Time Frame: 1 year prior to enrollment and annually at routine clinical care visits for up to 10 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).
  • Kansas City Cardiomyopathy Questionnaire (KCCQ) [ Time Frame: 1 year prior to enrollment and annually at routine clinical care visits for up to 10 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).
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title 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)
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
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition
  • Transthyretin-Mediated Amyloidosis
  • ATTR Amyloidosis
Intervention Not Provided
Study Groups/Cohorts
  • 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.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: September 18, 2020)
1500
Original Estimated Enrollment Same as current
Estimated Study Completion Date September 2030
Estimated Primary Completion Date September 2030   (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
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
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04561518
Other Study ID Numbers ALN-TTR02-013
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Alnylam Pharmaceuticals
Study Sponsor Alnylam Pharmaceuticals
Collaborators Not Provided
Investigators
Study Director: Medical Director Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date September 2021