Evaluating eculizumab for treating refractory generalized myasthenia gravis in Chinese adults

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)

This study is testing if a medication called eculizumab can help Chinese adults with hard-to-treat generalized myasthenia gravis feel better and stay safe while using it.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Alexion Pharmaceuticals, Inc. (industry)
• Drugs / interventions: eculizumab
• Locations: 10 sites (Changchun and 9 other locations)
• Trial ID: NCT06764160 on ClinicalTrials.gov

What this trial studies

This open-label, single-arm, multi-center study aims to assess the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of eculizumab in Chinese adults diagnosed with refractory generalized myasthenia gravis (gMG). The study will involve approximately 15 participants and will be conducted over a total duration of up to 38 weeks, including a screening period, a 26-week treatment period, and an 8-week safety follow-up. Participants will be monitored for their response to the treatment and any potential side effects throughout the study.

Who should consider this trial

Good fit: Ideal candidates for this study are Chinese adults diagnosed with refractory generalized myasthenia gravis who have failed multiple immunosuppressive therapies.

Not a fit: Patients with mild generalized myasthenia gravis or those who have not yet tried immunosuppressive therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new effective treatment option for patients suffering from refractory generalized myasthenia gravis.

How similar studies have performed: Other studies have shown success with eculizumab in treating generalized myasthenia gravis, indicating a promising approach for this patient population.

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

Where this trial is running

Changchun and 9 other locations

• Research Site — Changchun, China (NOT_YET_RECRUITING)
• Research Site — Changsha, China (NOT_YET_RECRUITING)
• Research Site — Fuzhou, China (NOT_YET_RECRUITING)
• Research Site — Guangzhou, China (NOT_YET_RECRUITING)
• Research Site — Guangzhou, China (NOT_YET_RECRUITING)
• Research Site — Qingdao, China (NOT_YET_RECRUITING)
• Research Site — Shanghai, China (RECRUITING)
• Research Site — Shenzhen, China (NOT_YET_RECRUITING)
• Research Site — Suzhou, China (NOT_YET_RECRUITING)
• Research Site — Wuhan, China (NOT_YET_RECRUITING)

Study contacts

• Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)

Conditions: Generalized Myasthenia Gravis, Refractory gMG, Eculizumab