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

Purpose

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

  • Generalized Myasthenia Gravis

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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).

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
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.

Arm Groups

ArmDescriptionAssigned Intervention
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.

Recruiting Locations

UK Center for Clinical and Translational Science and nearby locations

University of Kentucky
Lexington, Kentucky 40536

More Details

NCT ID
NCT03920293
Status
Recruiting
Sponsor
Alexion Pharmaceuticals

Study Contact

Alexion Pharmaceuticals Inc.
855-752-2356
clinicaltrials@alexion.com