A Phase 1 interventional study of YOLT-203 in Primary Hyperoxaluria Type 1 and PH1, sponsored by YolTech Therapeutics Co., Ltd. Recruiting at 1 site in China. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-08-20.
Sponsored by YolTech Therapeutics Co., Ltd · Phase 1, Interventional, and Treatment
This is a single-arm, open-label, single-dose dose-escalation study. It aims to evaluate the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 (PH1), and to preliminarily assess the effect of a single administration of YOLT-203 on 24-hour urinary oxalate excretion.
The clinical trial will first evaluate the safety and tolerability in adult patients. After the safety profile is preliminarily confirmed, the trial will be sequentially conducted in adolescents, older children, and younger children.
22 studies on the registry are indexed under Primary hyperoxaluria type 1; 8 are open to participants now.
This study's planned enrollment of 9 is below the median of 28 across 16 interventional studies indexed under Primary hyperoxaluria type 1.
Browse Primary hyperoxaluria type 1 studies →YolTech Therapeutics Co., Ltd is the lead sponsor of 3 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Subjects must meet ALL of the following criteria to be eligible for participation in this study:
Age ≥ 6 years at the time of signing the form. Documented confirmation of Primary Hyperoxaluria Type 1 (PH1) diagnosis via genetic analysis prior to enrollment.
Mean 24-hour urinary oxalate excretion level ≥ 0.7 mmol/24 h/1.73 m² obtained from at least two valid 24-hour urine collections.
If the subject is receiving vitamin B6, the stable treatment regimen must have been maintained for a minimum of 90 days prior to enrollment, and the subject is willing to continue this regimen from administration of study drug through the end of the study.
The subject is capable of understanding, willing, and able to comply with study requirements, and provides written informed consent. For subjects below the legal age of consent, a legal guardian must sign the informed consent form, and the subject shall provide assent in accordance with local and national requirements.
Exclusion Criteria:
Patients who respond well to vitamin B6 (pyridoxine) treatment, with urinary oxalate excretion returning to normal after treatment.
Clinical evidence of extrarenal systemic oxalosis.
Any of the following laboratory test results observed at screening:
Known history of infection or evidence of HIV infection (positive HIV antibody); active or chronic virus (HCV) infection (positive HCV antibody and positive HCV RNA polymerase chain reaction) or virus (HBV) infection (positive hepatitis B surface antigen [HBsAg]).
Estimated (eGFR) \< 45 mL/min/1.73 m² at screening (for subjects ≥ 18 years old: calculated using the 2021 Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation; for subjects \< 18 years old: calculated using the Schwartz bedside equation).
Receiving dialysis treatment (peritoneal dialysis [PD] and/or hemodialysis [HD]) or expected to require dialysis treatment during the study period.
Received an drug within 30 days or 5 half-lives (whichever is longer) prior to administration of the study drug, or participated in follow-up of another clinical study before enrollment.
Prior receipt of gene editing therapy, or receipt of small interfering ribonucleic acid (siRNA) therapy within the following timeframes (based on drug half-life and dosing frequency). RNAi therapy is prohibited unless administered as rescue therapy:
i. Lumasiran: less than 15 months from the last dose to administration of the study drug.
ii. Nedosiran: less than 150 days from the last dose to administration of the study drug.
History of kidney or liver transplantation, or expected to require liver and/or kidney transplantation during the study period.
Other medical conditions or comorbidities that, in the Investigator's judgment, may interfere with study compliance or data interpretation.
History of multiple drug allergies or reactions to oligonucleotides or lipid nanoparticles (LNPs).
Unwilling to comply with requirements throughout the entire study participation period until 6 months after the end of the study.
Female subjects who are pregnant, planning to become pregnant, or breastfeeding.
Unwilling or unable to limit alcohol intake throughout the study period. Daily alcohol intake exceeding 2 standard units during the study (1 standard unit: approximately 125 mL wine = approximately 29 mL spirits = approximately 284 mL beer).
History of alcohol abuse or substance abuse within 12 months prior to screening as judged by the Investigator.
Inability to adhere to standard supportive care (adequate water intake, potassium citrate, dietary requirements, etc.).
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Single intravenous infusion of YOLT-203 is administered to enrolled subjects, with a total planned enrollment of 3 to 9 patients. For pediatric subjects aged ≥6 years and \<18 years old, two weight-based dose cohorts are designed: 0.45 mg/kg (maximum capped dose of 35 mg) and 0.6 mg/kg (maximum capped dose of 45 mg). For adult subjects aged ≥18 years old, two fixed-dose cohorts are adopted at 35 mg and 45 mg respectively.
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 via intravenous infusion over a period of 1 hour.
Incidence of adverse events (AEs) and serious adverse events (SAEs)
To evaluate safety and tolerability by assessing the incidence and severity of adverse events (AEs) and serious adverse events (SAEs) after single intravenous infusion of YOLT-203 in Chinese patients with primary hyperoxaluria type 1 (PH1).
Time frame: Baseline, Day 1, Day 2, Day 3, Week 2, Week 4, Week 8, Week 16, Week 24, Week 52
Maximum observed plasma concentration (Cmax) of PK
Time frame: Day 1 through Week 4 after study drug administration
Pharmacodynamic (PD) characteristics following single intravenous administration of YOLT-203 in Chinese patients with primary hyperoxaluria type 1 (PH1)
Detect serum oxalate, serum glycolate levels, as well as 24-hour urinary oxalate and 24-hour urinary glycolate excretion at Week 2, 4, 8, 16, 24 and 52 after drug administration.
Time frame: Baseline to Week 52 post study drug administration
Change in estimated glomerular filtration rate (eGFR)
Time frame: Screening, Baseline, Day 1, Day 2, Day 3, Week 2, Week4, Week8 ,Week 16, Week 24, Week 52
Time to reach maximum concentration (Tmax) of PK
Time frame: Day 1 through Week 4 after study drug administration
Area under the plasma concentration-time curve (AUC) of PK
Time frame: Day 1 through Week 4 after study drug administration
Terminal elimination half-life (t1/2) of PK
Time frame: Day 1 through Week 4 after study drug administration
Incidence of urinary calculi events
Time frame: Screening, Baseline, Day 1, Day 2, Day 3, Week 2, Week4, Week8 ,Week 16, Week 24, Week 52
Plan to share: No
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YolTech Therapeutics Co., Ltd