Study of Ravulizumab in Pediatric Participants With Primary IgAN

Официальное название

A Phase 3, Open-Label, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Ravulizumab in Pediatric Participants (2 to < 18 Years of Age) With Primary Immunoglobulin A Nephropathy (IgAN)

Обзор

The primary objectives of this study are to characterize ravulizumab pharmacokinetics (PK) and pharmacodynamics (PD), and to evaluate safety and efficacy following ravulizumab IV dosing in pediatric participants with IgAN or IgAVN.

Вмешательства

• Препарат Ravulizumab
  Participants will receive Ravulizumab via intravenous (IV) infusion.

Первичные конечные точки

• Change from Baseline in Proteinuria Based on Urine Protein to Creatinine Ratio (UPCR) at Week 34 [Срок оценки: Baseline, Week 34]

Вторичные конечные точки (11)

• Maximum Observed Plasma Concentration (Cmax) of Ravulizumab [Срок оценки: Baseline up to Week 34]
• Trough Serum Concentration (Ctrough) of Ravulizumab [Срок оценки: Baseline up to Week 34]
• Change From Baseline in Serum Free Complement Component 5 (C5) Concentration [Срок оценки: Baseline up to Week 34]
• Change from Baseline in proteinuria based on Urine Protein to Creatinine Ratio (UPCR) at Week 10 [Срок оценки: Baseline, Week 10]
• Change from Baseline in Albuminuria based on Urine Albumin to Creatinine Ratio (UACR) at Week 34 [Срок оценки: Baseline, Week 34]
• Number of Participants with Partial Remission [Срок оценки: Week 34]
• Annualized Total Estimated Glomerular Filtraion Rate (eGFR) over 106 weeks [Срок оценки: Baseline up to Week 106]
• Change from Baseline in eGFR [Срок оценки: Baseline, Weeks 50 and 106]
• Number of Participants with UPCR <0.5 gram of protein per gram of creatinine [Срок оценки: Week 34]
• Number of Participants With Treatment Emergent Adverse Events, Treatment Emergent Serious Adverse Events and Adverse Events of Special Interest [Срок оценки: Baseline up to Week 106]
• Number of Participants with Antidrug Antibodies to Ravulizumab and Neutralizing Antibodies [Срок оценки: Baseline up to Week 106]

Критерии участия

Критерии включения

• Participant must be 2 to < 18 years of age at the time of signing the informed consent or assent.
• Stable and maximum allowed or tolerated RAASI (ACEI and/or ARB) dose for ≥ 3 months prior to Screening with no planned change during Screening through Week 106.
• UPCR ≥ 1.0 g/g from the mean of 3 first morning voids (FMV) collected within 1 week during the Screening Period
• Estimated GFR ≥ 30 mL/min/1.73 m2 during Screening
• Meningococcal infection vaccine
• Haemophilus influenzae type b and Streptococcus pneumoniae vaccine
• Participants who are receiving SGLT2i, DEARA (eg, sparsentan), MRA, ERA, or GLP-1 agonists must be on a stable and maximum allowed or tolerated dose for ≥ 3 months prior to Screening with no planned change in dose through Week 34.
• Established diagnosis of primary IgAN diagnosis based on kidney biopsy within 3 years prior to Screening or during the Screening Period

Критерии исключения

• Diagnosis of rapidly progressive glomerulonephritis
• Secondary forms of IgAN not in the context of primary IgAN or IgAV
• Concomitant clinically significant renal disease other than IgAN or IgAVN
• Clinical remission of IgAN/IgAVN or clinically significant improvement in proteinuria within the last 6 months.
• Uncontrolled diabetes mellitus with HbA1c > 8.5%
• History of kidney transplant or planned kidney transplant during the Primary Evaluation Period.
• History of other solid organ (heart, lung, small bowel, pancreas, or liver) or bone marrow transplant
• Splenectomy or functional asplenia
• Participants with nephrotic syndrome receiving albumin infusions or with acute kidney injury requiring dialysis within the last 6 months prior to Screening.
• Hemolytic uremic syndrome diagnosed any time prior to Screening.
• Planned urological surgery expected to influence kidney function within the study time frame.
• Congenital immunodeficiency
• Active systemic bacterial, viral, or fungal infection within 14 days prior to enrollment
• Received biologics for the treatment of IgAN or IgAVN within≤ 6 months prior to Screening

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

Испания · 4 центра

• Research Site — Almería
• Research Site — Barcelona
• Research Site — Barcelona
• Research Site — Seville

Италия · 3 центра

• Research Site — Genova
• Research Site — Roma
• Research Site — Torino

США · 2 центра

• Research Site — Palo Alto
• Research Site — Aurora

Китай · 2 центра

• Research Site — Пекин
• Research Site — Шанхай

Тайвань · 2 центра

• Research Site — Taipei
• Research Site — Taoyuan

Япония · 1 центр

• Research Site — Wakayama

South Korea · 1 центр

• Research Site — Seoul

Идентификаторы

NCT: NCT07024563 · D928FC00002 · 2024-520167-13-00 · ALXN1210-IgAN-325

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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