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OpenTrials
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NCT Number: NCT05644561

Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Generalized Myasthenia Gravis

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Generalized Myasthenia Gravis Autoimmune Diseases Autoimmune Diseases of the Nervous System Immune System Diseases Myasthenia Gravis Neoplasms Neoplasms by Site Nervous System Diseases Nervous System Neoplasms Neurodegenerative Diseases Neuromuscular Diseases Neuromuscular Junction Diseases Paraneoplastic Syndromes Paraneoplastic Syndromes, Nervous System gMG

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Research Site, Milan, Italy

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Ravulizumab

Drug

Ravulizumab will be administered by intravenous (IV) infusion.

Primary outcomes

  1. Serum Concentration of Ravulizumab

    Time frame: Day 1 predose through Week 18 predose

  2. Serum Free C5 Concentration

    Time frame: Day 1 predose through Week 18 predose

Secondary outcomes

  1. Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18

    Time frame: Baseline, Up to Week 18

  2. Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18

    Time frame: Baseline, Up to Week 18

  3. Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18

    Time frame: Baseline, Up to Week 18

  4. 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

    Time frame: Week 10, Up to Week 18

  5. Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18

    Time frame: Baseline, Up to Week 18

    Participants ≥8 years of age will be evaluated.

  6. Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18

    Time frame: Baseline, Up to Week 18

    Participants <8 years of age will be evaluated.

  7. Number of Participants With ≥5-point Reduction Compared to Baseline in the QMG Total Score Over Time Through Week 18

    Time frame: Baseline through Week 18

  8. Number of Participants With ≥3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18

    Time frame: Baseline through Week 18

  9. Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline

    Time frame: Baseline through Week 18

    Stable is defined as a ±5-point change from Baseline.

  10. Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline

    Time frame: Baseline through Week 18

    Stable is defined as a ±3-point change from baseline.

  11. Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events

    Time frame: Baseline up to Week 126 (8 weeks after last dose of study drug)

  12. Number of Participants With Anti-Drug Antibody (ADA) at Week 18

    Time frame: Baseline through Week 18

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

Phase 3, Open-label, Single-arm, Multicenter Study Evaluating Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants (6 to <18 Years) With Generalized Myasthenia Gravis (gMG)

Important dates

Study start
2023
Primary completion
2026
Study completion
2028
First posted
Dec 9, 2022
Registry last updated
Apr 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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