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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03431896
Other study ID # 17-1301
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
Start date February 1, 2018
Est. completion date December 31, 2026

Study information

Verified date April 2024
Source The Cleveland Clinic
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.


Description:

Recent advances in genetic testing have allowed for pathogenic mutation identification in family members of affected individuals prior to onset of symptoms. While the presence of mutation and the corresponding TTR kinetic stability have been directly linked to disease development, the molecular drivers of tissue specific degeneration have not been defined. We hypothesize that soluble misfolded TTR oligomer species may be circulating within the blood of these patients possibly years prior to amyloid deposition and could serve as an early biomarker and/or driver for disease development. In this line, The Scripps Research Institute has developed a peptide-based probe that specifically labels and integrates into misfolded TTR oligomers allowing the relative circulating concentration in the bloodstream to be determined. Longitudinal monitoring of untreated, asymptomatic TTR amyloid genetic carriers utilizing the Scripps probe is likely to provide novel insight into early disease progression. We also plan to utilize the Scripps probe to monitor disease progression in TTR amyloid genetic carriers currently undergoing treatment by observing how treatments affect the circulating misfolded TTR oligomers. Through enhanced understanding of early disease progression and treatment efficacy, our hope is to limit amyloid accumulation in cardiac and nerve tissue and delay the development of the invariably fatal TTR amyloid cardiomyopathy/neuropathy.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 30
Est. completion date December 31, 2026
Est. primary completion date September 30, 2026
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility Inclusion Criteria: - Patients with known hereditary ATTR amyloidosis genetic mutations as identified by genetic testing. Exclusion Criteria: - Patients with ATTR amyloidosis identified as wild-type.

Study Design


Locations

Country Name City State
United States Cleveland Clinic Cleveland Ohio

Sponsors (1)

Lead Sponsor Collaborator
The Cleveland Clinic

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Average % change in oligomers in patients with new onset TTR amyloid symptoms Change (%) for oligomer level at the time of TTR amyloid symptoms compared to baseline Annually over 5 years
Secondary % change of oligomer levels relative to baseline level in patients with ATTR specific medication changes Change (%) for oligomer level at the time of ATTR specific medication changes compared to baseline Annually over 5 years
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