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

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
YolTech Therapeutics Co., Ltd
Study ID
NCT07587021
Phase
PHASE2
Status
Recruiting
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Conditions

  • PH1
  • Primary Hyperoxaluria Type 1

Eligibility Criteria

Sex
ALL
Age
6 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • YOLT-203 — GENETIC
    YOLT-203 is an investigational gene editing therapy being evaluated for the treatment of Primary Hyperoxaluria Type 1 (PH1). It is administered by intravenous infusion over 1 hour.
  • Placebo — DRUG
    Placebo is a matching intravenous infusion administered over 1 hour.

Study Details

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. For patients interested in additional information on how to participate, please follow this link: https://mytomorro.ws/ph1trial.

Key Dates

First listed
May 14, 2026
Start date
Aug 31, 2026
Status verified
Aug 2026
Primary completion
Aug 31, 2027
Completion
Feb 29, 2028

Study Design

Enrollment
36 participants (estimated)
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Experimental: YOLT-203
    Participants will receive both study treatments in sequential treatment periods according to randomized assignment. Participants who initially receive YOLT-203 during the double-blind treatment period and will cross over to the placebo treatment in the subsequent treatment period, followed by safety and efficacy follow-up.
  • Placebo Comparator: Placebo
    Participants will receive both study treatments in sequential treatment periods according to randomized assignment. Participants who initially receive placebo during the double-blind treatment period and will cross over to the YOLT-203 treatment in the subsequent treatment period, followed by safety and efficacy follow-up.

Primary Outcome Measure

Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected) [ Time Frame: Months 2 to 6 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of Alabama at Birmingham (UAB Hospital)BirminghamAlabama35249
Kyle Wood, MD
[email protected]
205-934-3411

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By research site
University of Alabama at Birmingham (UAB Hospital)· Birmingham, AL

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