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出境医 / 临床实验 / Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis

Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis

Study Description
Brief Summary:
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).

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

Study Design
Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 175 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: All investigative site personnel, sponsor staff, sponsor designees, staff directly associated with the conduct of the study, and all participants will be blinded to treatment assignments.
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis
Actual Study Start Date : March 26, 2019
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2023
Arms and Interventions
Arm Intervention/treatment
Experimental: Ravulizumab
Participants will receive ravulizumab for the duration of the study.
 Biological: Ravulizumab
Concentrated sterile, preservative-free aqueous solution (10 milligrams [mg]/milliliter [mL]) in single-use, 30-mL vial for intravenous (IV) infusion. Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.
Other Names:
  • ALXN1210
  • Ultomiris
Placebo Comparator: Placebo
Participants will receive placebo during the 26-week randomized-controlled period of the study, after which they will enter the open-label extension period of the study and receive ravulizumab.
 Drug: Placebo
Matching, sterile, preservative-free aqueous solution in single-use, 30-mL vial for IV infusion. Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.
Outcome Measures
Primary Outcome Measures :
  1. Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26 [ Time Frame: Baseline, Week 26 ]
    To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the MG-ADL profile.


Secondary Outcome Measures :
  1. Change From Baseline In The Quantitative Myasthenia Gravis (QMG) Total Score At Week 26 [ Time Frame: Baseline, Week 26 ]
    To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the QMG total score.

  2. Change From Baseline In the Revised 15 Component Myasthenia Gravis Quality of Life (MG-QOL15r) At Week 26 [ Time Frame: Baseline, Week 26 ]
    To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the MG-QOL15r score

  3. Change From Baseline In The Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score of at least 3 points At Week 26 [ Time Frame: Baseline, Week 26 ]
    To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the MG-ADL total score

  4. Change From Baseline In the The Quantitative Myasthenia Gravis (QMG) total score by at least 5 points At Week 26 [ Time Frame: Baseline, Week 26 ]
    To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the QMG total score


Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Diagnosed with Myasthenia Gravis at least 6 months (180 days) prior to the date of the Screening Visit as confirmed by specific criteria.
  2. Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening.
  3. MG-ADL profile must be ≥ 6 at screening and randomization (Day 1).
  4. Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis).

Exclusion Criteria:

Medical Conditions

  1. 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.
  2. History of thymectomy within the 12 months prior to screening.
  3. History of N meningitidis infection.

  4. Use of the following within the time period specified below:

    • IV immunoglobulin within 4 weeks of randomization
    • Use of plasma exchange within 4 weeks of randomization
    • Use of rituximab within 6 months of screening
  5. Participants who have received previous treatment with complement inhibitors (for example, eculizumab).
Contacts and Locations

Locations
Show Show 144 study locations
Sponsors and Collaborators
Alexion Pharmaceuticals
Tracking Information
First Submitted Date  ICMJE April 16, 2019
First Posted Date  ICMJE April 18, 2019
Last Update Posted Date December 10, 2020
Actual Study Start Date  ICMJE March 26, 2019
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 16, 2019) 		Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26 [ Time Frame: Baseline, Week 26 ]
To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the MG-ADL profile.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 24, 2020)
  • Change From Baseline In The Quantitative Myasthenia Gravis (QMG) Total Score At Week 26 [ Time Frame: Baseline, Week 26 ]
    To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the QMG total score.
  • Change From Baseline In the Revised 15 Component Myasthenia Gravis Quality of Life (MG-QOL15r) At Week 26 [ Time Frame: Baseline, Week 26 ]
    To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the MG-QOL15r score
  • Change From Baseline In The Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score of at least 3 points At Week 26 [ Time Frame: Baseline, Week 26 ]
    To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the MG-ADL total score
  • Change From Baseline In the The Quantitative Myasthenia Gravis (QMG) total score by at least 5 points At Week 26 [ Time Frame: Baseline, Week 26 ]
    To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the QMG total score
Original Secondary Outcome Measures  ICMJE
 (submitted: April 16, 2019) 		Change From Baseline In The Quantitative Myasthenia Gravis (QMG) Total Score At Week 26 [ Time Frame: Baseline, Week 26 ]
To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the QMG total score.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis
Official Title  ICMJE A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis
Brief Summary The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description:
All investigative site personnel, sponsor staff, sponsor designees, staff directly associated with the conduct of the study, and all participants will be blinded to treatment assignments.
Primary Purpose: Treatment
Condition  ICMJE Generalized Myasthenia Gravis
Intervention  ICMJE
  • Biological: Ravulizumab
    Concentrated sterile, preservative-free aqueous solution (10 milligrams [mg]/milliliter [mL]) in single-use, 30-mL vial for intravenous (IV) infusion. Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.
    Other Names:
    • ALXN1210
    • Ultomiris
  • Drug: Placebo
    Matching, sterile, preservative-free aqueous solution in single-use, 30-mL vial for IV infusion. Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.
Study Arms  ICMJE
  • Experimental: Ravulizumab
    Participants will receive ravulizumab for the duration of the study.
    Intervention: Biological: Ravulizumab
  • Placebo Comparator: Placebo
    Participants will receive placebo during the 26-week randomized-controlled period of the study, after which they will enter the open-label extension period of the study and receive ravulizumab.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: December 9, 2020) 		175
Original Estimated Enrollment  ICMJE
 (submitted: April 16, 2019) 		160
Estimated Study Completion Date  ICMJE December 2023
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Diagnosed with Myasthenia Gravis at least 6 months (180 days) prior to the date of the Screening Visit as confirmed by specific criteria.
  2. Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening.
  3. MG-ADL profile must be ≥ 6 at screening and randomization (Day 1).
  4. Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis).

Exclusion Criteria:

Medical Conditions

  1. 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.
  2. History of thymectomy within the 12 months prior to screening.
  3. History of N meningitidis infection.

  4. Use of the following within the time period specified below:

    • IV immunoglobulin within 4 weeks of randomization
    • Use of plasma exchange within 4 weeks of randomization
    • Use of rituximab within 6 months of screening
  5. Participants who have received previous treatment with complement inhibitors (for example, eculizumab).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   Canada,   Czechia,   Denmark,   France,   Germany,   Israel,   Italy,   Japan,   Korea, Republic of,   Netherlands,   Portugal,   Spain,   Switzerland,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03920293
Other Study ID Numbers  ICMJE ALXN1210-MG-306
2018-003243-39 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
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  ICMJE Not Provided
Responsible Party Alexion Pharmaceuticals
Study Sponsor  ICMJE Alexion Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Alexion Pharmaceuticals
Verification Date September 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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