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)

YolTech Therapeutics Co., Ltd3 sites in 1 country31 target enrollmentMay 23, 2024

ConditionsTransthyretin Amyloidosis Polyneuropathy Transthyrexin Amyloidosis Cardiomyopathy

InterventionsYOLT-201

DrugsYOLT-201

Overview

Phase: Phase 1
Intervention: YOLT-201
Conditions: Transthyretin Amyloidosis Polyneuropathy
Sponsor: YolTech Therapeutics Co., Ltd
Enrollment: 31
Locations: 3
Primary Endpoint: Safety and Tolerability
Status: Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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).

Registry

clinicaltrials.gov

Start Date

May 23, 2024

End Date

June 30, 2026

Last Updated

last year

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

YolTech Therapeutics Co., Ltd

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Age 18 - 80 years old (including the critical values), regardless of gender;
•Body weight at the time of screening is between 40 - 90kg (including the critical values);
•TTR gene mutation is confirmed by genetic testing;
•At the time of screening, the following laboratory standards must be met:
•AST, ALT, and TBIL ≤ the upper limit of the normal value (ULN);
•For subjects with Gilbert syndrome, TBIL ≤ 2 times ULN;
•Glomerular filtration rate (GFR) ≥ 45 mL/min/1.73m2 (calculated according to the CKD-EPI formula);
•Platelet count ≥ 100 × 109/L;
•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;
•Vitamin A and vitamin B12 ≥ the lower limit of the reference value (LLN);

Exclusion Criteria

•Amyloidosis is not caused by TTR protein, such as light chain amyloidosis;
•There is meningeal transthyretin amyloidosis;
•Allergic to any lipid nanoparticle (LNP) component or has previously received LNP and experienced treatment-related laboratory abnormalities or adverse events;
•Use any of the following ATTR treatments within the prescribed time:
•In the dose escalation stage of the first stage, the use history of Patisiran, Inotersen, and Vutrisiran is excluded;
•In the dose expansion stage of the second stage, the following are excluded: Patisiran is used within 90 days before the administration of the investigational drug; Inotersen is used within 160 days before the administration of the investigational drug; Vutrisiran has a previous use history;
•Tafamidis: used within 10 days before the administration of the investigational drug;
•Diflunisal: used within 3 days before the administration of the investigational drug;
•Doxycycline and/or taurodeoxycholic acid: used within 14 days before the administration of the investigational drug;
•Previous use history of investigational gene editing drugs;