NCT06539208 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)

Trial Parameters

Eligibility Fast-Check

Enter your details for a quick preliminary check. This does not replace medical advice.

Brief Summary

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).

Eligibility Criteria

Inclusion Criteria: 1. Age 18 - 80 years old (including the critical values), regardless of gender; 2. Body weight at the time of screening is between 40 - 90kg (including the critical values); 3. TTR gene mutation is confirmed by genetic testing; 4. At the time of screening, the following laboratory standards must be met: 1. AST, ALT, and TBIL ≤ the upper limit of the normal value (ULN); 2. For subjects with Gilbert syndrome, TBIL ≤ 2 times ULN; 3. Glomerular filtration rate (GFR) ≥ 45 mL/min/1.73m2 (calculated according to the CKD-EPI formula); 4. Platelet count ≥ 100 × 109/L; 5. Partial thromboplastin time (APTT), prothrombin time (PT), and thrombin generation time (TGT) are all within the reference value range, fibrinogen (FIB) ≥ the lower limit of the normal value (LLN) and ≤ 1.5\*ULN, the international normalized ratio (INR) ≤ ULN, and if taking anticoagulant drugs, it is ≤ 2.5\*ULN; 6. Vitamin A and vitamin B12 ≥ the lower limit of the reference value (LLN); 7. Low-density lipop

Related Trials