ClinicalTrials.gov record NCT07776626
Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1)
Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1)
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase I, single-arm study tests a single intravenous dose of YOLT-203 in people with Primary Hyperoxaluria Type 1 (PH1). It is an open-label, dose-escalation study that first evaluates safety in people and, after initial safety is assessed, proceeds to adolescents and children. Participants receive one infusion and are followed with safety checks and laboratory and urine tests for up to 52 weeks.
What it is studying. The study is testing the safety and tolerability of a single intravenous administration of YOLT-203 in people with PH1, and it will collect early data on how the drug behaves in the body (pharmacokinetics) and its effects on blood and urine measures related to oxalate.
What the researchers hope to learn. To evaluate safety and tolerability after a single IV dose of YOLT-203; to measure pharmacokinetic parameters (Cmax, Tmax, AUC, t1/2); to assess pharmacodynamic markers including serum oxalate and glycolate and 24-hour urinary oxalate and glycolate; to monitor changes in kidney function (eGFR) and the occurrence of urinary calculi events.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Genetic
- YOLT-203
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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 informed consent 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
- Subjects with any of the following conditions will be excluded:
- 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:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 × upper limit of normal (ULN).
- Total bilirubin > 1.5 × ULN. Subjects diagnosed with Gilbert syndrome whose total bilirubin < 2 × ULN may be enrolled.
- International normalized ratio (INR) > 1.5 (subjects taking oral anticoagulants such as warfarin with INR < 3.5 are allowed to participate).
- Known history of HIV infection or evidence of HIV infection (positive HIV antibody); active or chronic hepatitis C virus (HCV) infection (positive HCV antibody and positive HCV RNA polymerase chain reaction) or hepatitis B virus (HBV) infection (positive hepatitis B surface antigen [HBsAg]).
- Estimated glomerular filtration rate (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 investigational 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 hypersensitivity reactions to oligonucleotides or lipid nanoparticles (LNPs).
- Unwilling to comply with contraception 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.).
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After giving informed consent, participants undergo screening and baseline assessments such as blood tests and multiple 24‑hour urine collections. Enrolled participants receive a single intravenous infusion of YOLT‑203 given over about one hour; dosing is assigned by age/weight cohort. After dosing, participants return for safety checks, blood samples for drug levels and serum oxalate/glycolate, repeat 24‑hour urine collections, kidney function tests, and monitoring for adverse events at scheduled visits through Week 52 (including Day 1, Day 2, Day 3, Week 2, Week 4, Week 8, Week 16, Week 24 and Week 52).
How the study is structured. Interventional, single-group, open-label Phase I trial. Participants receive one intravenous infusion of YOLT-203 given over about one hour. The study uses a single-dose, dose-escalation approach with adult fixed-dose cohorts (35 mg and 45 mg) and pediatric weight-based cohorts (0.45 mg/kg and 0.6 mg/kg, capped at 35 mg and 45 mg). Planned enrollment is 3 to 9 patients. Safety, blood tests, and 24-hour urine collections are scheduled at multiple time points through Week 52.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on primary hyperoxaluria
Important dates
- Study start
- August 2026 (estimated)
- Primary completion
- October 2027 (estimated)
- Study completion
- January 2028 (estimated)
- Target enrollment
- 9 participants (estimated)
- Last updated on registry
- Aug 20, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- YolTech Therapeutics Co., Ltd
- Collaborators
- RenJi Hospital
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Xiaoying MA 📞 +86 13372563535 ✉ xiaoyingma@yoltx.com
Participating sites (1)
- Renji Hospital, School of Medicine, Shanghai Jiao Tong UniversityRecruiting
Shanghai, Shanghai Municipality, 200127 · China
- Xia Qiang · +86 159671233432 · 9096@renji.com
Terms used on this page · explanations by MedicalUpdates.ai
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- dose-escalation
- Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
- informed consent form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- study drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- legal guardian
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- assent
- A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- Estimated glomerular filtration rate
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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