Ofatumumab in AQP4-IgG Seropositive NMOSD

Официальное название

Efficacy and Safety of Ofatumumab in AQP4-IgG Seropositive NMOSD: an Open-label, Single-arm, Multicentre Prospective Pilot Study

Обзор

This is an open-label, single-arm, multicentre prospective pilot study to assess the efficacy and safety of ofatumumab in patients with AQP4-IgG seropositive neuromyelitis optica spectrum disorder (NMOSD) in China.

Подробное описание

Neuromyelitis optica spectrum disorder (NMOSD) is a rare but severe demyelinating disorder that affects mainly adult patients. It is associated with a pathological B cell-mediated humoral immune response against the aquaporin-4 (AQP4) water channel. Monoclonal antibodies against CD20 have been shown to be effective for prevention of relapses in patients with NMOSD, and therefore been recommended as first-line therapy for this disorder. Ofatumumab (OFA), a fully humanized anti-CD20 monoclonal antibody, has been approved for multiple sclerosis treatment. However, prospective multicenter studies are needed to determine the efficacy and safety of ofatumumab in treating NMOSD.

Вмешательства

• Препарат Ofatumumab
  The enrolled patients will receive ofatumumab (20 mg/0.4 ml) subcutaneously administered at baseline, Day 7, Day 14 and monthly thereafter. Patients will receive ofatumumab therapy for a total of 48 weeks. The first 4 infusions will be administered at study center site; subsequent infusions will be given in the patient's home with a nurse online interview to administer the infusion.

Первичные конечные точки

• Change from baseline in annual relapse rate (ARR) at last follow-up visit [Срок оценки: baseline, 12 months]

Вторичные конечные точки (8)

• Change from baseline in Expanded Disability Status Scale (EDSS) score [Срок оценки: baseline, 3 months, 6 months, 9 months, 12 months]
• Change from baseline in lesion burden on MRI T2-weighted images [Срок оценки: baseline, 6 months, 12 months]
• Change from baseline in optic coherence tomography (OCT) measures [Срок оценки: baseline, 6 months, 12 months]
• Change from baseline in the frequencies of circulating B cell subsets [Срок оценки: baseline, 1 week, 2 weeks, 1 month, 3 months, 6 months, 9 months, 12 months]
• Change from baseline in immune landscape [Срок оценки: baseline, 1 week, 2 weeks, 1 month, 3 months, 6 months, 9 months, 12 months]
• Biochemical indicators monitoring [Срок оценки: baseline, 1 month, 3 months, 6 months, 9 months, 12 months]
• Assessment of functional questionnaire [Срок оценки: baseline, 1 month, 3 months, 6 months, 9 months, 12 months]
• Adverse events [Срок оценки: 1 week, 2 weeks, 1 month, 3 months, 6 months, 9 months, 12 months]

Критерии участия

Критерии включения

• Diagnosis of NMOSD according to the 2015 International Panel Diagnostic Criteria for NMOSD with AQP4-IgG.
• Clinical evidence of at least 2 relapses (including first attack) in past 24 months with at least 1 relapse occurring in the preceding 12 months.
• Adults aged ≥18 years old.
• Expanded disability status scale (EDSS) score between 0 and 7.5 (inclusive).
• Provision of written informed consent to participate in this study.
• Only oral corticosteroids were permitted at screening (≤10mg equivalent per day), which should be terminated within one month.
• Effective contraception was used for female patients with fertility during the treatment or at least 3 months after stopping medication.

Критерии исключения

• Progressive neurological deterioration unrelated to relapses of NMOSD, or presence of neurological findings suspected with PML.
• Pregnant or breastfeeding patients and those with family planning during the study period.
• Patients participating in any other clinical therapeutic study at the screening or within 30 days of screening.
• Patients with splenectomy or history of no spleen, and those with planned surgery (excluding minor surgery) during the study period.
• Presence of uncontrolled severe concurrent diseases; long-term glucocorticoids or immunosuppressants use due to other autoimmune diseases, or presence of other chronic diseases that cannot receiving immunosuppression.
• Active infection at within 4 weeks before baseline.
• Positive for HBV or HCV.
• Evidence of latent or active tuberculosis (TB).
• Have received any live or live-attenuated vaccine within 6 weeks before baseline.
• History of malignancy in past 5 years, including solid tumor, malignant hematopathy and carcinoma in situ.
• History of severe allergic reactions to biological agents.
• Inability to provide written informed consent.

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

Китай · 1 центр

• Tangdu Hospital — Сиань

Идентификаторы

NCT: NCT05504694 · K202206-13

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗