Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

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

Tracking Information

• First Submitted Date: December 1, 2022
• First Posted Date: December 9, 2022
• Last Update Posted Date: March 26, 2024
• Actual Study Start Date: June 24, 2023
• Estimated Primary Completion Date: July 15, 2026 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 1, 2022)

• Plasma Concentration of Ravulizumab [ Time Frame: Day 1 predose through Week 18 predose ]
• Serum Free C5 Concentration of Ravulizumab [ Time Frame: Day 1 predose through Week 18 predose ]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: December 1, 2022)

• Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18 [ Time Frame: Baseline, Up to Week 18 ]
• 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 ]
• Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18 [ Time Frame: Baseline, Up to Week 18 ]
• 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 ]
• 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.
• 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.
• 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 ]
• 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 ]
• 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.
• 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.
• 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) ]
• Number of Participants With Anti-Drug Antibody (ADA) at Week 18 [ Time Frame: Baseline through Week 18 ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)
• 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)
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Generalized Myasthenia Gravis
• gMG

Intervention

Drug: Ravulizumab

Ravulizumab will be administered by intravenous (IV) infusion.

Study Arms

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.

Intervention: Drug: Ravulizumab

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: December 1, 2022): 12
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: July 31, 2028
• Estimated Primary Completion Date: July 15, 2026 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No

Contacts

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

• Listed Location Countries: France, Italy, Japan, Netherlands, Serbia, Spain, Switzerland, United States

Administrative Information

• NCT Number: NCT05644561
• Other Study ID Numbers: ALXN1210-MG-319
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

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
• Supporting Materials: Statistical Analysis Plan (SAP)
• Supporting Materials: Clinical Study Report (CSR)

• Current Responsible Party: Alexion Pharmaceuticals, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Alexion Pharmaceuticals, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Alexion Pharmaceuticals, Inc.
• Verification Date: March 2024