ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) (ConTTRibute)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04561518|
Recruitment Status : Recruiting
First Posted : September 23, 2020
Last Update Posted : June 16, 2021
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|
|Study Type :||Observational|
|Estimated Enrollment :||1500 participants|
|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|
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 with a known disease-causing TTR mutation will be eligible for the study and will follow routine clinical care.
- 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.
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
|Contact: Alnylam Clinical Trial Information Line||1-877-ALNYLAMfirstname.lastname@example.org|
|Contact: Alnylam Clinical Trial Information Lineemail@example.com|
|United States, Texas|
|Clinical Trial Site||Recruiting|
|Austin, Texas, United States, 78756|
|Study Director:||Medical Director||Alnylam Pharmaceuticals|