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Trial record 1 of 1 for:    1210-MG-319
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Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

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ClinicalTrials.gov Identifier: NCT05644561
Recruitment Status : Recruiting
First Posted : December 9, 2022
Last Update Posted : March 26, 2024
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals, Inc.

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

Condition or disease Intervention/treatment Phase
Generalized Myasthenia Gravis gMG Drug: Ravulizumab Phase 3

Study Design
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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: A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG)
Actual Study Start Date : June 24, 2023
Estimated Primary Completion Date : July 15, 2026
Estimated Study Completion Date : July 31, 2028

Resource links provided by the National Library of Medicine

Drug Information available for: Ravulizumab

Arms and Interventions
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Arm Intervention/treatment
Experimental: Ravulizumab Intravenous (IV) Infusion
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.
 Drug: Ravulizumab
Ravulizumab will be administered by intravenous (IV) infusion.


Outcome Measures
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Primary Outcome Measures :
  1. Plasma Concentration of Ravulizumab [ Time Frame: Day 1 predose through Week 18 predose ]
  2. Serum Free C5 Concentration of Ravulizumab [ Time Frame: Day 1 predose through Week 18 predose ]

Secondary Outcome Measures :
  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 ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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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): NCT05644561


Contacts
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Contact: Alexion Pharmaceuticals, Inc. (Sponsor) 1-855-752-2356 clinicaltrials@alexion.com

Locations
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United States, California
Research Site Recruiting
Los Angeles, California, United States, 90078
Research Site Not yet recruiting
San Francisco, California, United States, 94143
United States, Illinois
Research Site Not yet recruiting
Chicago, Illinois, United States, 60611
United States, North Carolina
Research Site Recruiting
Chapel Hill, North Carolina, United States, 27514
United States, Ohio
Research Site Recruiting
Akron, Ohio, United States, 44308
United States, Pennsylvania
Research Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
France
Research Site Recruiting
Marseille, France, 13385
Research Site Recruiting
PARIS Cedex 12, France, 75571
Italy
Research Site Recruiting
Milano, Italy, 20133
Research Site Recruiting
Roma, Italy, 00165
Research Site Recruiting
Torino, Italy, 10126
Japan
Research Site Recruiting
Itabashi-ku, Japan, 173-0003
Netherlands
Research Site Not yet recruiting
Leiden, Netherlands, 2333 ZA
Serbia
Research Site Recruiting
Belgrade, Serbia, 11000
Research Site Recruiting
Belgrade, Serbia, 11070
Spain
Research Site Recruiting
Madrid, Spain, 28040
Research Site Recruiting
Malaga, Spain, 29010
Switzerland
Research Site Recruiting
Bern, Switzerland, 3010
Sponsors and Collaborators
Alexion Pharmaceuticals, Inc.
More Information
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Responsible Party: Alexion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT05644561    
Other Study ID Numbers: ALXN1210-MG-319
First Posted: December 9, 2022    Key Record Dates
Last Update Posted: March 26, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)

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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, Inc.:
Generalized Myasthenia Gravis
gMG
Additional relevant MeSH terms:
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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
Ravulizumab
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs