NCT04225871

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
200

participants targeted

Target at P25-P50 for phase_3

Timeline
0mo left

Started Dec 2019

Longer than P75 for phase_3

Geographic Reach
10 countries

70 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress99%
Dec 2019Jun 2026

First Submitted

Initial submission to the registry

October 2, 2019

Completed
3 months until next milestone

Study Start

First participant enrolled

December 23, 2019

Completed
21 days until next milestone

First Posted

Study publicly available on registry

January 13, 2020

Completed
6.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 2, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 2, 2026

Last Updated

April 24, 2026

Status Verified

April 1, 2026

Enrollment Period

6.4 years

First QC Date

October 2, 2019

Last Update Submit

April 23, 2026

Conditions

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (4)

  • Change from Baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score

    From Baseline (Day 1) to Week 12

  • Change from Baseline to Week 12 in the the Quantitative Myasthenia Gravis (QMG) Score

    From Baseline (Day 1) to Week 12

  • Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Score

    From Baseline (Day 1) to Week 12

  • Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QOL15r) Score

    From Baseline (Day 1) to Week 12

Study Arms (1)

0.3 mg/kg zilucoplan (RA101495)

EXPERIMENTAL
Drug: zilucoplan (RA101495)

Interventions

Daily subcutaneous (SC) injection

0.3 mg/kg zilucoplan (RA101495)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Completion of a qualifying zilucoplan study

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (70)

Mg0011 41

Mobile, Alabama, 36604, United States

Location

Mg0011 116

Phoenix, Arizona, 85028, United States

Location

Mg0011 31

Irvine, California, 92697, United States

Location

MG0011 4

Los Angeles, California, 90033, United States

Location

Mg0011 220

Pasadena, California, 91101, United States

Location

Mg0011 160

San Francisco, California, 94115, United States

Location

Mg0011 24

New Haven, Connecticut, 06511, United States

Location

Mg0011 27

Washington D.C., District of Columbia, 20052, United States

Location

Mg0011 25

Tampa, Florida, 33612, United States

Location

Mg0011 135

Augusta, Georgia, 30912, United States

Location

Mg0011 188

Glenview, Illinois, 60026-1339, United States

Location

Mg0011 156

Indianapolis, Indiana, 46202, United States

Location

Mg0011 32

Kansas City, Kansas, 66160, United States

Location

Mg0011 33

Detroit, Michigan, 48201, United States

Location

Mg0011 49

East Lansing, Michigan, 48824, United States

Location

Mg0011 134

Columbia, Missouri, 65201, United States

Location

Mg0011 117

Las Vegas, Nevada, 89145, United States

Location

Mg0011 30

Buffalo, New York, 14202-1102, United States

Location

Mg0011 123

Great Neck, New York, 11021, United States

Location

Mg0011 23

New York, New York, 10021, United States

Location

Mg0011 47

New York, New York, 10029, United States

Location

Mg0011 22

Chapel Hill, North Carolina, 27599, United States

Location

Mg0011 122

Cleveland, Ohio, 44195, United States

Location

Mg0011 38

Columbus, Ohio, 43210, United States

Location

Mg0011 40

Pittsburgh, Pennsylvania, 15212, United States

Location

Mg0011 128

Charleston, South Carolina, 29425-8908, United States

Location

Mg0011 28

Cordova, Tennessee, 38018, United States

Location

Mg0011 131

Austin, Texas, 78759, United States

Location

Mg0011 19

Dallas, Texas, 75390, United States

Location

Mg0011 39

Salt Lake City, Utah, 84105, United States

Location

Mg0011 164

Charlottesville, Virginia, 22903, United States

Location

Mg0011 154

Seattle, Washington, 98195, United States

Location

Mg0011 45

Milwaukee, Wisconsin, 53215, United States

Location

Mg0011 11

Montreal, Canada

Location

Mg0011 204

Lille, France

Location

Mg0011 118

Nice, France

Location

Mg0011 105

Paris, France

Location

Mg0011 137

Strasbourg, France

Location

Mg0011 150

Göttingen, Germany

Location

Mg0011 129

Tübingen, Germany

Location

Mg0011 132

Milan, Italy

Location

Mg0011 126

Roma, Italy

Location

Mg0011 151

Chiba, Japan

Location

Mg0011 136

Hanamaki-shi, Japan

Location

Mg0011 179

Kita-gun, Japan

Location

Mg0011 153

Meguro-ku, Japan

Location

Mg0011 146

Nagasaki, Japan

Location

Mg0011 169

Narita, Japan

Location

Mg0011 152

Sapporo, Japan

Location

Mg0011 144

Sendai, Japan

Location

Mg0011 163

Shinjuku-ku, Japan

Location

Mg0011 141

Tokyo, Japan

Location

Mg0011 140

Bergen, Norway

Location

Mg0011 143

Oslo, Norway

Location

Mg0011 195

Katowice, Poland

Location

Mg0011 213

Katowice, Poland

Location

Mg0011 192

Krakow, Poland

Location

Mg0011 193

Krakow, Poland

Location

Mg0011 211

Krakow, Poland

Location

Mg0011 214

Lodz, Poland

Location

Mg0011 205

Lublin, Poland

Location

Mg0011 194

Nowa Sól, Poland

Location

Mg0011 209

Poznan, Poland

Location

Mg0011 201

Warsaw, Poland

Location

Mg0011 210

Zabrze, Poland

Location

Mg0011 133

Barcelona, Spain

Location

Mg0011 168

Barcelona, Spain

Location

Mg0011 138

Bilbao, Spain

Location

Mg0011 119

Oxford, United Kingdom

Location

Mg0011 130

Sheffield, United Kingdom

Location

Related Publications (5)

  • Weiss MD, Freimer M, Leite MI, Maniaol A, Utsugisawa K, Bloemers J, Boroojerdi B, Howard E, Savic N, Howard JF Jr. Improvement of fatigue in generalised myasthenia gravis with zilucoplan. J Neurol. 2024 May;271(5):2758-2767. doi: 10.1007/s00415-024-12209-3. Epub 2024 Feb 24.

  • de la Borderie G, Chimits D, Boroojerdi B, Brock M, Duda PW, Grimson F, Mahoney P, Strimenopoulou F, Cutter G, Aban I, Brauner S, Petersson M, Howard JF Jr, Bennett N. Maintenance of zilucoplan efficacy in patients with generalised myasthenia gravis up to 24 weeks: a model-informed analysis. Ther Adv Neurol Disord. 2024 Sep 21;17:17562864241279125. doi: 10.1177/17562864241279125. eCollection 2024.

  • Hewamadduma C, Freimer M, Genge A, Leite MI, Utsugisawa K, Vu T, Boroojerdi B, Grimson F, Savic N, Vanderkelen M, Howard JF Jr; RAISE-XT study team. Changes in corticosteroid and non-steroidal immunosuppressive therapy with long-term zilucoplan treatment in generalized myasthenia gravis. J Neurol. 2025 Jun 12;272(7):457. doi: 10.1007/s00415-025-13113-0.

  • Howard JF Jr, Bresch S, Farmakidis C, Freimer M, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Nowak RJ, Sivakumar K, Smilowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Bloemers J, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Vanderkelen M, Leite MI; RAISE-XT Study Team*. Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study. Ther Adv Neurol Disord. 2024 Apr 17;17:17562864241243186. doi: 10.1177/17562864241243186. eCollection 2024.

  • Howard JF Jr, Bresch S, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Sivakumar K, Smilowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Lowcock R, Vanderkelen M, Leite MI; RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023 May;22(5):395-406. doi: 10.1016/S1474-4422(23)00080-7.

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

zilucoplan

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous SystemNervous System NeoplasmsNeoplasms by SiteNeoplasmsParaneoplastic SyndromesAutoimmune Diseases of the Nervous SystemNervous System DiseasesNeurodegenerative DiseasesNeuromuscular Junction DiseasesNeuromuscular DiseasesAutoimmune DiseasesImmune System Diseases

Study Officials

  • UCB Cares

    001 844 599 2273

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 2, 2019

First Posted

January 13, 2020

Study Start

December 23, 2019

Primary Completion (Estimated)

June 2, 2026

Study Completion (Estimated)

June 2, 2026

Last Updated

April 24, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

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.

Shared Documents
STUDY PROTOCOL, SAP, CSR
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.
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.
More information

Locations