Investigational PH1 gene editing therapy

A clinical trial evaluating YOLT-203 in children and adults with PH1.

This clinical trial is evaluating YOLT-203, an investigational, one time gene editing therapy given by IV infusion to people aged 6 and older with PH1. Study treatment includes two infusions: one with the therapy and one with a placebo. Start with a short eligibility check to see if this trial may be a good fit.

Start eligibility check

NCT07587021

About the clinical trial

Living with PH1 can mean ongoing treatment, regular monitoring, and concerns about kidney complications caused by excess oxalate. This clinical trial is studying YOLT-203, an investigational gene editing therapy, in children and adults aged 6 and older with PH1.

YOLT-203 is designed to target a liver gene, called HAO1, involved in oxalate production. Study treatment includes two infusions: one with the therapy and one with a placebo. Some participants will receive placebo during the first part of this study before receiving YOLT-203 later in the study.

What are researchers studying?

Researchers are evaluating the safety of YOLT-203 and whether it may help reduce urinary oxalate over time.

How it works

You’re not alone. Here’s how to get started

To start the eligibility check for this trial, please follow these three steps:

1

Share your contact details

Fill out the contact form so we can connect you with a patient navigator.
Participation is voluntary, and you may stop at any time.
Your personal information is kept private.

2

Quick eligibility check

Answer a few questions to see if you meet the basic criteria for the trial.
Our questions are simple and limited to the basic medical information required for this first assessment.
If you seem to qualify for the trial, you can schedule a free phone call with a navigator on a day and time that works for you.

3

Meet your patient navigator

Your navigator will explain the basics of the trial, including what to expect and what is required.
They will check if you meet requirements for the trial, guide you through the next steps, and answer any questions you may have.
If you are interested and seem to qualify, your patient navigator will connect you with a trial site.

Who may be eligible

This clinical trial has specific requirements to help protect participant safety and support the quality of the research.

You may be able to participate in this trial if you or your loved one:

Are 6 years of age or older
Have been diagnosed with Primary Hyperoxaluria Type 1 (PH1) *
Are not receiving dialysis
Have a kidney function level that may meet study requirements
Have not had a kidney or liver transplant

* You don’t need to have your genetic test results available before being considered for pre‑screening. If you move forward, the study site will perform the necessary genetic testing during the screening process to confirm whether you can take part in the trial.

Why participate

People choose to take part in clinical trials for different reasons. Depending on the trial and their situation, participation may offer regular check-ups and care from a specialized clinical trial team as well as an opportunity to contribute to PH1 research.

By taking part in this clinical trial, you or your loved one may:

receive trial-related care, tests, and assessments at no cost
access an investigational therapy not available outside clinical research
be monitored by the clinical trial team throughout the trial
qualify for reimbursement of trial-related travel expenses, where applicable
help advance research for people living with PH1

YOLT-203 is investigational, and there is no guarantee of benefit.

Frequently Asked Questions (FAQs)

Is starting the eligibility check a commitment to join the clinical trial?

No. Starting the eligibility check does not mean you are joining the clinical trial. It is a way to see whether this trial may be relevant and to understand what the next steps could look like. You can decide at each stage whether you want to continue.

What happens after I submit the form?

After you submit the form, you will complete a short eligibility questionnaire. If your answers suggest this clinical trial may be a fit, you can choose to book a call with a navigator. They can explain the trial, answer your questions, and guide you through the next steps. In your navigator call, if you seem to meet initial pre-screening criteria for trial participation, and you wish to proceed, the navigator can help match you with a clinical trial site for consideration for trial participation.

Who decides whether I’m eligible for the clinical trial?

The clinical trial team at the trial site decides whether someone is eligible for the trial. The eligibility check and navigator call can help identify whether you may meet the basic trial criteria, but they do not confirm final eligibility. The trial team will review the required medical information during screening.

Can I complete the eligibility check on behalf of my child or a loved one?

Yes. A parent, caregiver, or legal representative can complete the eligibility check on behalf of a child or loved one. You may also speak with a navigator to understand the clinical trial and next steps. If needed, the clinical trial team will explain what information or permissions are required later in the process.

Do I need genetic test results already?

No. Genetic test results do not need to be available for referral. Confirmatory genetic testing will be performed at the trial site during screening. The clinical trial team can explain what other medical information may be needed later in the process.

Will all participants receive YOLT-203?

Not at the start of the clinical trial. Some participants will receive YOLT-203 first, while others will receive placebo first, based on the trial group allocation. Participants who do not receive YOLT-203 in the first part of the clinical trial will receive it later in the second part of the trial.

Is there reimbursement for travel?

Yes, reimbursement for trial-related travel expenses is available. The exact support may depend on the trial site and your situation. The team at your trial site can explain what is covered and how reimbursement works.

Find a trial site near you

PH1-YOLT-203-2001 is a global clinical trial taking place at hospitals and clinics in multiple
countries. Each trial site can answer questions about the clinical trial, explain what
participation may involve, and assess eligibility.

Your navigator can help connect you with the most appropriate trial site and answer questions
about travel or logistics.

See available trial sites in the US on the map below:

Please read, your consent is required

If you or a loved one has been diagnosed with Primary Hyperoxaluria Type 1 (PH1), you are invited to complete some pre-screening questions on the following pages. These questions are designed to help determine whether you or the person you care for may be eligible for participation in this clinical research study. If you would like to complete the pre-screening questions on behalf of another person, you will need to provide certain personal and medical information on their behalf.

YolTech Therapeutics, the sponsor of this study, is responsible for the protection of your or your loved one’s personal information. YolTech has engaged myTomorrows (“myTomorrows”, “we” or “us”) to assist with clinical trial recruitment for the study by collecting pre-screening information through this website.

With your consent, we will use this information to evaluate your or your loved one’s eligibility for the study.
The information that could identify you or the person you care for will not be shared with YolTech. Only de-identified data will be shared with YolTech.

You may withdraw your consent at any time. Upon withdrawal of your consent, we will promptly delete your or your loved one’s personal data, except to the extent required to comply with our legal obligations. If you have a question, wish to withdraw consent, or exercise your other privacy rights, please contact myTomorrows at dataprotection@mytomorrows.com, as YolTech will not receive any information that can directly identify you. For more information about YolTech, visit https://www.yoltx.com/privacy.

After answering the pre-screener questions, you will have the opportunity to schedule a call with one of our Patient Navigators, who will collect additional information to assess initial eligibility for this trial. If potentially eligible, they will provide you with further information and answer questions about possible site locations and travel logistics. If we find that you or the person you care for may be eligible for this study, with your consent, we will share the personal information collected with a clinical trial site. The site will contact you to continue the screening process. Your information may also be shared with other vendors of the sponsor as needed for study operations.

If we find that you or the person you care for may be eligible for the trial, the collected data may be transferred internationally if you reside outside the countries where this study takes place. myTomorrows has implemented adequate security measures to ensure it is adequately protected during the transfer.

Even if you or the person you care for does not qualify for this study, it is possible to schedule a call with one of our patient navigators to discuss other options that may be available.

We will retain your or your loved one’s data for 2 years in order to get in touch with you should more options become available during this time period.