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

NCT06607627 | Recruiting Phase 3 FDA Drug FDA Device

• 4 other identifiers: D6780C000042024-515157-17-00ALXN1720-MG-302U1111-1327-6615
• Study Type: interventional
• Target: 12
• Locations: 5 countries
• Sites: 13

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

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

Outcome Measures

Primary Outcomes (2)

• Maximum Observed Serum Concentration (Cmax) of Gefurulimab

  Day 1 predose through Week 18 predose

• Serum Free Complement Component 5 (C5) Concentration

  Day 1 predose through Week 18 predose

Secondary Outcomes (8)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

  Day 1 through Week 134

• Change in Quantitative Myasthenia Gravis Score for Disease Severity (QMG) Total Score From Baseline Through Week 18

  Baseline through Week 18

• Number of Participants With ≥ 5-point Reduction From Baseline in the QMG Total Score Through Week 18

  Baseline through Week 18

• Change in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score From Baseline Through Week 18

  Baseline through Week 18

• Number of Participants With ≥ 3-point Reduction From Baseline in the MG-ADL Total Score Through Week 18

  Baseline through Week 18

Study Arms (1)

Gefurulimab

EXPERIMENTAL

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

Combination Product: Gefurulimab

Interventions

Gefurulimab COMBINATION_PRODUCT

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

Gefurulimab

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (13)

Research Site

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

RECRUITING

Research Site

Norfolk, Virginia, 23507, United States

RECRUITING

Research Site

Joinville, 89202-451, Brazil

RECRUITING

Research Site

Salvador, 41253-190, Brazil

RECRUITING

Research Site

São José do Rio Preto, 15090-000, Brazil

RECRUITING

Research Site

São Paulo, 0438-002, Brazil

RECRUITING

Research Site

São Paulo, 05403-000, Brazil

RECRUITING

Research Site

Saitama-Shi, 330-8777, Japan

RECRUITING

Research Site

Bydgoszcz, 85-065, Poland

WITHDRAWN

Research Site

Lodz, 93-338, Poland

WITHDRAWN

Research Site

Warsaw, 02-097, Poland

RECRUITING

Research Site

New Taipei City, 23561, Taiwan

RECRUITING

Research Site

Taipei, 11101, Taiwan

RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2024
• First Posted: September 23, 2024
• Study Start: November 13, 2024
• Primary Completion (Estimated): December 15, 2026
• Study Completion (Estimated): January 23, 2029
• Last Updated: April 1, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

US (2); PL (3); BR (5); JP (1); TW (2)