Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE-XT)

A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects With Generalized Myasthenia Gravis

The RAISE-XT study is an open-label extension study to evaluate the long-term efficacy, safety, and tolerability of zilucoplan in subjects with gMG who have previously participated in a qualifying Ra Pharmaceuticals sponsored zilucoplan study.

Study Overview

• Status: Active, not recruiting
• Conditions: Generalized Myasthenia Gravis
• Intervention / Treatment: Drug: zilucoplan (RA101495)

• Study Type: Interventional
• Enrollment (Actual): 200
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Montreal, Canada
    • Mg0011 11
• France
  • Lille, France
    • Mg0011 204
  • Nice, France
    • Mg0011 118
  • Paris, France
    • Mg0011 105
  • Strasbourg, France
    • Mg0011 137
• Germany
  • Göttingen, Germany
    • Mg0011 150
  • Tübingen, Germany
    • Mg0011 129
• Italy
  • Milano, Italy
    • Mg0011 132
  • Roma, Italy
    • Mg0011 126
• Japan
  • Chiba, Japan
    • Mg0011 151
  • Hanamaki-shi, Japan
    • Mg0011 136
  • Kita-gun, Japan
    • Mg0011 179
  • Meguro-ku, Japan
    • Mg0011 153
  • Nagasaki-shi, Japan
    • Mg0011 146
  • Narita, Japan
    • Mg0011 169
  • Sapporo, Japan
    • Mg0011 152
  • Sendai, Japan
    • Mg0011 144
  • Shinjuku-ku, Japan
    • Mg0011 163
  • Tokyo, Japan
    • Mg0011 141
• Norway
  • Bergen, Norway
    • Mg0011 140
  • Oslo, Norway
    • Mg0011 143
• Poland
  • Katowice, Poland
    • Mg0011 195
  • Katowice, Poland
    • Mg0011 213
  • Krakow, Poland
    • Mg0011 192
  • Krakow, Poland
    • Mg0011 193
  • Kraków, Poland
    • Mg0011 211
  • Lublin, Poland
    • Mg0011 205
  • Nowa Sol, Poland
    • Mg0011 194
  • Poznań, Poland
    • Mg0011 209
  • Warszawa, Poland
    • Mg0011 201
  • Zabrze, Poland
    • Mg0011 210
  • Łódź, Poland
    • Mg0011 214
• Spain
  • Barcelona, Spain
    • Mg0011 133
  • Barcelona, Spain
    • Mg0011 168
  • Bilbao, Spain
    • Mg0011 138
• United Kingdom
  • Oxford, United Kingdom
    • Mg0011 119
  • Sheffield, United Kingdom
    • Mg0011 130
• United States
  • Alabama
    • Mobile, Alabama, United States, 36604
      • Mg0011 41
  • Arizona
    • Phoenix, Arizona, United States, 85028
      • Mg0011 116
  • California
    • Irvine, California, United States, 92697
      • Mg0011 31
    • Los Angeles, California, United States, 90033
      • MG0011 4
    • Pasadena, California, United States, 91101
      • Mg0011 220
    • San Francisco, California, United States, 94115
      • Mg0011 160
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Mg0011 24
  • District of Columbia
    • Washington, District of Columbia, United States, 20052
      • Mg0011 27
  • Florida
    • Tampa, Florida, United States, 33612
      • Mg0011 25
  • Georgia
    • Augusta, Georgia, United States, 30912
      • Mg0011 135
  • Illinois
    • Glenview, Illinois, United States, 60026-1339
      • Mg0011 188
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Mg0011 156
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • Mg0011 32
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Mg0011 33
    • East Lansing, Michigan, United States, 48824
      • Mg0011 49
  • Missouri
    • Columbia, Missouri, United States, 65201
      • Mg0011 134
  • Nevada
    • Las Vegas, Nevada, United States, 89145
      • Mg0011 117
  • New York
    • Buffalo, New York, United States, 14202-1102
      • Mg0011 30
    • Great Neck, New York, United States, 11021
      • Mg0011 123
    • New York, New York, United States, 10021
      • Mg0011 23
    • New York, New York, United States, 10029
      • Mg0011 47
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • Mg0011 22
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Mg0011 122
    • Columbus, Ohio, United States, 43210
      • Mg0011 38
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15212
      • Mg0011 40
  • South Carolina
    • Charleston, South Carolina, United States, 29425-8908
      • Mg0011 128
  • Tennessee
    • Cordova, Tennessee, United States, 38018
      • Mg0011 28
  • Texas
    • Austin, Texas, United States, 78759
      • Mg0011 131
    • Dallas, Texas, United States, 75390
      • Mg0011 19
  • Utah
    • Salt Lake City, Utah, United States, 84105
      • Mg0011 39
  • Virginia
    • Charlottesville, Virginia, United States, 22903
      • Mg0011 164
  • Washington
    • Seattle, Washington, United States, 98195
      • Mg0011 154
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53215
      • Mg0011 45

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Completion of a qualifying zilucoplan study

Exclusion Criteria:

• With the exception of a prior zilucoplan trial, participation in another concurrent clinical trial involving an experimental therapeutic intervention (participation in observational studies and/or registry studies is permitted)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 0.3 mg/kg zilucoplan (RA101495)	Drug: zilucoplan (RA101495); Daily subcutaneous (SC) injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment-emergent adverse events (TEAEs)	A treatment-emergent adverse event is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication.	From Baseline (Day 1) to Safety Follow-Up Visit (up to 36 months)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score	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 8 items represents the ADL score. The ADL score can range from 0 (least severe) to 24 (most severe).	From Baseline (Day 1) to Week 12
Change from Baseline to Week 12 in the the Quantitative Myasthenia Gravis (QMG) Score	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).	From Baseline (Day 1) to Week 12
Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Score	The MGC is a 10-item scale that has been used to measure the clinical status of patients with Myasthenia Gravis (MG) in order to evaluate treatment response. The MGC has 4-point Likerttype Scale response options ranging from 0 to 2, 3, 4, 5, 6 or 9 according to the item (weighted response options). The total score is the sum of all items (range 0-50) where higher scores indicate more severe impairment due to the disease.	From Baseline (Day 1) to Week 12
Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QOL15r) Score	The MG-QOL15r is a 15-item survey that was designed to assess quality of life in patients with MG. The MG-QoL has 3-point Likert Scale response options ranging from 0 to 2. The MGQoL15r score can range from 0 to 30, where higher scores indicate more severe impact of the disease on aspects of the patient's life.	From Baseline (Day 1) to Week 12

Collaborators and Investigators

• Sponsor: Ra Pharmaceuticals, Inc.
• Investigators: Study Director: UCB Cares, 001 844 599 2273

Study record dates

Study Major Dates

• Study Start (Actual): December 23, 2019
• Primary Completion (Estimated): May 20, 2026
• Study Completion (Estimated): May 20, 2026

Study Registration Dates

• First Submitted: October 2, 2019
• First Submitted That Met QC Criteria: January 8, 2020
• First Posted (Actual): January 13, 2020

Study Record Updates

• Last Update Posted (Estimated): April 15, 2024
• Last Update Submitted That Met QC Criteria: April 11, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Neurologic Manifestations; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Nervous System Neoplasms; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Muscle Weakness; Myasthenia Gravis
• Other Study ID Numbers: RA101495-02.302

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
• IPD Sharing Time Frame: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• IPD Sharing Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No