36 Participants NeededMy employer runs this trial

YOLT-203 for Primary Hyperoxaluria

Recruiting at 3 trial locations
KM
CR
Overseen ByClaudia Rainolter
Age: Any Age
Sex: Any
Trial Phase: Phase 2
Sponsor: YolTech Therapeutics Co., Ltd
Must be taking: Vitamin B6
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called YOLT-203 for individuals with Primary Hyperoxaluria Type 1, a condition where excessive oxalate leads to kidney problems. The study aims to determine if YOLT-203 safely and effectively reduces oxalate levels in both children and adults. Participants will initially receive either YOLT-203 or a placebo (a substance with no active drug) and will switch treatments halfway through the study. The trial seeks individuals diagnosed with Primary Hyperoxaluria Type 1 through genetic testing who have high oxalate levels. For more information on participation, please visit the provided link.

As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important medical advancements.

Is there any evidence suggesting that YOLT-203 is likely to be safe for humans?

Research has shown that YOLT-203 is safe for humans. Studies have found that people can tolerate various doses of YOLT-203 without problems. Importantly, no reports of serious side effects have emerged, and no participants have withdrawn from the studies due to side effects. This bodes well for prospective trial participants, suggesting that most people handle the treatment well without major issues.12345

Why do researchers think this study treatment might be promising?

Unlike traditional treatments for primary hyperoxaluria, which often involve dietary management and increased fluid intake, YOLT-203 offers a novel approach by targeting the root cause of the condition at the molecular level. Researchers are excited about YOLT-203 because it uses a unique mechanism to reduce oxalate production, which is a key factor in the formation of kidney stones associated with this condition. This targeted action could potentially lead to more effective management of primary hyperoxaluria, reducing the need for more invasive treatments like dialysis or transplantation.

What evidence suggests that YOLT-203 might be an effective treatment for Primary Hyperoxaluria?

Research has shown that YOLT-203 might help treat Primary Hyperoxaluria Type 1. Early results indicated it could reduce oxalate levels in urine by nearly 70% over 24 hours. Since oxalate can lead to kidney stones, managing its levels is crucial. This reduction lasted up to 16 weeks, suggesting long-term benefits. Importantly, no serious side effects were reported, indicating YOLT-203 could be both safe and effective. Participants in this trial will receive YOLT-203 or a placebo in sequential treatment periods, allowing researchers to further evaluate its potential as a promising option for managing this condition.12467

Who Is on the Research Team?

LX

Ling Xiao, MD, PhD

Principal Investigator

YolTech Therapeutics

Are You a Good Fit for This Trial?

This trial is for children and adults aged 6 or older who have been diagnosed with Primary Hyperoxaluria Type 1 (PH1) by genetic testing. Participants must have stable vitamin B6 use if applicable, abnormal urine oxalate levels, and be able to follow study requirements.

Inclusion Criteria

I am at least 6 years old.
I have PH1 confirmed by genetic testing.
My urine oxalate levels are outside the required range on two tests.
See 2 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Initial 6-month double-blind, placebo-controlled period where participants receive either YOLT-203 or placebo

6 months

Crossover Treatment

Participants who initially received placebo will receive a single-dose of YOLT-203, and those who received YOLT-203 will receive a single-dose of placebo

1 month

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

What Are the Treatments Tested in This Trial?

Interventions

  • YOLT-203

Trial Overview

The study compares a new treatment called YOLT-203 to a placebo in people with PH1. For the first 6 months, participants are randomly assigned to receive either YOLT-203 or placebo without knowing which one they get. Afterward, everyone receives both treatments at different times.

How Is the Trial Designed?

2

Treatment groups

Experimental Treatment

Placebo Group

Group I: YOLT-203Experimental Treatment1 Intervention
Group II: PlaceboPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

YolTech Therapeutics Co., Ltd

Lead Sponsor

Trials
2
Recruited
40+

Citations

NCT07587021 | Study of YOLT-203 in Children and Adults ...

This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1.

YolTech Therapeutics Announces Positive Clinical Data for ...

For patients received the high dose, nearly 70% reduction of 24-hour urinary oxalate levels was observed and sustained through the 16-week ...

FDA greenlights pivotal trial of gene-editing therapy for PH1

Early phase 1 data showed a 70% reduction in urinary oxalate levels with no serious adverse events, indicating promising safety and efficacy.

YOLT-203 Awarded EMA Orphan Drug Designation for ...

Clinical data shared in February 2025 from an early Phase 1 trial of YOLT-203 shows promising results for treating PH1. That trial, which is sponsored by RenJi ...

YOLT-203 - Drug Targets, Indications, Patents

Evaluate the Efficacy and Safety of YOLT-203 in Children and Adults With Primary Hyperoxaluria. Positive Clinical Data for YOLT-203, an In Vivo Gene Editing ...

YolTech Reports Positive Clinical Results for YOLT-203 ...

Across all dosage levels, YOLT-203 was well-tolerated, with no serious adverse events, treatment discontinuations, or patient withdrawals ...

Phase I Exploratory Study of YOLT-203 in Patients With ...

It aims to evaluate the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 (PH1), and to preliminarily ...