Phase 2 Cardiomyopathy Trial, Recruiting

Written by an AI model from 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 (any gender)
• People who weigh between 40 and 90 kg at the time of screening
• People who have had a TTR gene mutation confirmed through genetic testing
• People whose liver function blood test results (AST, ALT, and bilirubin) are within the normal range at screening (people with a condition called Gilbert syndrome may have a slightly higher bilirubin level and still meet this criterion — confirm with trial site)
• People whose kidneys are filtering at an adequate level, based on a standard calculation called the CKD-EPI formula
• People whose platelet count and blood clotting measurements fall within acceptable ranges at screening
• People whose vitamin A and vitamin B12 levels are at or above the lower end of the normal range
• People whose LDL cholesterol is below 200 mg/dL (5.17 mmol/L)
• People for whom currently approved ATTR treatments are not available or accessible in China, or who cannot tolerate them for medical, financial, or other reasons, OR people who have been on approved ATTR treatment for at least 3 months but whose disease has continued to get worse as assessed by the trial doctor — with worsening measured through specific heart or nerve-related tests depending on the type of ATTR
• People who agree to stop drinking alcohol from the screening period until 28 days after receiving the study drug
• Female participants who have gone through menopause (no period for at least 1 year) or have had their uterus and/or ovaries surgically removed
• Male participants and their partners who have no plans for pregnancy from screening until 6 months after the trial ends, and who agree to use effective non-drug contraception during the trial
• People (or their legal representative) who understand and sign the informed consent form
• People who agree not to receive any other ATTR drug treatment for at least 8 weeks after receiving the study drug YOLT-201

For people with the nerve form of ATTR (ATTR-PN) only:

• People diagnosed with ATTR-PN according to the relevant clinical consensus guidelines, with nerve damage scores within a specific range (NIS score between 5 and 130, and PND score no worse than IIIb) at screening
• People with an NT-proBNP blood level below 600 pg/mL at screening (this is a marker related to heart stress)

For people with the heart form of ATTR (ATTR-CM) only:

• People diagnosed with ATTR-CM according to the relevant expert consensus guidelines
• People whose heart function is classified as Grade II or III on the New York Heart Association (NYHA) scale — meaning they have some symptoms but are not in the most severe category
• People who can walk at least 150 metres in a 6-minute walk test at screening
• People with an NT-proBNP blood level between 600 and 3,000 pg/mL at screening
• People whose heart ultrasound (echocardiogram) shows heart wall thickening of 12 mm or more, indicating the heart is affected

Who may not be able to join:

• People whose amyloidosis is not caused by the TTR protein (for example, those with light chain amyloidosis)
• People who have amyloidosis affecting the membranes around the brain and spinal cord
• People who are allergic to any component of lipid nanoparticles (the type of delivery system used in the study drug), or who have previously had significant side effects from a lipid nanoparticle treatment
• People who have recently used certain ATTR medications within specified time periods before the study drug is given — including Patisiran, Inotersen, Vutrisiran, Tafamidis, Diflunisal, Doxycycline, taurodeoxycholic acid, or other ATTR treatments (the exact time restrictions vary by drug and trial stage — confirm with trial site)
• People who have previously received any investigational gene-editing drug
• People who are unable or unwilling to take vitamin A supplements during the trial
• People with a history of multiple myeloma (a type of blood cancer)
• People whose eye examination at screening shows signs of vitamin A deficiency
• People with thyroid function test results that the trial doctor considers clinically significant
• People who have had a known or suspected active infection (viral, parasitic, or fungal) within 14 days before screening
• People with a history of hepatitis B, hepatitis C, or HIV/AIDS, or who test positive for these infections at screening
• People who have previously had a liver, heart, or other organ transplant, or a bone marrow transplant, or who are expected to need one within the next year (a history of corneal transplant is an exception)
• People with a history of bleeding disorders or blood clotting problems (such as cirrhosis, blood cancers, or antiphospholipid antibody syndrome)
• People who had an acute blood clot event (such as a heart attack or stroke) within 6 months before screening, or who test positive for certain inherited clotting risk factors (Leiden factor V or prothrombin gene mutations)
• People who have had a malignant (cancerous) tumour within the past 5 years — with exceptions for certain skin cancers and cervical carcinoma in situ that have been fully treated (confirm with trial site)
• People who are planning to have invasive heart surgery during the trial (such as stent placement, bypass surgery, or pacemaker implantation), or who have had such surgery or been hospitalised for heart failure within 90 days before screening
• People with a history of alcohol abuse within the past 3 years (defined as women drinking 4 or more drinks per day or 8 or more per week, or men drinking 5 or more per day or 15 or more per week, where 1 drink equals 14 g of pure alcohol)
• People whose life expectancy is less than 1 year
• People who the trial doctor considers unsuitable for any other reason

For people with the nerve form of ATTR (ATTR-PN) only:

• People who have another known condition causing nerve damage (such as diabetic neuropathy or nerve problems related to an autoimmune disease)
• People who have had type 1 or type 2 diabetes for 5 or more years
• People whose heart function was classified as NYHA Grade III or IV at any point in the 90 days before screening

For people with the heart form of ATTR (ATTR-CM) only:

• People whose heart function was classified as NYHA Grade IV at any point in the 90 days before screening
• People whose nerve damage score (PND score) is at Grade IIIa, IIIb, or IV at screening
• People who have a different type of heart muscle disease not caused by TTR (such as one related to high blood pressure, heart valve problems, or reduced blood supply to the heart)

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