- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03431896
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis (MED-hATTR)
April 3, 2024 updated by: The Cleveland Clinic
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
Study Overview
Status
Active, not recruiting
Detailed 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.
Study Type
Observational
Enrollment (Estimated)
30
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Mazen A Hanna, MD
- Phone Number: 216-444-3490
- Email: Hannam@ccf.org
Study Contact Backup
- Name: Lauren E Ives, RN
- Phone Number: 216-444-9066
- Email: ivesl@ccf.org
Study Locations
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Ohio
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Cleveland, Ohio, United States, 44195
- Cleveland Clinic
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Sampling Method
Non-Probability Sample
Study Population
Participants will be identified through clinical practice and from Amyloid support groups
Description
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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
---|
Primary
1.) To evaluate the relative amount of misfolded ATTR oligomers in asymptomatic ATTR amyloid genetic carriers and correlate their levels with clinical symptoms and outcomes.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Average % change in oligomers in patients with new onset TTR amyloid symptoms
Time Frame: Annually over 5 years
|
Change (%) for oligomer level at the time of TTR amyloid symptoms compared to baseline
|
Annually over 5 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
% change of oligomer levels relative to baseline level in patients with ATTR specific medication changes
Time Frame: Annually over 5 years
|
Change (%) for oligomer level at the time of ATTR specific medication changes compared to baseline
|
Annually over 5 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Mazen A Hanna, MD, The Cleveland Clinic
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
February 1, 2018
Primary Completion (Estimated)
September 30, 2026
Study Completion (Estimated)
December 31, 2026
Study Registration Dates
First Submitted
February 7, 2018
First Submitted That Met QC Criteria
February 7, 2018
First Posted (Actual)
February 13, 2018
Study Record Updates
Last Update Posted (Actual)
April 4, 2024
Last Update Submitted That Met QC Criteria
April 3, 2024
Last Verified
April 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Heart Diseases
- Cardiovascular Diseases
- Metabolic Diseases
- Nervous System Diseases
- Immune System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Lymphoproliferative Disorders
- Immunoproliferative Disorders
- Disease Attributes
- Genetic Diseases, Inborn
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Peripheral Nervous System Diseases
- Paraproteinemias
- Proteostasis Deficiencies
- Neoplasms, Plasma Cell
- Metabolism, Inborn Errors
- Heredodegenerative Disorders, Nervous System
- Amyloidosis, Familial
- Disease Progression
- Immunoglobulin Light-chain Amyloidosis
- Amyloidosis
- Cardiomyopathies
- Amyloid Neuropathies
- Amyloid Neuropathies, Familial
Other Study ID Numbers
- 17-1301
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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