PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis

NCT ID: NCT06607627

Last Updated: 2026-02-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-11-13

Study Completion Date

2029-01-23

Brief Summary

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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.

Detailed Description

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Conditions

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Generalized Myasthenia Gravis gMG

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Gefurulimab

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

Group Type EXPERIMENTAL

Gefurulimab

Intervention Type COMBINATION_PRODUCT

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

Interventions

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Gefurulimab

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

Intervention Type COMBINATION_PRODUCT

Eligibility Criteria

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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.


* 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)


* 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
Minimum Eligible Age

6 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Research Site

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

Site Status RECRUITING

Research Site

Norfolk, Virginia, United States

Site Status RECRUITING

Research Site

Joinville, , Brazil

Site Status RECRUITING

Research Site

Salvador, , Brazil

Site Status RECRUITING

Research Site

São José do Rio Preto, , Brazil

Site Status RECRUITING

Research Site

São Paulo, , Brazil

Site Status RECRUITING

Research Site

São Paulo, , Brazil

Site Status RECRUITING

Research Site

Saitama-shi, , Japan

Site Status NOT_YET_RECRUITING

Research Site

Bydgoszcz, , Poland

Site Status NOT_YET_RECRUITING

Research Site

Lodz, , Poland

Site Status NOT_YET_RECRUITING

Research Site

Warsaw, , Poland

Site Status NOT_YET_RECRUITING

Research Site

New Taipei City, , Taiwan

Site Status RECRUITING

Research Site

Taipei, , Taiwan

Site Status RECRUITING

Countries

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United States Brazil Japan Poland Taiwan

Central Contacts

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Alexion Pharmaceuticals, Inc. (Sponsor)

Role: CONTACT

1-855-752-2356

Other Identifiers

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2024-515157-17-00

Identifier Type: OTHER

Identifier Source: secondary_id

ALXN1720-MG-302

Identifier Type: OTHER

Identifier Source: secondary_id

U1111-1327-6615

Identifier Type: OTHER

Identifier Source: secondary_id

D6780C00004

Identifier Type: -

Identifier Source: org_study_id

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