Clinical trial NCT05644561: Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)
Official title: A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
gMG
Keywords: Generalized Myasthenia Gravis
gMG
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2022-12-09
Enrollment: 12
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.


Eligibility criteria: Inclusion Criteria:

* Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period
* Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening
* Participants receiving treatment must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period.
* Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days) and must have been on a stable dose for ≥ 2 months (60 days) prior to Screening.
* All participants must be vaccinated against meningococcal infection

Exclusion Criteria:

Medical Conditions

* Any untreated thymic malignancy, carcinoma, or thymoma.
* Participants with a history of treated benign thymoma
* History of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to Screening
* History of N meningitidis infection
* Known to be human immunodeficiency virus (HIV) positive
* History of unexplained infections
* Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to the start of the Screening Period

Primary outcomes:
measure: Serum Concentration of Ravulizumab
timeFrame: Day 1 predose through Week 18 predose
measure: Serum Free C5 Concentration
timeFrame: Day 1 predose through Week 18 predose
Secondary outcomes:
measure: Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18
timeFrame: Baseline, Up to Week 18
measure: Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18
timeFrame: Baseline, Up to Week 18
measure: Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18
timeFrame: Baseline, Up to Week 18
measure: Change in Status from Week 10 in Myasthenia Gravis Foundation of America Postintervention Status (MGFA-PIS) as Assessed by the Investigator or Neurologist at Up to Week 18
timeFrame: Week 10, Up to Week 18
measure: Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18
timeFrame: Baseline, Up to Week 18
measure: Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18
timeFrame: Baseline, Up to Week 18
measure: Number of Participants With ≥5-point Reduction Compared to Baseline in the QMG Total Score Over Time Through Week 18
timeFrame: Baseline through Week 18
measure: Number of Participants With ≥3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18
timeFrame: Baseline through Week 18
measure: Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline
timeFrame: Baseline through Week 18
measure: Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline
timeFrame: Baseline through Week 18
measure: Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
timeFrame: Baseline up to Week 126 (8 weeks after last dose of study drug)
measure: Number of Participants With Anti-Drug Antibody (ADA) at Week 18
timeFrame: Baseline through Week 18
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Research Site
state: California
city: San Francisco
country: United States
facility: Research Site
state: California
city: Chicago
country: United States
facility: Research Site
state: Illinois
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: Chapel Hill
country: United States
facility: Research Site
state: North Carolina
city: Akron
country: United States
facility: Research Site
state: Ohio
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Denton
country: United States
facility: Research Site
state: Texas
city: Marseille
country: France
facility: Research Site
city: Paris
country: France
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Torino
country: Italy
facility: Research Site
city: Itabashi-ku
country: Japan
facility: Research Site
city: Leiden
country: Netherlands
facility: Research Site
city: Belgrade
country: Serbia
facility: Research Site
city: Belgrade
country: Serbia
facility: Research Site
city: Madrid
country: Spain
facility: Research Site
city: Malaga
country: Spain
facility: Research Site
city: Bern
country: Switzerland
facility: Research Site
Study officials:
References: