Clinical trial NCT06764160: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adults With gMG
Official title: An Open-label, Single-arm, Multi-center Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Participants With Refractory Generalized Myasthenia Gravis (gMG)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis (gMG)
Refractory gMG
Keywords: Generalized Myasthenia Gravis (gMG)
Refractory gMG
Eculizumab
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2025-01-08
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is an open-label, single-arm, multi-center study to evaluate the efficacy, safety, PK, PD, and immunogenicity of eculizumab in Chinese participants with refractory gMG. Approximately 15 participants will be enrolled in the study.

Detailed description: This post-approval study is an open-label, single-arm, multi-center study to evaluate the efficacy, safety, PK, PD, and immunogenicity of eculizumab in Chinese participants with refractory gMG. There will be 3 periods in this study: Screening Period (up to 4 weeks), Treatment Period (26 weeks, including an Induction Phase and a Maintenance Phase), and Safety Follow-up Period (8 weeks). The overall study duration for an individual participant is estimated to be up to 38 weeks. Approximately 15 participants will be enrolled in the study.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of MG must be made by the following tests:

  1. Positive serologic test for anti-AChR Abs as confirmed at Screening, and
  2. One of the following:

     1. Abnormal neuromuscular transmission demonstrated by repetitive nerve stimulation, or
     2. History of positive anticholinesterase test, eg, neostigmine test, or
     3. Participant has demonstrated improvement in MG signs on oral cholinesterase inhibitors as assessed by the treating physician
* MGFA Clinical Classification Class II to IV at Screening
* MG-ADL total score must be ≥ 6 at Screening and Day 1
* Participants who have:

  1. Failed treatment with 2 or more ISTs over one year (either in combination or as monotherapy), ie, continue to have impairment ADLs (persistent weakness, experience crisis, or unable to tolerate IST) despite ISTs or,
  2. Failed at least one IST and require chronic PE or IVIg to control symptoms, ie, participants who require PE or IVIg on a regular basis for the management of muscle weakness at least 2 cycles over last 12 months

Exclusion Criteria:

* Any untreated thymic malignancy, carcinoma, or thymoma
* History of thymectomy or any other thymic surgery within 6 months prior to Screening. Participants with a history of treated thymic malignancy or carcinoma are eligible if they meet all of the following conditions:

  1. Treatment completed \> 5 years prior to the Screening Visit
  2. No recurrence within the 5 years prior to the Screening Visit
  3. No radiological indication of recurrence in a computed tomography (CT) or magnetic resonance imaging (MRI) scan, including administration of intravenous (IV) contrast, performed within 6 months of first dose on Day 1
* Weakness only affecting ocular or peri-ocular muscles (MGFA Class I)
* MG crisis at Screening (MGFA Class V). However, such participants may be rescreened with Alexion approval once they are treated and medically stable, in the opinion of the Investigator
* History of N meningitidis infection or unresolved meningococcal disease

Primary outcomes:
measure: To assess the efficacy of eculizumab in the treatment of refractory gMG based on the improvement in the MG-ADL
timeFrame: from baseline at Week 26
Secondary outcomes:
measure: To assess the efficacy of eculizumab in the treatment of refractory gMG by QMG
timeFrame: from baseline at Week 26
measure: To assess the efficacy of eculizumab in the treatment of refractory gMG by MGC
timeFrame: from baseline at Week 26
measure: To assess the efficacy of eculizumab in the treatment of refractory gMG by MG-QoL 15r
timeFrame: from baseline at Week 26
measure: To assess the efficacy of eculizumab in the treatment of refractory gMG by MG-ADL responder status
timeFrame: from baseline at Week 26
measure: To assess the efficacy of eculizumab in the treatment of refractory gMG by QMG responder analysis
timeFrame: from baseline at Week 26
measure: To assess the safety and tolerability of eculizumab in the treatment of refractory gMG
timeFrame: from baseline at Week 26
measure: To characterize the PK of eculizumab in participants with refractory gMG
timeFrame: from baseline at Week 26
measure: To characterize the PD of eculizumab in participants with refractory gMG
timeFrame: from baseline at Week 26
measure: To characterize the immunogenicity of eculizumab in participants with refractory gMG
timeFrame: from baseline at Week 26
Other outcomes:
Locations:
city: Changchun
country: China
facility: Research Site
city: Changsha
country: China
facility: Research Site
city: Fuzhou
country: China
facility: Research Site
city: Guangzhou
country: China
facility: Research Site
city: Guangzhou
country: China
facility: Research Site
city: Qingdao
country: China
facility: Research Site
city: Shanghai
country: China
facility: Research Site
city: Shenzhen
country: China
facility: Research Site
city: Suzhou
country: China
facility: Research Site
city: Wuhan
country: China
facility: Research Site
Study officials:
References: