A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Gravis (gMG)
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ClinicalTrials.gov Identifier: NCT03759366 |
Recruitment Status :
Active, not recruiting
First Posted : November 30, 2018
Last Update Posted : April 13, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Myasthenia Gravis Myasthenia Gravis, Juvenile Form Myasthenia Gravis, Generalized | Drug: Eculizumab | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 11 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 |
Actual Primary Completion Date : | January 6, 2022 |
Estimated Study Completion Date : | July 31, 2025 |

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. |
- 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) ]
- 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) ]
- 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) ]
- 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) ]
- 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) ]
- 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)] ]
- 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)] ]
- Pharmacokinetics: Plasma Concentration Of Eculizumab Over Time [ Time Frame: Baseline through Week 26 ]
- Free Complement Component 5 (C5) Concentrations Over Time [ Time Frame: Baseline through Week 26 ]
- Percentage Of Hemolysis (In Vitro Assay) Over Time [ Time Frame: Baseline through Week 26 ]

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

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
United States, California | |
Clinical Trial Site | |
Los Angeles, California, United States, 90027 | |
United States, Florida | |
Clinical Trial Site | |
Tampa, Florida, United States, 33612 | |
United States, Illinois | |
Clinical Trial Site | |
Chicago, Illinois, United States, 60611 | |
United States, Missouri | |
Clinical Trial Site | |
Columbia, Missouri, United States, 65211 | |
Clinical Trial Site | |
Saint Louis, Missouri, United States, 63104 | |
United States, North Carolina | |
Clinical Trial Site | |
Chapel Hill, North Carolina, United States, 27599-1651 | |
United States, Ohio | |
Clinical Trial Site | |
Akron, Ohio, United States, 44308 | |
United States, Pennsylvania | |
Clinical Trial Site | |
Philadelphia, Pennsylvania, United States, 19104 | |
United States, South Carolina | |
Clinical Trial Site | |
Charleston, South Carolina, United States, 29425-8572 | |
Japan | |
Clinical Trial Site | |
Chiba-shi, Chiba-Ken, Japan, 260-8677 | |
Clinical Trial Site | |
Saitama, Iruma-gun, Japan, 350-0495 | |
Clinical Trial Site | |
Tokyo, Itabasha-ku, Japan, 173-0003 | |
Clinical Trial Site | |
Tokyo, Shinjuku-ku, Japan, 162-8666 | |
Clinical Trial Site | |
Mibu, Tochigi, Japan, 3210293 | |
Netherlands | |
Clinical Trial Site | |
Amsterdam, Netherlands, 1105 | |
Clinical Trial Site | |
Leiden, Netherlands, 2333 |
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: | April 13, 2022 |
Last Verified: | April 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Myasthenia Gravis Myasthenia Gravis, Generalized |
Myasthenia Gravis Muscle Weakness Muscular Diseases Musculoskeletal Diseases Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Pathologic Processes Paraneoplastic Syndromes, Nervous System Nervous System Neoplasms Neoplasms by Site Neoplasms |
Paraneoplastic Syndromes Autoimmune Diseases of the Nervous System Neurodegenerative Diseases Neuromuscular Junction Diseases Neuromuscular Diseases Autoimmune Diseases Immune System Diseases Eculizumab Complement Inactivating Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |