Primary Hyperoxaluria Type 1Phase 1NCT07776626
Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1)
A Phase 1 study testing YOLT-203 in people with Primary Hyperoxaluria Type 1 and PH1, run by YolTech Therapeutics Co., Ltd at 1 site in China.
Trial summary
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. 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?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- Aug 2026to Jan 2028
- Phase
- Phase 1
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
- 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.
Who cannot join
- 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.
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- 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:
- Lumasiran: less than 15 months from the last dose to administration of the study drug.
- 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.).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Renji Hospital, School of Medicine, Shanghai Jiao Tong University
Shanghai, Shanghai Municipality, 200127
Xia Qiang, +86 159671233432, 9096@renji.com
About this study
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.
- Conditions
- Primary Hyperoxaluria Type 1, PH1
- Being tested
- YOLT-203
- Keywords
- Primary Hyperoxaluria Type 1, PH 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, ph1
- Medical subject headings
- Primary hyperoxaluria type 1, Hyperoxaluria, Primary, Kidney Calculi, Hyperoxaluria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
YOLT-203Genetic
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.
| Study group | Role | What they receive |
|---|---|---|
| YOLT-203 Treatment Group | Experimental | 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. Receives: Genetic: YOLT-203 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Baseline, Day 1, Day 2, Day 3, Week 2, Week 4, Week 8, Week 16, Week 24, Week 52
Secondary outcomes (7)
Maximum observed plasma concentration (Cmax) of PK
Measured: 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.
Measured: Baseline to Week 52 post study drug administration
Change in estimated glomerular filtration rate (eGFR)
Measured: 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
Measured: Day 1 through Week 4 after study drug administration
Area under the plasma concentration-time curve (AUC) of PK
Measured: Day 1 through Week 4 after study drug administration
Terminal elimination half-life (t1/2) of PK
Measured: Day 1 through Week 4 after study drug administration
Incidence of urinary calculi events
Measured: Screening, Baseline, Day 1, Day 2, Day 3, Week 2, Week4, Week8 ,Week 16, Week 24, Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2026
- Study startAug 2026 (estimated)
- Primary completionOct 2027 (estimated)
- Study completionJan 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- YolTech Therapeutics Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- RenJi Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07776626
- Sponsor's protocol ID
- PH1-YOLT-203-1001
- Status last verified
- Aug 2026
- First submitted
- 5 Aug 2026
- Last updated
- 20 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No