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Tests treatment safety and results for Transthyroxin Amyloidosis Cardiomyopathy
Official title Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy
ClinicalTrials.gov ID: NCT06082050
What this study is testing
What is YOLT-201?
YOLT-201 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for transthyroxin amyloidosis cardiomyopathy.
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 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.
- Phase 1: an early, usually small safety study
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 80
You may be able to join if
- Male or female people 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...
- Body weight must not be less than 40 kg at screening.
- people must meet the following laboratory criteria at screening: 4.1 Aspartate aminotransferase (AST), alanine aminotransferase (ALT), total...
- Acceptance of ATTR-CM medication treatment: 5.1 Unable to receive existing ATTR-CM-related medication due to regional medical resources, health...
You likely can't join if
- 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...
- Allergy to any lipid nanoparticle (LNP) components, or history of exposure to LNP components or laboratory abnormalities or adverse reaction events...
- Use of any of the following ATTR treatments within a certain period: 5.1 Patisiran (LNP small interfering RNA siRNA) treatment product (within 90...
- Other organic heart diseases, such as ischemic heart disease, uncontrolled hypertension, valvular heart disease, etc., leading to cardiomyopathy.
See the full eligibility criteria
Who can join
- Male or female people 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)...
- Body weight must not be less than 40 kg at screening.
- people must meet the following laboratory criteria at screening: 4.1 Aspartate aminotransferase (AST), alanine aminotransferase (ALT), total bilirubin (except for Gilbert's syndrome), International Normalized Ratio...
- Acceptance of ATTR-CM medication treatment: 5.1 Unable to receive existing ATTR-CM-related medication due to regional medical resources, health policies, personal financial capacity, or drug intolerance; 5.2 Despite...
- No intake of alcohol from the start of the screening period to 28 days after receiving the trial treatment medication.
- Female people must be postmenopausal for at least one year, or have undergone hysterectomy.
- Male people 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 people must not donate sperm within 84 days after receiving study medication.
- Voluntary signing of informed consent.
What rules you out
- 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...
- 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...
- Use of any of the following ATTR treatments within a certain period: 5.1 Patisiran (LNP small interfering RNA siRNA) treatment product (within 90 days after administration). 5.2 Previous treatment with Inotersen...
- 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...
- Expected to undergo invasive cardiovascular surgery (such as coronary artery stent, pacemaker implantation, etc.) within 28 days after medication.
- people who cannot or are unwilling to supplement vitamin A.
- people 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...
- History of alcohol abuse or drug abuse within 3 years before screening.
- Female people 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...
- Unwilling to comply with study procedures, including follow-ups as specified in the protocol, or unwilling to cooperate fully with the investigator.
The study team makes the final eligibility decision.
Where it's taking place
- Hangzhou, Zhejiang, China
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 80 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Hangzhou, Zhejiang, China. 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.