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

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

• Ages Any age
• All sexes
• No healthy volunteers
• Child, Adult, Older adult

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.

Trial locations and contacts

40 sites in 12 countries. Contact the study team or a site near you to ask about taking part.

About this study

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

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

Medical subject headings

Amyloidosis, Amyloid Neuropathies, Familial, Polyneuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Amyloid Neuropathies, Amyloidosis, Familial

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

What is being measured

The primary outcome is the main question the study is designed to answer.

1. Incidence of Adverse Events

   Measured: From time of enrollment for up to 10 years

2. Selected Events of Interest in Patients with Hereditary Transthyretin-mediated (hATTR) or Wild-type Transthyretin-mediated (wtATTR) 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 or wtATTR amyloidosis.

   Measured: From 1 year prior to enrollment for up to 10 years

3. 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.

   Measured: Up to 11 years

4. 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.

   Measured: Up to 11 years

5. 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.

   Measured: Up to 11 years

6. 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.

   Measured: Up to 11 years

7. HCP- Assessed Cardiopulmonary Exercise Testing (CPET) Performance

   Measured: Up to 11 years

8. 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).

   Measured: Up to 11 years

9. 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).

   Measured: Up to 11 years

10. 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.

    Measured: Up to 11 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1. First posted23 Sep 2020
2. Study start23 Nov 2020
3. Primary completion31 Dec 2033 (estimated)
4. Study completion31 Dec 2033 (estimated)

Resources

• Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
• Other trials for Transthyretin-Mediated AmyloidosisStudies of the same condition, open and under way.
• More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
• What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.