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Recruiting NCT06607627

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

Phase III Interventional Generalized Myasthenia Gravis gMG

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Gefurulimab.
Who it may be relevant to
Registry conditions: Generalized Myasthenia Gravis, gMG. Basic parameters: 6 years — 17 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Brazil, Japan, Poland, Taiwan
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)

Overview

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.

Interventions

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

Primary outcome measures

  • 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]
Secondary outcome measures (8)
  • Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) [Time frame: Day 1 through Week 134]
  • Change in Quantitative Myasthenia Gravis Score for Disease Severity (QMG) Total Score From Baseline Through Week 18 [Time frame: Baseline through Week 18]
  • Number of Participants With ≥ 5-point Reduction From Baseline in the QMG Total Score Through Week 18 [Time frame: Baseline through Week 18]
  • Change in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score From Baseline Through Week 18 [Time frame: Baseline through Week 18]
  • Number of Participants With ≥ 3-point Reduction From Baseline in the MG-ADL Total Score Through Week 18 [Time frame: Baseline through Week 18]
  • Number of Participants With Anti-drug Antibodies (ADAs) [Time frame: Baseline through Week 134]
  • Number of ADA Positive Participants With Pre-existing Immunoreactivity,Treatment-emergent or Treatment-boosted ADA Responses [Time frame: Baseline through Week 134]
  • Number of ADA Positive Participants With Neutralizing Antibody (NAb) Positive or Negative Status [Time frame: Baseline through Week 134]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Brazil · 5 centers
  • Research Site — Joinville
  • Research Site — Salvador
  • Research Site — São José do Rio Preto
  • Research Site — São Paulo
  • Research Site — São Paulo
Poland · 3 centers
  • Research Site — Bydgoszcz
  • Research Site — Lodz
  • Research Site — Warsaw
United States · 2 centers
  • Research Site — Washington D.C.
  • Research Site — Norfolk
Taiwan · 2 centers
  • Research Site — New Taipei City
  • Research Site — Taipei
Japan · 1 center
  • Research Site — Saitama-Shi

Identifiers

NCT: NCT06607627 · D6780C00004 · 2024-515157-17-00 · ALXN1720-MG-302 · U1111-1327-6615

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗