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Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis

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: NCT03315130
Recruitment Status : Recruiting
First Posted : October 19, 2017
Last Update Posted : December 27, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.

Condition or disease Intervention/treatment Phase
Generalized Myasthenia Gravis Drug: RA101495 Drug: Placebo Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RA101495 in Subjects With Generalized Myasthenia Gravis
Actual Study Start Date : October 11, 2017
Estimated Primary Completion Date : February 2019
Estimated Study Completion Date : April 2019

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: 0.1 mg/kg RA101495 Drug: RA101495
Daily subcutaneous (SC) injection
Experimental: 0.3 mg/kg RA101495 Drug: RA101495
Daily subcutaneous (SC) injection
Placebo Comparator: Placebo Drug: Placebo
Daily subcutaneous (SC) injection


Outcome Measures

Primary Outcome Measures :
  1. Change from baseline in Quantitative Myasthenia Gravis (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.


Secondary Outcome Measures :
  1. Change from baseline in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale [ 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.


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • Thymectomy within 6 months prior to Randomization or scheduled to occur during the 12 week Treatment Period
  • History of meningococcal disease
  • Current or recent systemic infection within 2 weeks prior to Randomization or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Randomization
Contacts and Locations

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): NCT03315130


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

  Show 28 Study Locations
Sponsors and Collaborators
Ra Pharmaceuticals
More Information

Responsible Party: Ra Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03315130     History of Changes
Other Study ID Numbers: RA101495-02.201
First Posted: October 19, 2017    Key Record Dates
Last Update Posted: December 27, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

Additional relevant MeSH terms:
Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Signs and Symptoms
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases