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

YolTech Therapeutics Co., Ltd

Status and phase: Begins enrollment this month; Phase 2

Conditions: PH1; Primary Hyperoxaluria Type 1

Treatments: Drug: Placebo; Genetic: YOLT-203

Study type: Interventional

Funder types: Industry

Identifiers

NCT07587021

PH1-YOLT-203-2001

Details and patient eligibility

About

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.

Enrollment: 36 estimated patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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).

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Triple Blind

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36 participants in 2 patient groups, including a placebo group

YOLT-203

Experimental group

Description:

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.

Treatment:

Genetic: YOLT-203

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo