PATHWAY-RCT: Preventing Admission To Hospital With Attr cardiomyopathY

NCT05098665 | Recruiting

• 1 other identifier: C21026
• Study Type: interventional
• Target: 320
• Locations: 1 country
• Sites: 1

Brief Summary

Cardiac amyloidosis is a condition where the heart muscle, amongst other tissues, is infiltrated by the abnormal build-up of proteins called amyloid. This stiffens and thickens the heart muscle over time which makes it less efficient and puts further stress and strain on the other chambers of the heart, leading to heart failure. The commonest form, that affects predominantly the elderly, is called 'wild-type' ATTR amyloid (TTR is the protein that accumulates). In this condition a patient has a 60% chance of admission to hospital each year after diagnosis. There is no current treatment for ATTR amyloid other than using water tablets to reduce excess fluid and prevent more serious fluid build up in lungs and other tissues. Increasing body weight is the most reliable clinical sign of this fluid build up. Tele-monitoring is the practice of monitoring patients from a distance and has been shown to reduce heart failure admissions and death in patients with heart failure from any cause. Due to reduced access to primary and secondary care during COVID-19 the investigators instigated tele-monitoring of heart failure in ATTR amyloid patients. This appeared to be an effective intervention in the pilot study. The investigators propose to monitor the weight of patients with cardiac amyloidosis at home and intervene where a build up of fluid is observed by telephone discussion with a doctor. The investigators propose to evidence this in a prospective clinical trial. The investigators will evaluate the effect fairly by comparing tele-monitoring with usual care.

Conditions

Cardiac Amyloidosis

Outcome Measures

Primary Outcomes (2)

• Heart-failure related hospitalisations

  Hospitalisation which can be attributed to heart failure

  Study duration (3 years)

• All-cause mortality

  Death by any cause, as an annual event rate

  Study duration (3 years)

Secondary Outcomes (1)

• All-cause hospitalisation

  Study duration (3 years)

Study Arms (2)

Arm A - RCT telemonitoring

ACTIVE COMPARATOR

160 ATTR-CM patients assigned to receive telemonitoring intervention

Combination Product: Telemonitoring service

Arm B - RCT usual care

NO INTERVENTION

Control group of 160 ATTR-CM patients assigned to receive usual care

Interventions

Telemonitoring service COMBINATION_PRODUCT

Patients in the active arm will receive a cellular network connected set of digital scales, and instructed to take their weight each morning at the same time. This device (BodyTrace) automatically uploads each daily weight reading to a central database. A clinical algorithm is applied to detect those at risk of acute and subacute decompensated heart failure. Patients are contacted within 24-48 hours of being flagged as at risk by a heart failure specialist. A clinical history is taken, and medication review undertaken, in line with a protocol. Diuretics are adjusted as per the protocolised changes, or for patients at ceiling of treatment, referral for local specialist review is made. A third protocol for follow-up is then followed to close the loop of intervention.

Arm A - RCT telemonitoring

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults aged ≥18 at the date of signing informed consent which is defined as the beginning of the Screening Period.
• An established diagnosis of ATTR cardiomyopathy as defined by protocol.
• Ability to provide written, personally signed and dated informed consent to participate in the study, in accordance with the ICH Good Clinical Practice (GCP) Guideline E6 (R2) (2016) and applicable regulations, before completing any study-related procedures.
• An understanding, ability, and willingness to fully comply with study procedures and restrictions.
• Currently a patient at a study site (NAC).

You may not qualify if:

• An inability to have or use BodyTrace device scales at usual residence (for example no mobile network cellular signal)
• On dialysis or end-stage renal failure (eGFR \<25mL/min)
• Serum albumin \<20g/dL or other non-hypervolaemia cause of tissue oedema (e.g. protein-losing enteropathy, nephrotic syndrome)
• Use of greater than 2 oral diuretics (e.g. on maximum oral diuretic therapy)

Sponsors & Collaborators

• Richmond Research Institute: lead
• Richmond Pharmacology Limited: collaborator
• National Amyloidosis Centre: collaborator

Study Sites (1)

Richmond Research Institute

London, SW17 0RE, United Kingdom

RECRUITING

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies

Study Officials

• Tamer Rezk, MBBS MRCP PhD

  National Amyloidosis Centre, Royal Free Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

James Rickard, MPharm

CONTACT

+44 02070425800; j.rickard@richmondresearchinstitute.org

Keith Berelowitz

CONTACT

02070425800; k.berelowitz@richmondpharmacology.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Masking Details: It is not possible to mask this type of intervention for participant or care provider, but outcomes will be assessed in a blinded fashion.
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomised Controlled Trial of telemonitoring intervention versus usual care

Study Record Dates

• First Submitted: October 15, 2021
• First Posted: October 28, 2021
• Study Start: January 1, 2025
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: October 3, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)