Primary Hyperoxaluria Type 1Phase 2NCT07587021
Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
A Phase 2 study testing YOLT-203 in people with Primary Hyperoxaluria Type 1 and PH1, run by YolTech Therapeutics Co., Ltd at 4 sites in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Primary Hyperoxaluria Type 1, PH1
- What is being tested?
- YOLT-203
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 4 sitesUnited States
- Study dates
- 16 Sep 2026to Feb 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
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- 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 locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Childrens Hospital of Colorado
Aurora, Colorado, 80045
Kati Dugan, 720-777-8215, Kati.dugan@childrenscolorado.org
Sarah Nelson-Taylor, MD
- Recruiting
University of Alabama at Birmingham (UAB Hospital)
Birmingham, Alabama, 35249
Crystal Taylor, 205-934-3411, crystalmtaylor@uabmc.edu
Kyle Wood, MD
- Not yet recruiting
University of Pennsylvania Health System (UPHS) - Penn Medicine
Philadelphia, Pennsylvania, 19107
Kim Clinton, (800) 789-7366, Kimberly.Clinton@pennmedicine.upenn.edu
Siddharth Shah, MD
- Not yet recruiting
Washington University/Barnes Jewish Hospital
St Louis, Missouri, 63108
Muhammad Khan, 3143628344, khanmu@wustl.edu
Tarek Alhamad, MD
About this study
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
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.
- Conditions
- Primary Hyperoxaluria Type 1, PH1
- Being tested
- YOLT-203, Placebo
- Keywords
- ph1, primary hyperoxaluria Type 1, yoltech, yolt203, primary hyperoxaluria, recurrent kidney stones, rare kidney stones, YOLT-203, genetic kidney stones, gene editing, gene therapy, AGXT gene, CRISPR-Cas9, Alanine Glyoxylate Aminotransferase Gene
- Medical subject headings
- Primary hyperoxaluria type 1, Hyperoxaluria, Hyperoxaluria, Primary, Kidney Calculi
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
YOLT-203Genetic
Also called Investigational Gene Editing Therapy
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.
PlaceboDrug
Placebo is a matching intravenous infusion administered over 1 hour.
| Study group | Role | What they receive |
|---|---|---|
| YOLT-203 | Experimental | 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. Receives: Genetic: YOLT-203 |
| Placebo | Placebo comparator | 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. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected)
Percent change from baseline in 24-hour urinary oxalate excretion corrected for body surface area (BSA) across Months 2 to 6.
Measured: Months 2 to 6
Secondary outcomes (7)
Change From Baseline in Plasma Oxalate
Percent and absolute change from baseline in plasma oxalate levels across Months 2 to 6.
Measured: Months 2 to 6
Change in Estimated Glomerular Filtration Rate (eGFR)
Change from baseline in estimated glomerular filtration rate (eGFR).
Measured: Baseline to Month 6
Change in Kidney Stone Burden
Change from baseline in number and summed surface area of kidney stones as assessed by renal ultrasound.
Measured: Baseline to Month 6
Proportion of Participants Achieving Urinary Oxalate Thresholds
Proportion of participants achieving urinary oxalate levels ≤ upper limit of normal (ULN) and ≤ 1.5 × ULN at Month 6.
Measured: Month 6
Incidence of Adverse Events
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs), graded using CTCAE v6.0.
Measured: Day 1 to Month 12
Pharmacokinetic Parameters of YOLT-203 (Cmax)
Cmax for YOLT-203 components (mRNA, CRISPR RNA, and lipid nanoparticles).
Measured: Dosing through Month 1
Pharmacokinetic Parameters of YOLT-203 (AUC0-inf)
AUC0-inf for YOLT-203 components (mRNA, CRISPR RNA, and lipid nanoparticles).
Measured: Dosing through Month 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2026
- Study start16 Sep 2026
- Primary completionAug 2027 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Hyperoxaluria Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- After the initial 6-month double-blind period, participants will enter a 6-month treatment extension 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.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- YolTech Therapeutics Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Wensu Yu, Study director, YolTech Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07587021
- Sponsor's protocol ID
- PH1-YOLT-203-2001
- Status last verified
- Oct 2026
- First submitted
- 1 May 2026
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07587021
- Acronym / sponsor ID
- PH1-YOLT-203-2001
- Conditions
- Primary Hyperoxaluria Type 1, PH1
- Keywords
- ph1, primary hyperoxaluria Type 1, yoltech, yolt203, primary hyperoxaluria, recurrent kidney stones, rare kidney stones, YOLT-203
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- YolTech Therapeutics Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 16 Sep 2026 (actual)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 14 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- After the initial 6-month double-blind period, participants will enter a 6-month treatment extension 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.
| Arm | Type | Receives |
|---|---|---|
| YOLT-203 | Experimental | Genetic: YOLT-203Participants 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 | Placebo comparator | Drug: PlaceboParticipants 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. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| YOLT-203Also: Investigational Gene Editing TherapyYOLT-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. | Genetic | YOLT-203 |
| PlaceboPlacebo is a matching intravenous infusion administered over 1 hour. | Drug | Placebo |
Primary outcome
Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected)
Time frame: Months 2 to 6
Percent change from baseline in 24-hour urinary oxalate excretion corrected for body surface area (BSA) across Months 2 to 6.
7 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2026.
- For patients interested in additional information on how to participate, please follow this link:Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Wensu Yu study director
YolTech Therapeutics
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing YOLT-203 in people with Primary Hyperoxaluria Type 1 and PH1, run by YolTech Therapeutics Co., Ltd at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kim Marie Grabbe, MS, MBA (217-851-5517, Kim.Marie.Grabbe@yoltx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected) (time frame: Months 2 to 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2026.