Actively Recruiting
A Phase I/IIa，Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
Led by YolTech Therapeutics Co., Ltd · Updated on 2024-08-06
31
Participants Needed
3
Research Sites
109 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of YOLT-201 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and participants with hereditary transthyretin amyloidosis with cardiomyopathy (ATTRv-CM).
CONDITIONS
Official Title
A Phase I/IIa，Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to 80 years old, any gender
- Body weight between 40 and 90 kg
- Confirmed TTR gene mutation by genetic testing
- Laboratory values within normal or specified limits for liver enzymes, bilirubin, kidney function, platelets, clotting tests, vitamin A and B12, and LDL cholesterol
- Not currently receiving approved ATTR treatments or disease progressing despite these treatments
- Agree to stop alcohol consumption from screening through 28 days after treatment
- Female participants are menopausal or have had uterine/ovarian removal; males and partners agree to effective contraception during study
- Able to understand and sign informed consent
- Agree not to use other ATTR treatments for at least 8 weeks after YOLT-201 administration
- For ATTR-PN: diagnosis confirmed with NIS score 5-130 and PND score up to IIIb; NT-proBNP under 600 pg/mL
- For ATTR-CM: diagnosis confirmed; NYHA class II-III; 6-minute walk test ≥150 m; NT-proBNP between 600 and 3000 pg/mL; echocardiography showing cardiac involvement with specified heart wall thickness
You will not qualify if you...
- Amyloidosis not caused by TTR protein
- Presence of meningeal transthyretin amyloidosis
- Allergy to lipid nanoparticle components or prior adverse reactions to LNP
- Recent use of certain ATTR treatments within specified timeframes before study drug administration
- Unable or unwilling to take vitamin A supplements
- History of multiple myeloma
- Eye exam showing vitamin A deficiency
- Significant abnormal thyroid function
- Recent or active systemic infections
- History of hepatitis B, hepatitis C, HIV, or positive viral markers
- Prior or planned organ or bone marrow transplant within 1 year (except corneal)
- History of bleeding or clotting disorders
- Recent acute thrombosis or positive clotting factor tests
- History of cancer within 5 years (except certain skin and cervical cancers)
- Planned or recent invasive cardiovascular surgery or recent heart failure hospitalization
- History of alcohol abuse within 3 years
- Life expectancy under 1 year
- Other investigator-determined exclusion reasons
- For ATTR-PN: other known neuropathy causes, diabetes diagnosed ≥5 years, NYHA class III or IV
- For ATTR-CM: NYHA class IV, PND score IIIa or worse, other cardiomyopathies not caused by TTR
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100000
Actively Recruiting
2
The Second Xiangya Hospital of Central South University
Changsha, Hunan, China, 410000
Actively Recruiting
3
The First Affiliated Hospital, School of Medicine, Zhejiang University
Hanzhou, Zhejiang, China, 310000
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here