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Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04115293
Recruitment Status : Recruiting
First Posted : October 4, 2019
Last Update Posted : October 7, 2019
Sponsor:
Information provided by (Responsible Party):
Ra Pharmaceuticals

Brief Summary:
The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or placebo for 12 weeks.

Condition or disease Intervention/treatment Phase
Myasthenia Gravis, Generalized Drug: zilucoplan (RA101495) Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 130 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
Actual Study Start Date : October 1, 2019
Estimated Primary Completion Date : January 2021
Estimated Study Completion Date : April 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: 0.3 mg/kg zilucoplan (RA101495) Drug: zilucoplan (RA101495)
Daily subcutaneous (SC) injection

Placebo Comparator: Placebo Drug: Placebo
Daily subcutaneous (SC) injection




Primary Outcome Measures :
  1. Change from Baseline (CFB) in the MG-ADL Score [ Time Frame: 12 Weeks ]
    The MG-ADL profile provides an assessment of MG symptom severity and measures 8 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the ADL score. The ADL score can range from 0 (least severe) to 24 (most severe).


Secondary Outcome Measures :
  1. Change from Baseline (CFB) in the QMG Score [ Time Frame: 12 Weeks ]
    The QMG test is a standardized quantitative strength scoring system and measures 13 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the QMG score. The QMG score can range from 0 (least severe) to 39 (most severe).



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening
  • Positive serology for acetylcholine receptor (AChR) autoantibodies
  • MG-ADL Score of ≥ 6 at Screening and Baseline
  • QMG score ≥ 12 at Screening and Baseline
  • No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
  • No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period

Exclusion Criteria:

  • Thymectomy within 12 months prior to Baseline or scheduled to occur during the 12 week Treatment Period
  • History of meningococcal disease
  • Current or recent systemic infection within 2 weeks prior to Baseline or injection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline

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): NCT04115293


Contacts
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Contact: Sponsor Ra Pharmaceuticals, Inc. +1 617 401 4060 trials@rapharma.com

Locations
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Sponsors and Collaborators
Ra Pharmaceuticals
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Responsible Party: Ra Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04115293    
Other Study ID Numbers: RA101495-02.301
First Posted: October 4, 2019    Key Record Dates
Last Update Posted: October 7, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases