Status: Not yet recruiting

Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)

This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1. After the initial randomized, 6-month double-blind, placebo-controlled period, participants who were initially assigned to placebo will receive a single-dose of YOLT-203 treatment whereas participants in the YOLT-203 group will receive a single-dose of placebo infusion.

Participants needed: 36

Trial details

Phase: Phase 2Age: 6+Biological sex: AllType: InterventionalSponsor: YolTech Therapeutics Co., LtdUpdated: May 14, 2026

Eligibility criteria

You are 6 years old or older. [+4]

You have signs that oxalate has affected parts of your body outside the kidneys. [+8]

Status: Recruiting

Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD

The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.

Participants needed: 28

Trial details

Phase: Phase 2Biological sex: AllType: InterventionalSponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyUpdated: Dec 24, 2025Locations: 18

Eligibility criteria

adults and adolescents (aged ≥ 12 years) [+16]

Prior hepatic transplantation; or scheduled transplantation within 6 months of D... [+16]

Status: Not yet recruiting

Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3

Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3.

Participants needed: 42

Trial details

Phase: Phase 3Age: 6+Biological sex: AllType: InterventionalSponsor: BiocodexUpdated: Jun 18, 2024

Eligibility criteria

1. Male or female patients aged ≥ 6 years at the time of consent signature [+10]

1. Any relevant change in the use of any component of the standard of care (flui... [+17]