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

Primary Purpose

Generalized Myasthenia Gravis, gMG

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Ravulizumab
Sponsored by
Alexion
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Generalized Myasthenia Gravis focused on measuring Generalized Myasthenia Gravis, gMG

Eligibility Criteria

6 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Clinical Trial SiteRecruiting
  • Clinical Trial SiteRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Ravulizumab Intravenous (IV) Infusion

Arm Description

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.

Outcomes

Primary Outcome Measures

Plasma Concentration of Ravulizumab
Serum Free C5 Concentration of Ravulizumab

Secondary Outcome Measures

Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18
Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18
Change From Baseline in Myasthenia Gravis Composite (MGC) Score at 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
Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at 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
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
Number of Participants With ≥3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18
Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline
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
Stable is defined as a ±3-point change from baseline.
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
Number of Participants With Anti-Drug Antibody (ADA) at Week 18

Full Information

First Posted
December 1, 2022
Last Updated
October 9, 2023
Sponsor
Alexion
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1. Study Identification

Unique Protocol Identification Number
NCT05644561
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)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 9, 2023 (Actual)
Primary Completion Date
July 5, 2026 (Anticipated)
Study Completion Date
July 5, 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Generalized Myasthenia Gravis, gMG
Keywords
Generalized Myasthenia Gravis, gMG

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Ravulizumab Intravenous (IV) Infusion
Arm Type
Experimental
Arm Description
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 Type
Drug
Intervention Name(s)
Ravulizumab
Intervention Description
Ravulizumab will be administered by intravenous (IV) infusion.
Primary Outcome Measure Information:
Title
Plasma Concentration of Ravulizumab
Time Frame
Day 1 predose through Week 18 predose
Title
Serum Free C5 Concentration of Ravulizumab
Time Frame
Day 1 predose through Week 18 predose
Secondary Outcome Measure Information:
Title
Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18
Time Frame
Baseline, Up to Week 18
Title
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
Title
Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18
Time Frame
Baseline, Up to Week 18
Title
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
Title
Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18
Description
Participants ≥8 years of age will be evaluated.
Time Frame
Baseline, Up to Week 18
Title
Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18
Description
Participants <8 years of age will be evaluated.
Time Frame
Baseline, Up to Week 18
Title
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
Title
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
Title
Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline
Description
Stable is defined as a ±5-point change from Baseline.
Time Frame
Baseline through Week 18
Title
Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline
Description
Stable is defined as a ±3-point change from baseline.
Time Frame
Baseline through Week 18
Title
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)
Title
Number of Participants With Anti-Drug Antibody (ADA) at Week 18
Time Frame
Baseline through Week 18

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Alexion Pharmaceuticals, Inc.
Phone
1-855-752-2356
Email
clinicaltrials@alexion.com
Facility Information:
Facility Name
Clinical Trial Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Tokyo
ZIP/Postal Code
1738606
Country
Japan
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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.

Learn more about this trial

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

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