Health TrialsbyHealthPanel

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

ConditionsGeneralized Myasthenia GravisgMG
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age6-17
SponsorAlexion Pharmaceuticals, Inc.

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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)

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • Gefurulimab

    Combination product

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

Treatment groups

12 Participants
are divided into 1 treatment group
Group A: GefurulimabExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Maximum Observed Serum Concentration (Cmax) of Gefurulimab

Time frame
Day 1 predose through Week 18 predose
2

Serum Free Complement Component 5 (C5) Concentration

Time frame
Day 1 predose through Week 18 predose

Secondary outcomes

1

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

Time frame
Day 1 through Week 134
2

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

Time frame
Baseline through Week 18
3

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

Time frame
Baseline through Week 18
4

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

Time frame
Baseline through Week 18

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Alexion Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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