Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

Официальное название

Phase 3 Single-Arm Open-Label Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent Participants (Age 12 to <18 Years) With Homozygous Familial Hypercholesterolemia (SPRUCE)

Обзор

This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to \<18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and low-density lipoprotein cholesterol (LDL-C) ≥116 milligrams per deciliter (mg/dL) on maximally tolerated lipid-lowering therapy.

Вмешательства

• Препарат Zodasiran
  By sc injection

Первичные конечные точки

• Percent Change from Baseline to Month 12 in Fasting LDL-C [Срок оценки: Baseline, Month 12]

Вторичные конечные точки (12)

• Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB) [Срок оценки: Baseline, Month 12]
• Change from Baseline to Month 12 in Fasting ApoB [Срок оценки: Baseline, Month 12]
• Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C) [Срок оценки: Baseline, Month 12]
• Change from Baseline to Month 12 in Fasting Non-HDL-C [Срок оценки: Baseline, Month 12]
• Change from Baseline to Month 12 in Fasting LDL-C [Срок оценки: Baseline, Month 12]
• Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C [Срок оценки: Baseline, Month 12]
• Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs) [Срок оценки: Baseline, Month 12]
• Change from Baseline to Month 12 in Fasting TGs [Срок оценки: Baseline, Month 12]
• Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3) [Срок оценки: Baseline, Month 12]
• Change from Baseline to Month 12 in Fasting ANGPTL3 [Срок оценки: Baseline, Month 12]
• Percent Change from Baseline to Month 12 in Fasting Total Cholesterol [Срок оценки: Baseline, Month 12]
• Change from Baseline to Month 12 in Fasting Total Cholesterol [Срок оценки: Baseline, Month 12]

Критерии участия

Критерии включения

• Adolescents 12 to <18 years of age who are nonpregnant, nonlactating, and do not plan to become pregnant during the study
• Body weight ≥35 kilograms (kg) at screening
• HoFH based on a supportive genetic test (from a source-verifiable medical record or based on screening genotype) or clinical diagnosis
• Screening LDL-C ≥116 mg/dL (3 mmol/L)
• Screening hemoglobin A1c (HbA1c) ≤9.5%
• Total bilirubin <2×upper limit of normal (ULN), unless in previously confirmed cases of Gilbert's syndrome
• Alanine aminotransferase or aspartate aminotransferase <3×ULN

Критерии исключения

• Use of a hepatocyte-targeted siRNA within 365 days before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)
• Use of an antisense oligonucleotide molecule within 3 months before Day 1
• Use of evinacumab within 3 months before Day 1
• Non-response to evinacumab, defined as LDL-C reduction <15% from baseline after 2 doses
• Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen)

NOTE: Additional inclusion/exclusion criteria may apply per protocol

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07473843 · AROANG3-3003 · 2025-523662-24-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗