Actively Recruiting
Study to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
Led by Alexion Pharmaceuticals, Inc. · Updated on 2026-04-01
12
Participants Needed
13
Research Sites
110 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the pharmacokinetics (how the drug moves through the body) and pharmacodynamics (the drug's effects) of gefurulimab in children aged 6 to under 18 years who have generalized myasthenia gravis with acetylcholine receptor antibodies (AChR+ gMG). This open-label, single-arm Phase 3 study aims to understand how gefurulimab behaves in pediatric patients with this condition and to assess its safety and efficacy. Participants will receive a weight-based loading dose of gefurulimab on the first day, followed by weekly maintenance doses for up to 122 weeks. Gefurulimab is given as a combination product via subcutaneous injection. This regimen is designed to monitor the treatment's effects over an extended period while capturing safety data. During the study, children will have their blood tested to measure drug concentration and complement component 5 levels from Day 1 through Week 18. Researchers will also monitor adverse events and changes in disease severity using standardized scores through Week 18. Antibody responses to the drug will be tracked through Week 134. Participants will be regularly assessed for safety and efficacy over the study duration, which may last over two years.
CONDITIONS
Brief Title
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant must be 6 to < 18 years of age (12 to < 18 years in the USA) at the time of signing the informed consent/assent
- Must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y (or A, C, W, Y and B where available) within 3 years prior to study intervention
- Diagnosis of generalized myasthenia gravis with muscle weakness meeting Myasthenia Gravis Foundation of America Class II, III, or IV
- Positive serological test for acetylcholine receptor antibodies (AChR+)
You will not qualify if you...
- History of thymectomy or any thymic surgery within 12 months prior to screening
- Untreated thymic malignancy, carcinoma, or thymoma
- History of Neisseria meningitidis infection
- Pregnancy, breastfeeding, or intention to conceive during the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 122 weeks
Participants receive Gefurulimab, a combination product administered weekly via subcutaneous injection, starting with a weight-based loading dose on Day 1 followed by weekly maintenance doses.
Weekly visits for up to 122 weeks
Duration - Up to Week 134
Participants are monitored for safety and efficacy outcomes after completing treatment, including adverse events and antibody responses.
Visits as scheduled through Week 134
Trial Site Locations
Total: 13 locations
1
Research Site
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
2
Research Site
Norfolk, Virginia, United States, 23507
Actively Recruiting
3
Research Site
Joinville, Brazil, 89202-451
Actively Recruiting
4
Research Site
Salvador, Brazil, 41253-190
Actively Recruiting
5
Research Site
São José do Rio Preto, Brazil, 15090-000
Actively Recruiting
6
Research Site
São Paulo, Brazil, 0438-002
Actively Recruiting
7
Research Site
São Paulo, Brazil, 05403-000
Actively Recruiting
8
Research Site
Saitama-Shi, Japan, 330-8777
Actively Recruiting
9
Research Site
Bydgoszcz, Poland, 85-065
Withdrawn
10
Research Site
Lodz, Poland, 93-338
Withdrawn
11
Research Site
Warsaw, Poland, 02-097
Actively Recruiting
12
Research Site
New Taipei City, Taiwan, 23561
Actively Recruiting
13
Research Site
Taipei, Taiwan, 11101
Actively Recruiting
Research Team
A
Alexion Pharmaceuticals, Inc. (Sponsor)
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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