Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)

Recruiting Early Phase 1 Interventional Study
Type 1 Primary Hyperoxaluria
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
2 and older
Sex
Any
Trial phase
Early Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
21 (estimated)
Sponsor
RenJi Hospital · Other
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About This Trial
This study is a single-arm, open-label, single-dose, dose-escalation, and dose-expansion trial, aiming to assess the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 (PH1), and to preliminarily evaluate the effect of a single dose of YOLT-203 on plasma oxalate levels. In this study, the maximum duration of the screening period is 60 days, with the treatmen…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * The age is ≥ 2 years old at the time of signing the informed consent. * Have AGXT gene mutations and be diagnosed with primary hyperoxaluria (PH1); eGFR ≥ 30 ml/min/1.73m2. * At least 2 times of 24-hour urinary oxalate excretion ≥ 0.7 mmol/1.73m2/day or the ratio of urinary ox…
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