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Not yet recruiting NCT07587021

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

Phase II Interventional Primary Hyperoxaluria Type 1 PH1

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: YOLT-203, Placebo.
Who it may be relevant to
Registry conditions: Primary Hyperoxaluria Type 1, PH1. Basic parameters: from 6 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Randomized, Double-blind, Placebo-controlled Study Followed by a Treatment Extension to Evaluate the Efficacy and Safety of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1

Overview

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.

Interventions

  • Genetic YOLT-203
    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.
  • Drug Placebo
    Placebo is a matching intravenous infusion administered over 1 hour.

Primary outcome measures

  • Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected) [Time frame: Months 2 to 6]
Secondary outcome measures (7)
  • Change From Baseline in Plasma Oxalate [Time frame: Months 2 to 6]
  • Change in Estimated Glomerular Filtration Rate (eGFR) [Time frame: Baseline to Month 6]
  • Change in Kidney Stone Burden [Time frame: Baseline to Month 6]
  • Proportion of Participants Achieving Urinary Oxalate Thresholds [Time frame: Month 6]
  • Incidence of Adverse Events [Time frame: Day 1 to Month 12]
  • Pharmacokinetic Parameters of YOLT-203 (Cmax) [Time frame: Dosing through Month 1]
  • Pharmacokinetic Parameters of YOLT-203 (AUC0-inf) [Time frame: Dosing through Month 1]

Eligibility criteria

Inclusion criteria

  • You are 6 years old or older.
  • You have been diagnosed with PH1 (primary hyperoxaluria type 1) through genetic testing.
  • Urine tests show that your oxalate levels are not within certain limits (based on at least two 24-hour urine collections).
  • If you take vitamin B6, your dose has been stable for at least 3 months, and you are willing to keep it the same during the study.
  • You (and/or your parent or legal guardian, if you are under 18) understand the study and agree to follow all study requirements, including signing a consent form.

Exclusion criteria

  • You have signs that oxalate has affected parts of your body outside the kidneys.
  • Your liver blood tests are not within certain limits.
  • You have certain abnormal blood clotting results (unless controlled with medication like warfarin).
  • You have HIV, or active or chronic hepatitis C.
  • You are pregnant, planning to become pregnant, or breastfeeding.
  • You are not willing to use birth control during the study and for 6 months after (if applicable).
  • You are unable to limit alcohol intake to no more than about 2 drinks per day.
  • You have had alcohol or drug abuse in the past year.
  • You are unable to follow standard treatments for PH1 (such as drinking plenty of fluids, taking prescribed medications like potassium citrate, or following dietary advice).

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Crossover
Masking
Triple blind
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07587021 · PH1-YOLT-203-2001

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗