A Randomized, Double-blind, Placebo-controlled Study Followed by a Treatment Extension to Evaluate the Efficacy and Safety of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 36
- 试验地点
- 4
- 主要终点
- Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected)
研究概览
简要总结
This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1.
After the initial randomized, 6-month double-blind, placebo-controlled period, participants who were initially assigned to placebo will receive a single-dose of YOLT-203 treatment whereas participants in the YOLT-203 group will receive a single-dose of placebo infusion.
For patients interested in additional information on how to participate, please follow this link: https://mytomorro.ws/ph1trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •You are 6 years old or older.
- •You have been diagnosed with PH1 (primary hyperoxaluria type 1) through genetic testing.
- •Urine tests show that your oxalate levels are not within certain limits (based on at least two 24-hour urine collections).
- •If you take vitamin B6, your dose has been stable for at least 3 months, and you are willing to keep it the same during the study.
- •You (and/or your parent or legal guardian, if you are under 18) understand the study and agree to follow all study requirements, including signing a consent form.
排除标准
- •You have signs that oxalate has affected parts of your body outside the kidneys.
- •Your liver blood tests are not within certain limits.
- •You have certain abnormal blood clotting results (unless controlled with medication like warfarin).
- •You have HIV, or active or chronic hepatitis C.
- •You are pregnant, planning to become pregnant, or breastfeeding.
- •You are not willing to use birth control during the study and for 6 months after (if applicable).
- •You are unable to limit alcohol intake to no more than about 2 drinks per day.
- •You have had alcohol or drug abuse in the past year.
- •You are unable to follow standard treatments for PH1 (such as drinking plenty of fluids, taking prescribed medications like potassium citrate, or following dietary advice).
研究组 & 干预措施
Placebo
Participants will receive both study treatments in sequential treatment periods according to randomized assignment. Participants who initially receive placebo during the double-blind treatment period and will cross over to the YOLT-203 treatment in the subsequent treatment period, followed by safety and efficacy follow-up.
干预措施: Placebo (Drug)
YOLT-203
Participants will receive both study treatments in sequential treatment periods according to randomized assignment. Participants who initially receive YOLT-203 during the double-blind treatment period and will cross over to the placebo treatment in the subsequent treatment period, followed by safety and efficacy follow-up.
干预措施: YOLT-203 (Genetic)
结局指标
主要结局
Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected)
时间窗: Months 2 to 6
Percent change from baseline in 24-hour urinary oxalate excretion corrected for body surface area (BSA) across Months 2 to 6.
次要结局
- Incidence of Adverse Events(Day 1 to Month 12)
- Pharmacokinetic Parameters of YOLT-203 (Cmax)(Dosing through Month 1)
- Proportion of Participants Achieving Urinary Oxalate Thresholds(Month 6)
- Change From Baseline in Plasma Oxalate(Months 2 to 6)
- Change in Estimated Glomerular Filtration Rate (eGFR)(Baseline to Month 6)
- Change in Kidney Stone Burden(Baseline to Month 6)
- Pharmacokinetic Parameters of YOLT-203 (AUC0-inf)(Dosing through Month 1)