Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)

Официальное название

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

Обзор

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.

Вмешательства

• Генная терапия YOLT-203
  YOLT-203 is an investigational gene editing therapy being evaluated for the treatment of Primary Hyperoxaluria Type 1 (PH1). It is administered by intravenous infusion over 1 hour.
• Препарат Placebo
  Placebo is a matching intravenous infusion administered over 1 hour.

Первичные конечные точки

• Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected) [Срок оценки: Months 2 to 6]

Вторичные конечные точки (7)

• Change From Baseline in Plasma Oxalate [Срок оценки: Months 2 to 6]
• Change in Estimated Glomerular Filtration Rate (eGFR) [Срок оценки: Baseline to Month 6]
• Change in Kidney Stone Burden [Срок оценки: Baseline to Month 6]
• Proportion of Participants Achieving Urinary Oxalate Thresholds [Срок оценки: Month 6]
• Incidence of Adverse Events [Срок оценки: Day 1 to Month 12]
• Pharmacokinetic Parameters of YOLT-203 (Cmax) [Срок оценки: Dosing through Month 1]
• Pharmacokinetic Parameters of YOLT-203 (AUC0-inf) [Срок оценки: Dosing through Month 1]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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).

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Перекрёстный дизайн

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07587021 · PH1-YOLT-203-2001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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