Clinical trial NCT06607627: PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
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+)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
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
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2024-09-23
Enrollment: 12
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Maximum Observed Serum Concentration (Cmax) of Gefurulimab
timeFrame: Day 1 predose through Week 18 predose
measure: Serum Free Complement Component 5 (C5) Concentration
timeFrame: Day 1 predose through Week 18 predose
Secondary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
timeFrame: Day 1 through Week 134
measure: Change in Quantitative Myasthenia Gravis Score for Disease Severity (QMG) Total Score From Baseline Through Week 18
timeFrame: Baseline through Week 18
measure: Number of Participants With ≥ 5-point Reduction From Baseline in the QMG Total Score Through Week 18
timeFrame: Baseline through Week 18
measure: Change in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score From Baseline Through Week 18
timeFrame: Baseline through Week 18
measure: Number of Participants With ≥ 3-point Reduction From Baseline in the MG-ADL Total Score Through Week 18
timeFrame: Baseline through Week 18
measure: Number of Participants With Anti-drug Antibodies (ADAs)
timeFrame: Baseline through Week 134
measure: Number of ADA Positive Participants With Pre-existing Immunoreactivity,Treatment-emergent or Treatment-boosted ADA Responses
timeFrame: Baseline through Week 134
measure: Number of ADA Positive Participants With Neutralizing Antibody (NAb) Positive or Negative Status
timeFrame: Baseline through Week 134
Other outcomes:
Locations:
city: Washington
country: United States
facility: Research Site
state: District of Columbia
city: Norfolk
country: United States
facility: Research Site
state: Virginia
city: Joinville
country: Brazil
facility: Research Site
city: Salvador
country: Brazil
facility: Research Site
city: Sao Jose Do Rio Preto
country: Brazil
facility: Research Site
city: Sao Paulo
country: Brazil
facility: Research Site
city: São Paulo
country: Brazil
facility: Research Site
city: Bydgoszcz
country: Poland
facility: Research Site
city: Warszawa
country: Poland
facility: Research Site
city: Łódź
country: Poland
facility: Research Site
city: Taipei
country: Taiwan
facility: Research Site
city: Taipei
country: Taiwan
facility: Research Site
Study officials:
References: