Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
Monitoring Early Hereditary ATTR Amyloidosis Progression
Plain English Summary
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis is a Not Applicable clinical trial sponsored by The Cleveland Clinic studying Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy, Amyloid - Primary, Transthyretin Amyloidosis, AL Amyloidosis. This study tracks a specific protein (misfolded ATTR oligomers) in people with a genetic form of amyloidosis who don't yet have symptoms. It is for individuals who have a known genetic mutation for hereditary transthyretin amyloidosis. Participation involves regular blood tests over five years to measure protein levels. Currently, there are no specific alternatives for monitoring this early stage of the disease outside of clinical observation. The trial aims to enroll 37 participants.
Official Summary
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
Who Can Participate
Here is what you need to know about eligibility for this trial. You can join if you have a confirmed genetic mutation for hereditary transthyretin amyloidosis. You cannot join if your amyloidosis is not caused by a genetic mutation (wild-type). Age and specific health requirements are not detailed but focus on the genetic condition. This trial is studying Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy, Amyloid - Primary, Transthyretin Amyloidosis, AL Amyloidosis, so participants generally need a confirmed diagnosis.
What They're Measuring
The primary outcome measures how the amount of a specific misfolded protein changes each year in people who develop symptoms, helping doctors understand disease progression. The specific primary outcome measures are: Average % change in oligomers in patients with new onset TTR amyloid symptoms (Annually over 5 years). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.
About This Phase
This study does not have a traditional clinical phase designation. It may be an observational study that follows patients without intervening in their care, an expanded access or compassionate use program, or other non-interventional research. These studies contribute valuable data about disease progression, treatment patterns, and patient outcomes.
Why This Trial Matters
This trial matters because it aims to understand how a specific protein changes over time in people at high risk for hereditary transthyretin amyloidosis, potentially leading to earlier detection and This research targets Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy, Amyloid - Primary, Transthyretin Amyloidosis, AL Amyloidosis, where improved treatment options are needed.
Investor Insight
This observational study provides valuable data on disease markers, which could inform future drug development and market strategies for hereditary transthyretin amyloidosis treatments.
Is This Trial Right for Me?
Ask your doctor if you have a known genetic mutation for hereditary transthyretin amyloidosis and what this study means for you. Participation involves regular blood draws over five years to monitor protein levels. You will be monitored for any new symptoms of amyloidosis. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.
AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.
Study Design
- Study Type: OBSERVATIONAL
- Enrollment: 37 participants
Primary Outcomes
- Average % change in oligomers in patients with new onset TTR amyloid symptoms (Annually over 5 years)
Secondary Outcomes
- % change of oligomer levels relative to baseline level in patients with ATTR specific medication changes (Annually over 5 years)
Full Eligibility Criteria
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.
Trial Locations
- Cleveland Clinic, Cleveland, Ohio, United States
Frequently Asked Questions
What is clinical trial NCT03431896?
NCT03431896 is a Not Applicable OBSERVATIONAL study titled "Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis." It is currently completed and is sponsored by The Cleveland Clinic. The trial targets enrollment of 37 participants.
What conditions does NCT03431896 study?
This trial investigates treatments for Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy, Amyloid - Primary, Transthyretin Amyloidosis, AL Amyloidosis. The primary condition under study is Amyloidosis.
What does Not Applicable mean for NCT03431896?
This study does not have a defined clinical phase. It may be an observational study, expanded access program, or other non-interventional research.
What is the current status of NCT03431896?
This trial is currently "Completed." It started on 2018-02-01. The estimated completion date is 2026-03-09.
Who is sponsoring NCT03431896?
NCT03431896 is sponsored by The Cleveland Clinic. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How many people can participate in NCT03431896?
The trial aims to enroll 37 participants. The trial status is completed.
How is NCT03431896 designed?
This is a observational study.
What are the primary outcomes being measured in NCT03431896?
The primary outcome measures are: Average % change in oligomers in patients with new onset TTR amyloid symptoms (Annually over 5 years). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT03431896 being conducted?
This trial is being conducted at 1 site, including Cleveland, Ohio (United States).
Where can I find official information about NCT03431896?
The official record for NCT03431896 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT03431896. This government database provides the most up-to-date and detailed information about the trial.
What is NCT03431896 testing in simple terms?
This study tracks a specific protein (misfolded ATTR oligomers) in people with a genetic form of amyloidosis who don't yet have symptoms. It is for individuals who have a known genetic mutation for hereditary transthyretin amyloidosis.
Why is this trial significant?
This trial matters because it aims to understand how a specific protein changes over time in people at high risk for hereditary transthyretin amyloidosis, potentially leading to earlier detection and
What are the potential risks of participating in NCT03431896?
The main risk is the discomfort of regular blood draws. There are no direct treatment risks as this is an observational study. Potential risks are related to the natural progression of the underlying amyloidosis condition. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT03431896?
Ask your doctor if you have a known genetic mutation for hereditary transthyretin amyloidosis and what this study means for you. Participation involves regular blood draws over five years to monitor protein levels. You will be monitored for any new symptoms of amyloidosis. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.
What does NCT03431896 signal from an investment perspective?
This observational study provides valuable data on disease markers, which could inform future drug development and market strategies for hereditary transthyretin amyloidosis treatments. This is a Not Applicable trial, which is in early development stages.
What happens if the treatment in this trial doesn't work?
Participation involves regular blood tests over five years to measure protein levels. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.
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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.