Amyloidosis study: Acoramidis
Official title Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
ClinicalTrials.gov ID: NCT06563895
What this study is testing
What is Acoramidis?
an investigational oral medicine being studied as a potential treatment for transthyretin amyloidosis (ATTR). is designed to bind tightly to transthyretin (TTR) in the blood and stabilize its structure, preventing it from breaking apart and forming amyloid plaques.
Also referred to as AG10, ALXN2060.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study aims to find out if taking acoramidis early can stop or slow down the buildup of harmful protein clumps in the heart or nerves.
- Who it's for
- The study is looking for adults who have a specific inherited gene change but do not yet have any signs or symptoms of the disease.
- What's involved
- Participants will take the experimental drug acoramidis and undergo regular medical tests to check their heart and nerve health.
- Time commitment
- The study involves long-term follow-up with regular visits to monitor health over time.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 75
You may be able to join if
- Male or female ≥ 18 to ≤ 75 years of age inclusive.
- Participants must have an established genotype (hetero- or homozygosity) through a medically-indicated genetic test of a TTR gene variant that is...
- Participant's age is within 10 years younger than or older than PADO. Key
You likely can't join if
- Evidence of ATTR-CM or ATTR-PN.
- Current or past (within last 1 to 12 months, depending on specific agent) treatment with other TTR modifying therapies.
- Contraindication to or inability to undergo cardiac magnetic resonance testing.
- Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy
- Other diseases or conditions such has cancer within 5 years, untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C...
- Major surgery within the past 3 months or planned during the next 12 months.
See the full eligibility criteria
- Male or female ≥ 18 to ≤ 75 years of age inclusive.
- Participants must have an established genotype (hetero- or homozygosity) through a medically-indicated genetic test of a TTR gene variant that is known to be pathogenic or likely pathogenic (eg, V30M/p.V50M...
- Participant's age is within 10 years younger than or older than PADO. Key
- Evidence of ATTR-CM or ATTR-PN.
- Current or past (within last 1 to 12 months, depending on specific agent) treatment with other TTR modifying therapies.
- Contraindication to or inability to undergo cardiac magnetic resonance testing.
- Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy
- Other diseases or conditions such has cancer within 5 years, untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C, HIV.
- Major surgery within the past 3 months or planned during the next 12 months.
- Known hypersensitivity to acoramidis.
The study team makes the final eligibility decision.
Where it is enrolling
- La Jolla, California, United States
- Los Angeles, California, United States
- San Francisco, California, United States
- Stanford, California, United States
- Aurora, Colorado, United States
- New Haven, Connecticut, United States
- Washington D.C., District of Columbia, United States
- Jacksonville, Florida, United States
- Miami, Florida, United States
- Weston, Florida, United States
- Atlanta, Georgia, United States
- Chicago, Illinois, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Detroit, Michigan, United States
- Rochester, Minnesota, United States
- Kansas City, Missouri, United States
- St Louis, Missouri, United States
- New Brunswick, New Jersey, United States
- New York, New York, United States
+ 68 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at La Jolla, California, United States; Los Angeles, California, United States; San Francisco, California, United States; Stanford, California, United States; Aurora, Colorado, United States; New Haven, Connecticut, United States and 82 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.