Early Phase 1 Cardiomyopathy Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People aged 18 to 80 years old at the time of signing the consent form.
• People diagnosed with transthyretin cardiac amyloidosis (a condition where a protein called transthyretin builds up in the heart), either the inherited form or the age-related form, according to recognised medical guidelines.
• People whose heart failure symptoms fall into category I, II, or III on a standard medical scale (meaning symptoms range from none during normal activity to noticeable during light activity, but not severe at rest).
• People who can walk at least 150 metres in 6 minutes at the screening stage.
• People whose heart scans show thickening of the heart wall (the inner wall measuring 12 mm or more).
• People whose blood test for a heart stress marker (NT-proBNP) is below 2000 pg/mL at screening.
• People who are already on a stable, standard course of heart medications.
• People who weigh at least 40 kg at screening.
• People whose blood test results for liver function, blood clotting, kidney function, platelet count, cholesterol, vitamin A, and vitamin B12 all fall within the ranges required by the trial (confirm with trial site for specific values).
• People who are either unable to access existing ATTR-CM medications due to local availability, health policy, personal financial situation, or medication intolerance — OR people whose condition has continued to get worse despite at least 6 months of ATTR-CM treatment, shown by at least one measurable sign of decline (such as more heart failure hospital admissions, worsening symptoms, reduced walking distance, or changes on heart scans or blood tests).
• People who agree not to drink alcohol from the start of screening until 28 days after receiving the trial treatment.
• Female participants who have gone through menopause (at least one year) or have had their uterus removed.
• Male participants whose doctor agrees they will use highly effective contraception throughout the trial and for at least 5 months after it ends, and who agree not to donate sperm for at least 84 days after receiving the study medication.
• People who are willing to sign a consent form agreeing to take part voluntarily.

Who may not be able to join:

• People whose amyloidosis is caused by something other than the TTR protein, such as the AL (light chain) type.
• People with a history of multiple myeloma (a type of blood cancer).
• People with a condition called monoclonal gammopathy of undetermined significance (MGUS) or abnormal light chain protein ratios in the blood, unless a biopsy has confirmed the amyloid is TTR-related and not light chain-related (confirm with trial site for exceptions).
• People who have had an allergic reaction to lipid nanoparticle (LNP) components (a type of substance used in some injected medicines), or who have had significant abnormal test results or serious side effects after receiving an LNP-based treatment.
• People who have recently received certain ATTR treatments, including Patisiran (within the past 90 days), Inotersen (within the past 160 days), Vutrisiran (any prior use), Tafamidis (within the past 10 days), Diflunisal (within the past 3 days), Doxycycline and/or tauroursodeoxycholic acid (within the past 14 days), or any other ATTR-CM drug within 30 days or 5 half-lives, whichever is longer.
• People with other significant heart conditions — such as blocked heart arteries, uncontrolled high blood pressure, or heart valve disease — that are causing their heart muscle problems.
• People who currently have, or previously had, the most severe level of heart failure symptoms (category IV), or whose heart failure got significantly worse in the 90 days before or during screening.
• People who were hospitalised or had invasive heart or brain surgery (such as for a heart attack, stroke, or device implantation) in the 90 days before or during screening.
• People who are expected to need invasive heart procedures (such as a stent or pacemaker) within 28 days of receiving the study medication.
• People who are unwilling or unable to take vitamin A supplements.
• People who are unwilling or unable to follow the required medication plan before starting treatment.
• People who have taken blood-thinning or antiplatelet medications (such as aspirin, clopidogrel, warfarin, or apixaban) in the 14 days before the study drug is given.
• People with a history of bleeding disorders or clotting problems (such as liver cirrhosis, blood cancers, or antiphospholipid antibody syndrome).
• People whose treating doctor believes they are unlikely to survive for more than 2 years.
• People whose eye examination shows signs consistent with vitamin A deficiency.
• People with a known or suspected active systemic viral, parasitic, or fungal infection, or who are expected to need antibiotic treatment for a bacterial infection within 14 days of screening.
• People with a history of hepatitis B or C infection, or whose screening blood test is positive for hepatitis B or C markers.
• People with a history of HIV infection.
• People with thyroid function problems that the treating doctor considers clinically significant.
• People who have had a liver, heart, other solid organ, or bone marrow transplant within the past year, or who are expecting to have one (note: corneal transplant history or plans are not a concern here).
• People with a history of active cancer in the past 5 years, with the exception of certain treated skin cancers, treated cervical cancer, or low-grade prostate cancer being managed without active treatment.
• People with a history of alcohol or drug misuse in the past 3 years.
• Women who are able to become pregnant, or who are currently breastfeeding.
• People with physical, social, or psychological conditions — including use of illicit drugs or alcohol — that the treating doctor believes could affect their safety or the reliability of trial results.
• People who are unwilling to follow the study procedures or attend required follow-up appointments.

Important: Always verify eligibility with the trial site directly before applying.