Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

Официальное название

A Multicenter, Single Arm, Open-label Study to Evaluate Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With aHUS

Обзор

The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in study participants with aHUS.

Подробное описание

The study is designed as a multicenter, single-arm, open label study to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in participants with aHUS. It consists of a screening period of up to 14 weeks followed by a 12-Month Core Treatment period and 12-Month Extension Treatment period.

The study will assess the effects of iptacopan on a range of efficacy assessments relevant to aHUS.

Вмешательства

• Препарат Iptacopan
  Open Label

Первичные конечные точки

• Percentage of participants free of TMA manifestation [Срок оценки: 12 months]

Вторичные конечные точки (12)

• Percentage of participants free of TMA manifestation in study participants with functionally significant mutations in complement genes or positive anti FH antibodies [Срок оценки: 12 months, 24 months]
• Percentage of participants free of TMA manifestation [Срок оценки: 24 months]
• Time to TMA manifestation [Срок оценки: 12 months, 24 months]
• Change from baseline in platelets [Срок оценки: Baseline, month 12, month 24]
• Change from baseline in LDH [Срок оценки: Baseline, month 12, month 24]
• Change from baseline in hemoglobin [Срок оценки: Baseline, month 12, month 24]
• Change from baseline in serum creatinine [Срок оценки: Baseline, month 12, month 24]
• Change from baseline in UPCR [Срок оценки: Baseline, month 12, month 24]
• Change from baseline in eGFR [Срок оценки: Baseline, month 12, month 24]
• Change from baseline in CKD stage [Срок оценки: Baseline, month 12, month 24]
• Number of participants who require dialysis [Срок оценки: month 12 and month 24]
• Percentage of participants with TMA related events. [Срок оценки: month 12 and month 24]

Критерии участия

Критерии включения

• Male and female adult participants ≥ 18 years of age with diagnosis of aHUS for whom etiologies of other types of TMA and non-aHUS kidney disease have been excluded.

•. Currently on the recommended (as per label) dosage regimen of anti-C5 antibody treatment, for at least 3 months prior to entering the screening period.

• In the opinion of the investigator the participant has responded to anti-C5 antibodytreatment prior to screening and has clinical evidence of response (in absence of PE/PI) during the Screening period.

Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as:

• Hematological normalization in platelet count ≥150 x 10\^9/L and LDH below upper limit of normal \[ULN\], and
• Stable kidney function as defined by serum creatinine values within ±15% during the Screening period

• Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan.
• If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations.

Критерии исключения

• History of aHUS disease relapse while on anti-C5 antibody treatment.
• eGFR < 30 ml/min/1.73m\^2
• Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae.
• Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration.
• Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation
• Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study
• Any medical condition deemed likely to interfere with the patient's participation in the study

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

Франция · 6 центров

• Novartis Investigative Site — Bordeaux
• Novartis Investigative Site — Paris
• Novartis Investigative Site — Paris
• Novartis Investigative Site — Rouen
• Novartis Investigative Site — Toulouse
• Novartis Investigative Site — Tours

Испания · 6 центров

• Novartis Investigative Site — Santiago Compostela
• Novartis Investigative Site — Barcelona
• Novartis Investigative Site — Córdoba
• Novartis Investigative Site — Málaga
• Novartis Investigative Site — Seville
• Novartis Investigative Site — Valencia

Китай · 4 центра

• Novartis Investigative Site — Нанкин
• Novartis Investigative Site — Пекин
• Novartis Investigative Site — Пекин
• Novartis Investigative Site — Шанхай

Turkey (Türkiye) · 4 центра

• Novartis Investigative Site — Izmir
• Novartis Investigative Site — Köseköy
• Novartis Investigative Site — Ankara
• Novartis Investigative Site — Mersin

Италия · 3 центра

• Novartis Investigative Site — Ranica
• Novartis Investigative Site — Milan
• Novartis Investigative Site — Roma

Япония · 3 центра

• Novartis Investigative Site — Matsumoto-shi
• Novartis Investigative Site — Iruma-gun
• Novartis Investigative Site — Bunkyo Ku

Великобритания · 3 центра

• Novartis Investigative Site — Glasgow
• Novartis Investigative Site — Newcastle upon Tyne
• Novartis Investigative Site — London

Германия · 2 центра

• Novartis Investigative Site — Essen
• Novartis Investigative Site — Kiel

Идентификаторы

NCT: NCT05935215 · CLNP023F12302

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗