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Daily injection shows promise for Long-Term control of rare muscle disease

NCT ID NCT04225871

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Aug 13, 2026 · Updated 7 times

Summary

This study tests the long-term safety and effectiveness of zilucoplan, a daily injection, in people with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. About 200 participants who completed a prior zilucoplan trial will receive the drug for an extended period. Researchers will track side effects and changes in daily living scores to see if the treatment remains helpful over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
zilucoplan (a daily injection under the skin)
What this could lead to
If successful, this could confirm zilucoplan as a long-term treatment option to control symptoms of generalized myasthenia gravis.
What could go wrong
This is an open-label extension, so there is no placebo group for comparison. Long-term safety and effectiveness are still being evaluated, and side effects may emerge over time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

200 people

The number who actually took part.

Started

Dec 2019

Finished

Jul 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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)

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • MG0011 4

    Los Angeles, California, 90033, United States

  • Mg0011 105

    Paris, France

  • Mg0011 11

    Montreal, Canada

  • Mg0011 116

    Phoenix, Arizona, 85028, United States

  • Mg0011 117

    Las Vegas, Nevada, 89145, United States

  • Mg0011 118

    Nice, France

  • Mg0011 119

    Oxford, United Kingdom

  • Mg0011 122

    Cleveland, Ohio, 44195, United States

  • Mg0011 123

    Great Neck, New York, 11021, United States

  • Mg0011 126

    Roma, Italy

  • Mg0011 128

    Charleston, South Carolina, 29425-8908, United States

  • Mg0011 129

    Tübingen, Germany

  • Mg0011 130

    Sheffield, United Kingdom

  • Mg0011 131

    Austin, Texas, 78759, United States

  • Mg0011 132

    Milan, Italy

  • Mg0011 133

    Barcelona, Spain

  • Mg0011 134

    Columbia, Missouri, 65201, United States

  • Mg0011 135

    Augusta, Georgia, 30912, United States

  • Mg0011 136

    Hanamaki-shi, Japan

  • Mg0011 137

    Strasbourg, France

  • Mg0011 138

    Bilbao, Spain

  • Mg0011 140

    Bergen, Norway

  • Mg0011 141

    Tokyo, Japan

  • Mg0011 143

    Oslo, Norway

  • Mg0011 144

    Sendai, Japan

  • Mg0011 146

    Nagasaki, Japan

  • Mg0011 150

    Göttingen, Germany

  • Mg0011 151

    Chiba, Japan

  • Mg0011 152

    Sapporo, Japan

  • Mg0011 153

    Meguro-ku, Japan

  • Mg0011 154

    Seattle, Washington, 98195, United States

  • Mg0011 156

    Indianapolis, Indiana, 46202, United States

  • Mg0011 160

    San Francisco, California, 94115, United States

  • Mg0011 163

    Shinjuku-ku, Japan

  • Mg0011 164

    Charlottesville, Virginia, 22903, United States

  • Mg0011 168

    Barcelona, Spain

  • Mg0011 169

    Narita, Japan

  • Mg0011 179

    Kita-gun, Japan

  • Mg0011 188

    Glenview, Illinois, 60026-1339, United States

  • Mg0011 19

    Dallas, Texas, 75390, United States

  • Mg0011 192

    Krakow, Poland

  • Mg0011 193

    Krakow, Poland

  • Mg0011 194

    Nowa Sól, Poland

  • Mg0011 195

    Katowice, Poland

  • Mg0011 201

    Warsaw, Poland

  • Mg0011 204

    Lille, France

  • Mg0011 205

    Lublin, Poland

  • Mg0011 209

    Poznan, Poland

  • Mg0011 210

    Zabrze, Poland

  • Mg0011 211

    Krakow, Poland

  • Mg0011 213

    Katowice, Poland

  • Mg0011 214

    Lodz, Poland

  • Mg0011 22

    Chapel Hill, North Carolina, 27599, United States

  • Mg0011 220

    Pasadena, California, 91101, United States

  • Mg0011 23

    New York, New York, 10021, United States

  • Mg0011 24

    New Haven, Connecticut, 06511, United States

  • Mg0011 25

    Tampa, Florida, 33612, United States

  • Mg0011 27

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

  • Mg0011 28

    Cordova, Tennessee, 38018, United States

  • Mg0011 30

    Buffalo, New York, 14202-1102, United States

  • Mg0011 31

    Irvine, California, 92697, United States

  • Mg0011 32

    Kansas City, Kansas, 66160, United States

  • Mg0011 33

    Detroit, Michigan, 48201, United States

  • Mg0011 38

    Columbus, Ohio, 43210, United States

  • Mg0011 39

    Salt Lake City, Utah, 84105, United States

  • Mg0011 40

    Pittsburgh, Pennsylvania, 15212, United States

  • Mg0011 41

    Mobile, Alabama, 36604, United States

  • Mg0011 45

    Milwaukee, Wisconsin, 53215, United States

  • Mg0011 47

    New York, New York, 10029, United States

  • Mg0011 49

    East Lansing, Michigan, 48824, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.