Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

Alexion Pharmaceuticals

Status and phase: Enrolling; Phase 3

Conditions: gMG; Generalized Myasthenia Gravis

Treatments: Drug: Ravulizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT05644561

ALXN1210-MG-319

Details and patient eligibility

About

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

Enrollment: 12 estimated patients

Sex: All

Ages: 6 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

Ravulizumab Intravenous (IV) Infusion

Experimental group

Description:

All participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing \< 20 kg, for a total of 122 weeks of treatment.

Treatment:

Drug: Ravulizumab