Recruiting
Phase 3

Gefurulimab

Sponsor:

Alexion Pharmaceuticals, Inc.

Code:

NCT06607627

Conditions

Generalized Myasthenia Gravis

gMG

Eligibility Criteria

Sex: All

Age: 6 - 17

Healthy Volunteers: Not accepted

Interventions

Gefurulimab

Study Details

Brief summary:

The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.

Conditions

Generalized Myasthenia Gravis

gMG

Study ID

NCT06607627

Start date

Nov 13, 2024

Status verified date

Mar, 2026

Completion date

Jan 23, 2029

Anticipated

Primary completion date

Dec 15, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6 - 17

Healthy Volunteers: Not accepted

Inclusion Criteria:

United States of America (USA) specific inclusion criterion:

  • Participant must be 12 to < 18 years of age at the time of signing the informed consent/assent.
  • All participants must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y within 3 years and at least 2 weeks prior to the first dose of study intervention administration.

Rest of World (ROW) specific inclusion criteria:

  • Participant must be 6 to < 18 years of age at the time of signing the informed consent/assent.
  • All participants must be vaccinated against meningococcal infection from serogroups A, C, W, Y (and B where available) within 3 years prior to study intervention on Day 1. If vaccination occurs < 2 weeks prior to Day 1, the participants will receive prophylactic antibiotics for at least 2 weeks after initial meningococcal vaccination for serogroups A,C,W,Y (and B, where available)

Global inclusion criteria:

  • Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV
  • Positive serological test for autoantibodies against AChR

Exclusion Criteria:

  • History of thymectomy, or any other 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 course of the study

Study Design

Enrollment

12 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Gefurulimab

On Day 1, participants will receive a weight based loading dose followed by a weekly maintenance dose for up to 122 weeks.

Interventions

Gefurulimab

Combination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection.

Primary outcome measure

  • Maximum Observed Serum Concentration (Cmax) of Gefurulimab [ Time Frame: Day 1 predose through Week 18 predose ]
  • Serum Free Complement Component 5 (C5) Concentration [ Time Frame: Day 1 predose through Week 18 predose ]

Central Contacts and Locations

Central contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

1-855-752-2356clinicaltrials@alexion.com

Locations

Research Site

Recruiting

Washington D.C., District of Columbia, United States, 20010

Research Site

Recruiting

Norfolk, Virginia, United States, 23507

More Information

Sponsor

Alexion Pharmaceuticals, Inc.

Last update posted

Apr 1, 2026

Last verified

Mar, 2026

Keywords

  • Acetylcholine Receptor antibodies
  • AChR+
  • Generalized Myasthenia Gravis
  • gMG
  • ALXN1720
  • anti-acetylcholine receptor antibody-positive
  • acetylcholine receptor
  • AChR
  • myasthenia gravis
  • MG
  • complement component 5
  • C5
  • VHH antibody
  • pediatric

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc. on 2026-04-01.