ClinicalTrials.gov record NCT06082050

Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhaseEarly phase 1A very early study, usually in a small number of people, to learn how the body handles a treatment. It is not designed to show whether the treatment works.
Age18 years old to 80 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a single-arm, open-label early phase 1 dose-escalation study of intravenous YOLT-201 in people with transthyretin cardiac amyloidosis. The study focuses on safety and tolerability and collects early measures of how the drug behaves in the body and its effects on cardiac and functional tests. Participants receive an IV infusion on Day 1 and may optionally receive a second infusion at the identified optimal biological dose. Follow-up and outcome measurements continue through approximately one year.

What it is studying. The study is testing the safety and tolerability of a single IV infusion of YOLT-201 and collecting early data on pharmacokinetics, immune responses, transthyretin levels, cardiac imaging, and functional measures.

What the researchers hope to learn. Scientific goals include measuring adverse events and tolerability; defining pharmacokinetic parameters (Cmax, Tmax, AUC, t1/2, CL/F); assessing changes in transthyretin and other biomarkers (for example NT-proBNP, troponin, NFL); recording functional outcomes (6-minute and 10-meter walk tests, NYHA class, quality-of-life scores); evaluating heart structure and function by echocardiography and cardiac magnetic resonance; and measuring anti-drug and cas9 antibody levels.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This study is a single-arm, open-label, single-dose escalation trial aimed at evaluating the safety and tolerability of YOLT-201 treatment in patients with Transthyretin Amyloidosis Cardiomyopathy （ATTR-CM）, as well as assessing the preliminary efficacy of subjects treated with YOLT-201.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • YOLT-201

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Male or female subjects aged ≥18 and ≤80 years old at the time of signing informed consent.
  • Diagnosis of transthyretin cardiac amyloidosis (ATTR-CM) based on the 2021 "Chinese Expert Consensus on the Diagnosis and Treatment of Transthyretin Cardiac Amyloidosis," the 2021 European Society of Cardiology (ESC) guidelines, and the 2023 American College of Cardiology (ACC) guidelines, including hereditary ATTR cardiac amyloidosis (ATTRv-CM) and wild-type ATTR cardiac amyloidosis (ATTRwt-CM); and relevant indicators meet the following criteria:
  • 1 New York Heart Association (NYHA) functional classification of I to III; 2.2 6-minute walk test (6-MWT) ≥ 150 meters at screening; 2.3 Echocardiogram evidence of cardiac involvement: left ventricular wall thickness (interventricular septum and/or posterior wall thickness ≥12 mm); 2.4 Brain natriuretic peptide N-terminal prohormone (NT-proBNP) < 2000 pg/mL at screening; 2.5 Receiving stable and standardized cardiovascular medication treatment.
  • Body weight must not be less than 40 kg at screening.
  • Subjects must meet the following laboratory criteria at screening:
  • 1 Aspartate aminotransferase (AST), alanine aminotransferase (ALT), total bilirubin (except for Gilbert's syndrome), International Normalized Ratio (INR) < upper limit of normal (ULN); 4.2 Subjects with a history of Gilbert's syndrome have a total bilirubin not exceeding twice the upper limit of normal (ULN) during the screening period; 4.3 Estimated glomerular filtration rate (eGFR) > 45 mL/min/1.73m\^2 at screening; 4.4 Platelet count ≥ 100 × 10\^9/L; 4.5 Coagulation function at screening: fibrinogen, activated partial thromboplastin time (APTT), prothrombin time (PT), and thrombin time (TT) are all within the normal range; 4.6 Low-density lipoprotein (LDL) cholesterol < 200 mg/dL; 4.7 Vitamin A level ≥ lower limit of normal (LLN); 4.8 Vitamin B12 level ≥ lower limit of normal (LLN).
  • Acceptance of ATTR-CM medication treatment:
  • 1 Unable to receive existing ATTR-CM-related medication due to regional medical resources, health policies, personal financial capacity, or drug intolerance; 5.2 Despite receiving at least 6 months of ATTR-CM medication treatment, the subject's ATTR-CM condition still progresses (meeting at least one of the following): Increased hospitalizations related to heart failure; Worsening of NYHA classification; 6-MWT decrease of at least 30 meters; NT-proBNP increase by 30%; Troponin increase by 30%; Echocardiogram indicates: increase in left ventricular wall thickness by 2 mm, or decrease in left ventricular ejection fraction by ≥5%, or decrease in global longitudinal strain by ≥1%, or decrease in stroke volume by ≥5%, or worsening diastolic function; New conduction block on electrocardiogram.
  • No intake of alcohol from the start of the screening period to 28 days after receiving the trial treatment medication.
  • Female subjects must be postmenopausal for at least one year, or have undergone hysterectomy.
  • Male subjects and their partners must use highly effective contraceptive measures recognized by the doctor throughout the trial process and for at least 5 months after the trial ends.
  • Male subjects must not donate sperm within 84 days after receiving study medication.
  • Voluntary signing of informed consent.

Exclusion criteria

  • Amyloidosis that is not related to TTR protein degeneration, such as immunoglobulin light chain (AL) amyloidosis.
  • History of multiple myeloma.
  • Indeterminate monoclonal gammopathy (MGUS) and/or immunoglobulin free light chain (FLC) ratio alterations, unless fat, bone marrow, or cardiac biopsy confirms the absence of light chains but presence of TTR protein through mass spectrometry or immunoelectron microscopy. For chronic kidney disease (CKD) subjects with no monoclonal protein in blood and urine, the acceptable FLC ratio is 0.26 to 2.25. If the risks of biopsy outweigh the benefits, different results may be discussed with local hematologists, investigators, and medical monitors.
  • Allergy to any lipid nanoparticle (LNP) components, or history of exposure to LNP components or laboratory abnormalities or adverse reaction events related to their treatment: 4.1 Normal baseline, ALT or AST > 3 × ULN or appearance of baseline triple value after receiving LNP product. 4.2 Normal baseline, INR, APTT, or D-dimer > 1.5 × ULN; if baseline is higher than normal after receiving LNP product, it is 1.5 times the baseline value. 4.3 Any adverse reactions related to LNP product treatment are defined as grade 3 or higher (CTCAE). Injection site reactions (IRR) requiring treatment or discontinuation of infusion; slowing down the infusion rate to alleviate infusion-related reactions. The investigator believes that any adverse reaction events related to LNP treatment should be excluded.
  • Use of any of the following ATTR treatments within a certain period:
  • 1 Patisiran (LNP small interfering RNA siRNA) treatment product (within 90 days after administration).
  • 2 Previous treatment with Inotersen (antisense oligonucleotide ASO) (within 160 days after administration).
  • 3 Previous use of Vutrisiran (investigational siRNA therapeutic GalNAc conjugate).
  • 4 Tafamidis (TTR stabilizer): less than 10 days since last drug administration before study drug administration.
  • 5 Diflunisal (TTR stabilizer): less than 3 days since last drug administration before study drug administration.
  • 6 Doxycycline and/or tauroursodeoxycholic acid (TTR chaperone): less than 14 days since last drug administration before study drug administration.
  • 7 Any other drugs used for the treatment of ATTR-CM: less than 30 days or 5 half-lives (whichever is longer) since last drug administration before study drug administration.
  • Other organic heart diseases, such as ischemic heart disease, uncontrolled hypertension, valvular heart disease, etc., leading to cardiomyopathy.
  • Current or previous NYHA class IV symptoms or worsening heart failure symptoms within 90 days before or during screening.
  • Hospitalization or invasive surgery due to cardiovascular and cerebrovascular diseases within 90 days before or during screening. For example, acute coronary syndrome, unstable angina, stroke, TIA, coronary artery reconstruction, cardiac device implantation, valvular repair, or major surgery, etc.
  • Expected to undergo invasive cardiovascular surgery (such as coronary artery stent, pacemaker implantation, etc.) within 28 days after medication.
  • Subjects who cannot or are unwilling to supplement vitamin A.
  • Subjects who cannot or are unwilling to accept the required medication treatment plan before treatment.
  • Antiplatelet (such as aspirin, clopidogrel) or antithrombotic treatment (such as warfarin, dabigatran, apixaban) within 14 days before study drug administration.
  • History of bleeding diathesis or coagulation disorders (such as liver cirrhosis, malignant hematological diseases, antiphospholipid antibody syndrome).
  • The investigator believes that the expected survival is less than 2 years.
  • Ophthalmological examination results consistent with vitamin A deficiency.
  • Known or suspected systemic viral, parasitic, or fungal infections, or expected to receive antibiotic treatment for bacterial infections within 14 days after screening.
  • History of hepatitis B or C infection or positive for hepatitis B surface antigen (HBsAg) or hepatitis C virus antibody (HCV Ab) at screening.
  • History of human immunodeficiency virus (HIV) positivity.
  • Clinically significant thyroid function abnormalities judged by the investigator.
  • History of liver, heart, or other solid organ transplantation or bone marrow transplantation within 1 year at screening, or expected transplantation. Note: No history or plan for corneal transplantation.
  • History of active malignant tumors within 5 years before screening, except for basal cell carcinoma of the skin, cured squamous cell carcinoma of the skin, cured cervical cancer, or low-grade prostate cancer under observation.
  • History of alcohol abuse or drug abuse within 3 years before screening.
  • Female subjects with childbearing potential or who are breastfeeding.
  • Other physical, social, or psychological diseases, including the use of illicit drugs or alcohol, which the investigator believes may adversely affect the subject's safety, impair the assessment of study results, or prevent the subject from complying with any conditions of this study, laboratory abnormalities, or other reasons.
  • Unwilling to comply with study procedures, including follow-ups as specified in the protocol, or unwilling to cooperate fully with the investigator.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive an intravenous (IV) infusion of YOLT‑201 on Day 1 and may voluntarily receive a second infusion at the study's optimal biological dose. They will be followed for safety and testing for about one year with repeated blood draws (for safety labs, drug levels, and anti‑drug/anti‑Cas9 antibodies) and monitoring of NT‑proBNP and troponin. Cardiac imaging (echocardiogram and a cardiac MRI at Week 52), walking tests (6‑minute walk and other timed walks), and quality‑of‑life questionnaires will occur at scheduled visits; some people with unclear blood test results may need tissue biopsy (fat, bone marrow, or cardiac) to confirm the diagnosis. Participants must avoid alcohol from screening through 28 days after dosing, be willing to take vitamin A supplementation as required, follow the trial's contraception rules, and attend follow‑up visits.

How the study is structured. Early Phase 1, interventional single-group (no control), open-label, single-dose escalation design. Participants receive an IV infusion of YOLT-201 on Day 1; some may receive a second infusion at the determined optimal biological dose. No blinding or placebo. Safety monitoring, blood tests, heart imaging, and functional assessments are performed with follow-up for about one year.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under Transthyroxin Amyloidosis Cardiomyopathy. Does it apply to my diagnosis?
  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on wild-type ATTR amyloidosis

This trial is also listed on this site under ATTRv amyloidosis.

Clinical trials121 trials listed for wild-type ATTR amyloidosisEvery trial the registry lists for wild-type ATTR amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in wild-type ATTR amyloidosis researchLatest monthly update: September 2026. Includes: Frailty and support needs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Nov 2, 2023
Primary completion
Jul 3, 2025 (estimated)
Study completion
Feb 10, 2026 (estimated)
Target enrollment
7 participants (estimated)
Last updated on registry
Feb 14, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Zhejiang University
Principal investigator
TingBo Liang, Professor
Zhejiang University
Collaborators
  • YolTech Therapeutics Co., Ltd

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

single-arm
All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
dose escalation
Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
6-minute walk test
A test of how far a person can walk on a flat surface in six minutes. It is used to measure stamina and to track change over time.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Echocardiogram
An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
Estimated glomerular filtration rate
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
electrocardiogram
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
free light chain
Small pieces of antibody protein made by plasma cells and measured in a blood test. In some conditions, such as AL amyloidosis, abnormal free light chains are the source of the disease.
biopsy
Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
investigational
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
5 half-lives
The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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