Actively Recruiting
Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy
Led by Zhejiang University · Updated on 2025-02-14
7
Participants Needed
1
Research Sites
118 weeks
Total Duration
On this page
Sponsors
Z
Zhejiang University
Lead Sponsor
Y
YolTech Therapeutics Co., Ltd
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female subjects aged 18 to 80 years.
- Diagnosis of transthyretin cardiac amyloidosis (ATTR-CM) according to 2021 Chinese, 2021 European Society of Cardiology, and 2023 American College of Cardiology guidelines.
- New York Heart Association (NYHA) functional class I to III.
- Ability to walk at least 150 meters in 6 minutes.
- Echocardiogram showing left ventricular wall thickness of at least 12 mm.
- Brain natriuretic peptide N-terminal prohormone (NT-proBNP) less than 2000 pg/mL.
- Receiving stable and standardized cardiovascular medication treatment.
- Body weight of at least 40 kg.
- Laboratory values within specified limits for liver enzymes, bilirubin, coagulation, kidney function, platelets, cholesterol, vitamin A, and vitamin B12.
- Either unable to receive existing ATTR-CM medications or showing disease progression despite at least 6 months of treatment.
- No alcohol intake from screening to 28 days after treatment.
- Female subjects must be postmenopausal for at least one year or have had a hysterectomy.
- Male subjects and partners must use effective contraception during the study and for 5 months after.
- Male subjects must not donate sperm within 84 days after treatment.
- Voluntary signing of informed consent.
You will not qualify if you...
- Amyloidosis not related to transthyretin protein, such as light chain amyloidosis.
- History of multiple myeloma.
- Indeterminate monoclonal gammopathy or abnormal free light chain ratio unless biopsy confirms ATTR-CM.
- Allergy or adverse reactions to lipid nanoparticle components.
- Use of certain ATTR treatments within specified timeframes before study drug.
- Other heart diseases causing cardiomyopathy.
- NYHA class IV symptoms or worsening heart failure within 90 days.
- Recent hospitalization or invasive cardiovascular surgery within 90 days.
- Planned invasive cardiovascular surgery within 28 days after treatment.
- Inability or unwillingness to supplement vitamin A.
- Inability or unwillingness to follow medication treatment plan.
- Use of antiplatelet or anticoagulant therapy within 14 days before study drug.
- History of bleeding or coagulation disorders.
- Expected survival less than 2 years.
- Vitamin A deficiency on eye exam.
- Active or recent infections requiring antibiotics.
- History or presence of hepatitis B, hepatitis C, or HIV.
- Significant thyroid function abnormalities.
- Recent organ or bone marrow transplantation.
- Recent or active malignancy within 5 years except certain skin or low-grade cancers.
- History of alcohol or drug abuse within 3 years.
- Female subjects who are pregnant, breastfeeding, or of childbearing potential.
- Other conditions that may affect safety or study compliance.
- Unwillingness to comply with study procedures and follow-ups.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
the First Affiliated Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China, 310009
Actively Recruiting
Research Team
Q
Qi Zhang, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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