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Prevention study for Amyloidosis
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?
Acoramidis is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for amyloidosis.
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
- The study aims to see if acoramidis can prevent or delay heart and nerve problems in people who carry a faulty TTR gene but have no symptoms yet.
- Who it's for
- The study is looking for adults who have a genetic variant that puts them at risk for transthyretin amyloidosis but are currently asymptomatic.
- What's involved
- Participants will take the drug acoramidis and may have regular check-ups and tests to monitor their health.
- Time commitment
- The study lasts for an unspecified period, with regular visits for assessments and treatment.
- 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's taking place
- 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
- 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
- Rosedale, New York, United States
+ 67 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 - 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?
Study sites include 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 81 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.