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 statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-80
SponsorYolTech Therapeutics Co., Ltd

About this trial

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

Qualifiers

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;

AST, ALT, and TBIL ≤ the upper limit of the normal value (ULN);

Disqualifiers

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;

In the dose escalation stage of the first stage, the use history of Patisiran, Inotersen, and Vutrisiran is excluded;

Trial design

Treatments tested in this trial

  • YOLT-201

Treatment groups

31 Participants
are divided into 1 treatment group

Sponsors and collaborators