Clinical trial NCT06724809: Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD
Official title: An Open-label, Single-arm, Multi-Center, Interventional Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adult Participants With Anti-Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorders (NMOSD)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: NMOSD
Neuromyelitis Optica Spectrum Disorders
Keywords: NMOSD
Neuromyelitis Optica Spectrum Disorders
eculizumab
anti-AQP4
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
Collaborators: name: AstraZeneca
First posted: 2024-12-09
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Maximum age (years): 130
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of Eculizumab in Chinese Adults with Neuromyelitis Optica Spectrum Disorders (NMOSD).


Eligibility criteria: Inclusion Criteria:

* Participants with diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria
* Anti-AQP4 antibody positive
* At least 1 attack or relapse in the last 12 months prior to the Screening Period
* EDSS score ≤ 7
* If a participant enters the study receiving IST(s) for relapse prevention, the participant must be on a stable maintenance dose of IST(s) as follows, prior to screening and must remain on that dose for the duration of the study, unless the participant experiences a relapse
* Female participants of childbearing potential must have a negative pregnancy test (serum HCG at screening
* Male participants are eligible to participate if they agree to the following during the study intervention Treatment Period and for at least 5 months after the last dose of study intervention:

  * Refrain from donating fresh unwashed semen. PLUS, either,
  * Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent. OR
  * Must agree to use barrier as detailed below:

    * Agree to use a male condom when having sexual intercourse with a WOCBP who is not currently pregnant.

Exclusion Criteria:

* Pregnant, breastfeeding, or intending to conceive during the course of the study
* Prior history of N meningitidis infection or unresolved meningococcal disease
* Any systemic bacterial or other infection which is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics
* Presence of fever ≥ 38 C within 7 days prior to study intervention administration on Day 1
* Hypersensitivity to murine proteins or to one of the excipients of study intervention
* Use of rituximab, inebilizumab, or other B cell-depleting therapy within 6 months prior to Day 1 and during the study
* Use of mitoxantrone or satralizumab within 3 months prior to screening and during the study
* Use of IVIg within 3 weeks prior to screening
* If a participant enters the study receiving oral corticosteroid(s) with or without other ISTs, the daily corticosteroid dose must be no more than prednisone 20 mg/day (or equivalent) prior to screening and the participant must remain on that dose for the duration of the study or until the participant experiences a relapse (specific medications listed in Section 6.9.1 may be allowed)
* Has previously received treatment with C5 inhibitors
* Participation in any other investigational drug study or exposure to an investigational drug or device within 5 half-lives of treatment (if known) or 30 days, which is longer, before the first dose administration

Primary outcomes:
measure: The efficacy of eculizumab in anti-AQP4 antibody positive participants with NMOSD measured by Adjudicated On-trial annualized relapse rate (ARR).
timeFrame: Baseline through Week 52
measure: The efficacy of eculizumab in anti-AQP4 antibody positive participants with NMOSD measured by Adjudicated On-trial annualized relapse rate (ARR).
timeFrame: Baseline through Week 52
Secondary outcomes:
measure: The efficacy of eculizumab by assessing change from baseline to end of study in Expanded Disability Status Scale (EDSS) Score
timeFrame: Baseline through Week 52
measure: The efficacy of eculizumab by assessing change from baseline to end of study in Ambulatory Function as Measured by Hauser Ambulation Index (HAI)
timeFrame: Baseline through Week 52
measure: The efficacy of eculizumab by assessing change from baseline to end of study in European Quality of Life 5-Dimension Questionnaire (EQ-5D) Index Score
timeFrame: Baseline through Week 52
measure: Change From Baseline to end of study in EQ-5D Visual Analogue Scale (VAS) Score
timeFrame: Baseline through Week 52
measure: Serum Eculizumab Concentrations Over Time
timeFrame: Baseline through Week 52
measure: Serum Free Complement Component 5 (C5) Concentrations Over Time
timeFrame: Baseline through Week 52
measure: Number of Treatment-emergent Antidrug Antibody (ADA) Positive Participants
timeFrame: Baseline through Week 52
measure: To characterize the overall safety of eculizumab in the treatment of NMOSD
timeFrame: Baseline through Week 52
Other outcomes:
Locations:
city: Beijing
country: China
facility: Research Site
city: Dongguan
country: China
facility: Research Site
city: Jinan
country: China
facility: Research Site
city: Shanghai
country: China
facility: Research Site
city: Shenyang
country: China
facility: Research Site
city: Taiyuan
country: China
facility: Research Site
city: Wenzhou City
country: China
facility: Research Site
city: Wuhan
country: China
facility: Research Site
city: Wuhan
country: China
facility: Research Site
city: Zhengzhou
country: China
facility: Research Site
Study officials:
affiliation: Huashan Hospital
name: Xiangjun Chen, M.D
role: PRINCIPAL_INVESTIGATOR
References: