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A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Gravis (gMG)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03759366
Recruitment Status : Recruiting
First Posted : November 30, 2018
Last Update Posted : August 19, 2020
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of eculizumab in the treatment of pediatric refractory gMG based on change from Baseline in the Quantitative Myasthenia Gravis (QMG) score for disease severity.

Condition or disease Intervention/treatment Phase
Myasthenia Gravis Myasthenia Gravis, Juvenile Form Myasthenia Gravis, Generalized Drug: Eculizumab Phase 3

Detailed Description:
The study will consist of an up to 4-week Screening Period, 26-week Primary Evaluation Treatment Period, an additional (up to) to 208-week Extension Period, and an 8-week Safety Follow-up Period.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients With Refractory Generalized Myasthenia Gravis
Actual Study Start Date : December 21, 2018
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : July 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Eculizumab

Arm Intervention/treatment
Experimental: Eculizumab Intravenous (IV) Infusion

In the Primary Evaluation Treatment Period (26 weeks), eculizumab will be administered weekly during the initial induction phase and every 2 weeks during the maintenance phase.

In the Extension Period (up to 208 weeks), participants will continue to receive eculizumab every 2 weeks.

Eculizumab will be administered at doses of 300, 600, 900, or 1200 milligrams (mg), based on the participant's current body weight.

Drug: Eculizumab
Eculizumab will be administered by IV infusion.




Primary Outcome Measures :
  1. Change From Baseline In The QMG Total Score Over Time Regardless Of Rescue Treatment [ Time Frame: Baseline, up to Week 26 (Primary Evaluation Period) and Week 208 (Extension Period) ]

Secondary Outcome Measures :
  1. Change From Baseline In The Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Score Over Time Regardless Of Rescue Treatment [ Time Frame: Baseline, up to Week 26 (Primary Evaluation Period) and Week 208 (Extension Period) ]
  2. Change From Baseline In The MG Composite (MGC) Score Over Time Regardless Of Rescue Treatment [ Time Frame: Baseline, up to Week 26 (Primary Evaluation Period) and Week 208 (Extension Period) ]
  3. Change From Baseline In European Quality Of Life 5-Dimension Youth (EQ-5D-Y) Score Over Time Regardless Of Rescue Treatment [ Time Frame: Baseline, up to Week 26 (Primary Evaluation Period) and Week 208 (Extension Period) ]
  4. Change From Baseline In Neurological Quality Of Life (Neuro-QoL) Pediatric Fatigue Score Over Time Regardless Of Rescue Treatment [ Time Frame: Baseline, up to Week 26 (Primary Evaluation Period) and Week 208 (Extension Period) ]
  5. Myasthenia Gravis Foundation Of America (MGFA) Post-Interventional Status Over Time Regardless Of Rescue Treatment [ Time Frame: Baseline through Week 26 (Primary Evaluation Period) and Week 208 (Extension Period)] ]
  6. Number Of Participants With Clinical Deteriorations, Myasthenic Crises, And Rescue Therapy Use Over Time [ Time Frame: Baseline through Week 26 (Primary Evaluation Period) and Week 208 (Extension Period)] ]
  7. Pharmacokinetics: Plasma Concentration Of Eculizumab Over Time [ Time Frame: Baseline through Week 26 ]
  8. Free Complement Component 5 (C5) Concentrations Over Time [ Time Frame: Baseline through Week 26 ]
  9. Percentage Of Hemolysis (In Vitro Assay) Over Time [ Time Frame: Baseline through Week 26 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   6 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female pediatric participants 6 to <18 years of age at time of assent/consent.
  • Vaccinated against Neisseria meningitidis.
  • Documented vaccination against Haemophilus influenzae and Streptococcus pneumoniae infections prior to dosing as per local and country specific immunization guidelines for the appropriate age group.
  • Diagnosis of MG confirmed by positive serologic test for anti-acetylcholine receptor antibodies at Screening, and 1 of the following: (a) history of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography or repetitive nerve stimulation; (b) history of positive anticholinesterase test (for example, edrophonium chloride or neostigmine test); or (c) participant demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors, as assessed by the Investigator.
  • Presence of refractory gMG, defined as participants with gMG who have 1 or more of the following: (a) failed treatment ≥1 year with at least 1 immunosuppressive therapies (IST), defined as follows: (1) persistent weakness with impairment of activities of daily living; (2) myasthenia gravis (MG) exacerbation and/or crisis while on treatment; or (3) intolerance to ISTs due to side effect or comorbid condition(s). (b) Require maintenance plasma exchange (PE) or intravenous immunoglobulin (IVIg) to control symptoms; and/or (c) in the opinion of the Investigator, MG poses a significant functional burden despite current MG treatment.
  • MGFA Clinical Classification of Class II to IV at Screening.
  • In patients aged 12 to 18 years, QMG total score ≥ 12 at Screening; in patients aged 6 to 11 years, no minimum QMG is required for inclusion; however, patients must have documented limb weakness in at least one limb.
  • All MG-specific treatment has been administered at a stable dosing regimen of adequate duration prior to Screening.

Exclusion Criteria:

  • Parent or legal guardian is an Alexion employee.
  • Any active or untreated thymoma. History of thymic carcinoma or thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥5 years before Screening.
  • History of thymectomy within 12 months prior to Screening.
  • Are pregnant or lactating.
  • Any unresolved acute, or chronic, systemic bacterial or other infection, which is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics.
  • Use of PE within 4 weeks prior to first dose.
  • Use of rituximab within 6 months prior to first dose.
  • Patients who are under 15 kg and are receiving maintenance IVIg.
  • Participation in another interventional treatment study or use of any experimental therapy within 30 days before initiation of study drug on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater.
  • Have previously received treatment with eculizumab or other complement inhibitors.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03759366


Contacts
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Contact: Alexion Pharmaceuticals Inc. 855-752-2356 clinicaltrials@alexion.com

Locations
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United States, California
Children's Hospital Los Angeles, Division of Neurology Recruiting
Los Angeles, California, United States, 90027
United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33612
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
United States, Missouri
University of Missouri-Columbia Recruiting
Columbia, Missouri, United States, 65211
St. Louis University/SSM Health Cardinal Glennon Children's Hospital Recruiting
Saint Louis, Missouri, United States, 63104
United States, North Carolina
University of North Carolina at Chapel Hill Recruiting
Chapel Hill, North Carolina, United States, 27599-1651
United States, Ohio
Akron Children's Hospital Recruiting
Akron, Ohio, United States, 44308
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425-8572
Japan
Chiba University Hospital Recruiting
Chiba-shi, Chiba-Ken, Japan, 260-8677
Saitama Medical University Recruiting
Saitama, Iruma-gun, Japan, 350-0495
Teikyo University Recruiting
Tokyo, Itabasha-ku, Japan
Tokyo Women's Medical University Hospital Recruiting
Tokyo, Shinjuku-ku, Japan, 162-8666
Dokkyo Medical University Hospital Recruiting
Mibu, Tochigi, Japan, 3210293
Sponsors and Collaborators
Alexion Pharmaceuticals
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03759366    
Other Study ID Numbers: ECU-MG-303
First Posted: November 30, 2018    Key Record Dates
Last Update Posted: August 19, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion Pharmaceuticals:
Myasthenia Gravis
Myasthenia Gravis, Generalized
Additional relevant MeSH terms:
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Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases