Study of Ianalumab in Adults With Primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) Who Have Previously Benefited From Ianalumab

Официальное название

A Phase 2 Study of Ianalumab in Adults With Primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) Who Have Previously Benefited From Ianalumab.

Обзор

This study is intended to explore the efficacy and safety of a second course of ianalumab after experiencing treatment failure in the pivotal Primary Immune Thrombocytopenia (ITP) trials (CVAY736I12301, CVAY736Q12301) and after loss of durable response in the pivotal Warm Autoimmune Hemolytic Anemia (wAIHA) trial (CVAY736O12301).

Подробное описание

This is a multicenter, Phase 2 exploratory study to assess the efficacy and safety of a second course of ianalumab in adults with ITP and wAIHA who have previously benefited from ianalumab.

All participants will receive ianalumab at the dose of study treatment from which they previously derived benefit.

After completion of the screening period, participants who do not meet treatment failure criteria during treatment period will enter the safety and efficacy follow-up, whereas participants who reported a treatment failure during the treatment period will enter the safety follow-up only.

The study will end once all participants have completed safety and/or efficacy follow-up since their last dose of ianalumab or discontinued the study earlier.

Вмешательства

• Биопрепарат Ianalumab
  Concentrate for solution for infusion for intravenous use

Первичные конечные точки

• Treatment failure free (yes, no) for participants with Immune Thrombocytopenia (ITP) [Срок оценки: From treatment start date until the events indicative of treatment failure (up to 2 years after last dose)]
• Durable response for participants with Warm Autoimmune Hemolytic Anemia (wAIHA) [Срок оценки: At least 8 consecutive weeks, between week 9 and week 25]

Вторичные конечные точки (10)

• Response rate in participants with ITP only [Срок оценки: from treatment start to end of study (up to 2 years after last dose)]
• Complete response rate in participants with ITP only [Срок оценки: from treatment start to end of study (up to 2 years after last dose)]
• Number and percentage of participants receiving new ITP therapy only [Срок оценки: from treatment start to end of study (up to 2 years after last dose)]
• Response and complete response in participants with wAIHA only [Срок оценки: from treatment start to end of study (up to 2 years after last dose)]
• Number and percentage of participants receiving new wAIHA therapy only [Срок оценки: from treatment start to end of study (up to 2 years after last dose)]
• Number and percentage of participants receiving rescue treatment [Срок оценки: from treatment start to end of study (up to 2 years after last dose)]
• Number of severe infections and percentage of participants with severe infection [Срок оценки: from treatment start to end of study (up to 2 years after last dose)]
• Ianalumab concentration in serum [Срок оценки: after first dose (pre dose 0 and 672 hours) and at study end (up to 2 years after last dose of Ianalumab)]
• Incidence of anti-ianalumab antibodies in serum (ADA assay) over time [Срок оценки: up to end of treatment (Week 16), end of study (up to 2 years after last dose of Ianalumab)]
• Titer of anti-ianalumab antibodies in serum (ADA assay) over time [Срок оценки: up to end of treatment (Week 16), end of study (up to 2 years after last dose of Ianalumab)]

Критерии участия

Критерии включения

• Signed informed consent obtained prior to participation in the study.
• Male or female participants aged 18 years and older on the day of signing informed consent

Primary ITP patients:

• Previously enrolled and treated either with ianalumab/placebo in addition to first-line corticosteroids on protocol CVAY736I12301 or with ianalumab/placebo in addition to eltrombopag in the second line on protocol CVAY736Q12301, and who experienced treatment failure (TF) by parent trial definition ≥ 2 years after the last infusion of ianalumab/placebo
• Rescue medication and/or bridging therapy are allowed to be started within the 28 days prior to screening; platelet count results obtained prior to the start of the therapy must be used to assess eligibility and have to be collected within 30 days prior to screening

For Primary or Secondary wAIHA patients:

• Previously documented by a positive direct antiglobulin test (DAT) specific for anti-IgG or anti-IgA, previously enrolled and treated with ianalumab/placebo in blinded cohort or placebo followed by crossover to open label ianalumab in protocol CVAY736O12301, having experienced durable response lasting beyond 2 years from the last infusion of ianalumab/placebo in blinded cohorts or a durable response beyond week 20 from last dose of first course of ianalumab in the crossover arm.
• Relapsed wAIHA with hemoglobin concentration ≥5 g/dL and <10 g/dL and presence of symptoms related to anemia during screening or within 14 days before screening window or within 28 days before screening window if rescue medication/bridging therapy has been initiated.
• Rescue medication and/or bridging therapy are allowed to be started during the screening and within 28 days prior to screening; hemoglobin level result for eligibility assessment needs to be obtained prior to the start of the treatment within 30 days prior to screening
• Supportive care is allowed in the case the participant received it in the parent trial when the relapse occurred and has remained stable at least 4 weeks prior screening

Критерии исключения

• Evans syndrome or any cytopenia other than thrombocytopenia (for ITP participants) or anemia (for wAIHA), except for grade 1 anemia due to blood loss or iron deficiency.
• Secondary wAIHA with BM involvement for wAIHA patients
• Current life-threatening bleeding or history of life-threatening bleeding due to thrombocytopenia
• Therapy for ITP or wAIHA other than ianalumab/placebo, bridging/rescue therapies and supportive care prior to the beginning of the screening window
• After primary analysis of each respective parent trial, participants whose treatment was unblinded and who received placebo only will be excluded.
• ITP participants only: Participants with concurrent coagulation disorders and/or receiving anti-platelet or anti-coagulant medication except for low dose of acetylsalicylic acid (≤150 mg per day)

Other protocol-defined inclusion/exclusion criteria may apply.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Италия · 4 центра

• Novartis Investigative Site — Florence
• Novartis Investigative Site — Roma
• Novartis Investigative Site — Trieste
• Novartis Investigative Site — Vicenza

Китай · 3 центра

• Novartis Investigative Site — Гуанчжоу
• Novartis Investigative Site — Dalian
• Novartis Investigative Site — Тяньцзинь

Малайзия · 3 центра

• Novartis Investigative Site — George Town
• Novartis Investigative Site — Johor Bahru
• Novartis Investigative Site — Kuala Selangor

Испания · 3 центра

• Novartis Investigative Site — Barcelona
• Novartis Investigative Site — Madrid
• Novartis Investigative Site — Murcia

Франция · 2 центра

• Novartis Investigative Site — Caen
• Novartis Investigative Site — Rennes

Turkey (Türkiye) · 2 центра

• Novartis Investigative Site — Samsun
• Novartis Investigative Site — Ankara

Аргентина · 1 центр

• Novartis Investigative Site — Buenos Aires

Бельгия · 1 центр

• Novartis Investigative Site — Roeselare

Чехия · 1 центр

• Novartis Investigative Site — Ostrava

Германия · 1 центр

• Novartis Investigative Site — Jena

Венгрия · 1 центр

• Novartis Investigative Site — Debrecen

Румыния · 1 центр

• Novartis Investigative Site — Bucharest

Сингапур · 1 центр

• Novartis Investigative Site — Singapore

Великобритания · 1 центр

• Novartis Investigative Site — London

Идентификаторы

NCT: NCT07039422 · CVAY736Q12202B · 2024-518231-11-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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