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Type 1 Primary HyperoxaluriaEarly Phase 1PH1NCT06892301

Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)

A Early Phase 1 study testing YOLT-203 in people with Type 1 Primary Hyperoxaluria, run by Guangzhou Women and Children's Medical Center at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This study is a single-arm, open-label, single-dose, dose-escalation trial, aiming to evaluate the safety and tolerability of YOLT-203 in the Chinese population with type 1 primary hyperoxaluria (PH1); and to preliminarily assess the effect of a single dose of YOLT-203 on the plasma oxalate level.In this study, the… Read more about this study

Who is it for?
Ages 2 to 18 years
Conditions the study is for
Type 1 Primary Hyperoxaluria
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
2 participants
Where
1 siteChina
Study dates
27 Feb 2025to Jul 2026
Phase
Early Phase 1
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 2 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • The age is 2≤ years <18 years old at the time of signing the informed consent.
  • Have AGXT gene mutations and be diagnosed with primary hyperoxaluria (PH1); eGFR ≥ 30 ml/min/1.73m2.
  • At least 2 times of 24-hour urinary oxalate excretion ≥ 0.7 mmol/1.73m2/ day or the ratio of urinary oxalate to creatinine in a single urine collection must be higher than the upper limit of normal (ULN) for the corresponding age.
  • If treated with vitamin B6, the treatment has been stable for 90 days before enrollment in the study and is willing to maintain the stable treatment plan unchanged during the study.
  • The patient himself/herself or the guardian voluntarily signs the informed consent.

Who cannot join

  • The investigator judges that there is clinical evidence of systemic extrarenal oxalate deposition.
  • Have any of the following laboratory parameter assessment results at screening:
  • Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 x the upper limit of normal (ULN).
  • Total bilirubin > 1.5 x ULN. If the increase in total bilirubin is caused by diagnosed Gilbert's syndrome and the total bilirubin < 2 x ULN, it is eligible.
  • International normalized ratio (INR) > 1.5 (Patients on oral anticoagulants [such as warfarin] and with INR < 3.5 will be allowed to participate).
  • Known to have active human immunodeficiency virus (HIV) infection; or have evidence of current or chronic hepatitis C virus (HCV) or hepatitis B virus (HBV) infection.
Show all 17Show fewer
  • The estimated glomerular filtration rate (GFR) at screening is less than 30 mL/min/1.73m² (For patients ≥ 18 years old, it will be calculated according to the Modification of Diet in Renal Disease [MDRD] formula; for patients < 18 years old, it will be calculated according to the Schwartz bedside formula). See the attachment.
  • Have received an investigational drug within the last 30 days or 5 halflives (whichever is longer) before the first administration of the study drug, or have participated in the follow-up of another clinical study before randomization.
  • Have a history of kidney or liver transplantation.
  • According to the investigator's opinion, have other medical conditions or comorbidities that may interfere with study compliance or data interpretation.
  • Page 4 of 5 [DRAFT] -
  • Have a history of multiple drug allergies or allergic reaction history to oligonucleotides or LNP.
  • Have a history of subcutaneous injection intolerance.
  • Unwilling to comply with contraceptive requirements throughout the study participation period until 6 months after the end of the main study trial.
  • Female patients are pregnant, planning to become pregnant or breastfeeding.
  • Unwilling or unable to limit alcohol consumption throughout the study. Alcohol consumption during the study exceeds 2 units per day (1 unit: approximately 125 ml of wine = approximately 29 ml of spirits = approximately 284 ml of beer, will be excluded.
  • The investigator believes that there is a history of alcohol abuse within 12 months before screening.

Trial locations and contacts

1 site in 1 country.

China1 site

  • Guangzhou Women and Children's Medical Center

    Guangzhou, Guangdong, 510623

Other trials in China

About this study

This study is a single-arm, open-label, single-dose, dose-escalation trial, aiming to evaluate the safety and tolerability of YOLT-203 in the Chinese population with type 1 primary hyperoxaluria (PH1); and to preliminarily assess the effect of a single dose of YOLT-203 on the plasma oxalate level.In this study, the maximum screening period of the main study is 60 days, the treatment day is Day 1 (D1), and the safety follow-up period is up to Week 52 after administration. In addition, subjects within the first dose group can voluntarily receive a second treatment with the test drug at the effective dose level. After the end of the main study, the subjects will undergo long-term followup. According to the requirements of the "Technical Guidelines for Long-Term Follow-up Clinical Studies of Gene Therapy Products (Trial)" issued by the CDE, the long-term follow-up is up to 15 years after administration. The most updated protocol is V1.2 , 22 Jan 2025

Being tested
YOLT-203
Medical subject headings
Primary hyperoxaluria type 1

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • YOLT-203Drug

    The IP is administered intravenously at the predetermined dose.

Study groups in NCT06892301
Study groupRoleWhat they receive
Open Label single doseExperimentalYOLT-203
Receives: Drug: YOLT-203

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of Treatment-Emergent Adverse events

    An Adverse Event(AE) is any untoward medical occurrence in a participant or clinical investigational participant adminstered a medicinal product and which does not necessarily have a casual relationship with this treatment

    Measured: through week 52

Secondary outcomes (8)

  1. The Peak Plasma Concentration (Cmax) of YOLT-203

    The Peak Plasma Concentration (Cmax) of YOLT-203 in the plasma after a dose is given

    Measured: through Day 14

  2. Area under the plasma concentration versus time curve (AUC)of YOLT-203

    Area under the plasma concentration versus time curve (AUC). 0.5 predose,6,24,48,144,and312 hours after treatment

    Measured: through Day 14

  3. Time to Maximum Plasma Concentration (Tmax)of YOLT-203

    Tmax is the time it takes for YOLT-203 to reach the maximun concentration (Tmax) after administration

    Measured: through Day 14

  4. Drug half-life (T1/2)of YOLT-203

    The amount of time it takes for the plasma YOLT-203 drug concentration to drop to half

    Measured: through Day 14

  5. The changes in blood glycolic acid levels

    After medication, at 1, 2, 4, 8, 16, 24, 36, and 52 weeks, the changes in blood glycolic acid levels

    Measured: through week 52

  6. The changes in 24-hour urinary oxalic acid excretion

    After medication, at 1,2,4, 8, 16, 24, 36and 52 weeks, the changes in 24-hour urinary oxalic acid excretion compared to the baseline value.

    Measured: through week 52

  7. The changes in 24-hour urinary glycolic acid excretion

    After medication, at 1, 2, 4, 8, 16, 24, 36, and 52 weeks, the changes in 24-hour urinary glycolic acid excretion

    Measured: through week 52

  8. The changes in eGFR

    After medication, at 1, 2, 4, 8, 16, 24, 36, and 52 weeks, the changes in eGFR.

    Measured: through week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Mar 2025
  2. Study start27 Feb 2025
  3. Primary completionJul 2026 (estimated)
  4. Study completionJul 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
0.45mg/kg
Masking
None (open label)
Enrolment
2 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06892301
Sponsor's protocol ID
PH1-YOLT-203-01
Acronym
PH1
Status last verified
Feb 2025
First submitted
26 Feb 2025
Last updated
24 Mar 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing YOLT-203 in people with Type 1 Primary Hyperoxaluria, run by Guangzhou Women and Children's Medical Center at 1 site in China.

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 17 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: under way, not looking for participants, as of the sponsor's last update on 24 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. 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. 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?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Treatment-Emergent Adverse events (time frame: through week 52). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06892301.

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 24 Mar 2025.

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