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Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1)

ConditionsPrimary Hyperoxaluria Type 1PH1
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age6+
SponsorYolTech Therapeutics Co., Ltd

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

None

Disqualifiers

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).

Trial design

Design model

Single group

Treatments tested in this trial

  • YOLT-203

    Genetic

    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.

Treatment groups

9 Participants
are divided into 1 treatment group
Group A: YOLT-203 Treatment GroupExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

Secondary outcomes

1

Maximum observed plasma concentration (Cmax) of PK

Time frame
Day 1 through Week 4 after study drug administration
2

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
3

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
4

Time to reach maximum concentration (Tmax) of PK

Time frame
Day 1 through Week 4 after study drug administration

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

YolTech Therapeutics Co., Ltd

Lead sponsor

RenJi Hospital

Collaborator

Source ClinicalTrials.gov
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