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New shot could replace IV drips for rare muscle disease

NCT ID NCT05514873

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 26, 2026 · Updated 2 times

Summary

This study tested whether people with generalized myasthenia gravis could safely switch from intravenous (IV) infusions of complement inhibitors to daily injections of zilucoplan. Twenty-six participants who were stable on IV therapy switched to the subcutaneous shot. The main focus was on safety and tolerability, with secondary measures of symptom control.

What this could mean

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

Active substance
zilucoplan
What this could lead to
If successful, this could offer a more convenient at-home injection option for people with generalized myasthenia gravis who currently need regular IV infusions.
What could go wrong
This is a small, single-arm study (26 people) with no comparison group, so results may not apply broadly. The main goal is safety, not proof of superior effectiveness.

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

26 people

The number who actually took part.

Started

Oct 2022

Finished

Oct 2024

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 to 85 years

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: * Participant has been treated with an intravenous (IV) complement component 5 (C5) inhibitor approved for the treatment of generalized myasthenia gravis (gMG) at the recommended dose regimen for at least 3 months (for eculizumab) or 4 months (for ravulizumab) prior to Screening with a clinically stable disease as per the Investigator's judgment. * Participant is willing to switch from his/her current IV C5 inhibitor to subcutaneous (SC) zilucoplan (ZLP) * Participant has a documented diagnosis of gMG (Myasthenia Gravis Foundation of America; MGFA Class II-IVa) at Screening based on participant history and supported by previous evaluations * Participant has a well-documented record of positive serology for acetylcholine receptor binding autoantibodies prior to Screening * Participant has no more than a 2-point change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Baseline compared with the Screening Visit * Participant has had no change in corticosteroid dose during the Screening Period and no change in corticosteroid dose is anticipated to occur during the 12-week Main Treatment Period * Participant has had no change in immunosuppressive therapy, including dose, during the Screening Period and no change in immunosuppressive therapy is anticipated to occur during the 12-week Main Treatment Period * Participant has a record of vaccination with at least 1 dose of a quadrivalent meningococcal vaccine and meningococcal serotype B vaccine at least 14 days prior to the first dose of ZLP if not vaccinated within 3 years prior to the start of study medication * Male and/or female * A male participant is recommended to agree to use contraception during the study and for at least 40 days (5 half lives) after the last dose of study medication, and refrain from donating sperm during this period. * A female participant is eligible to participate if she is not pregnant; not breastfeeding, and at least one of the following conditions applies: * Not a woman of childbearing potential (WOCBP) OR * A WOCBP who agrees to follow the contraceptive guidance during the study and for at least 40 days (5 half lives) after the last dose of study medication. * Participant is capable of giving signed informed consent Exclusion Criteria: * Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the participant's ability to participate in this study * Participant has a known hypersensitivity to any components of the study medication as stated in this protocol * Participant has had a thymectomy within 6 months prior to Baseline or has one scheduled to occur during the 12-week Main Treatment Period * Participant has a history of meningococcal disease * Participant has or has had a current or recent systemic infection within 2 weeks prior to Baseline or an infection requiring IV antibiotics within 4 weeks prior to Baseline * Participant has active malignancy (except curatively resected squamous or basal cell carcinoma of the skin) requiring surgery, chemotherapy, or radiation within the prior 12 months (participants with a history of malignancy who have undergone curative resection or otherwise not requiring treatment for at least 12 months prior to Screening with no detectable recurrence are allowed). * Participant has a lifetime history of suicide attempt (including an actual attempt, interrupted attempt, or aborted attempt), or has had suicidal ideation with at least some intent to act in the past 6 months as indicated by a positive response ("Yes") to either question 4 or question 5 of the "Screening/Baseline" version of the C-SSRS at Screening. * Participant has alanine transaminase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP) \>2.5x upper limit of normal (ULN) * Participant has bilirubin \>1.5xULN (isolated bilirubin \>1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin \<35%). * Participant has current unstable liver or biliary disease per Investigator assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis. * QTc interval \>450msec for male participants, QTc \>470msec for female participants, or QTc \>480 msec in participants with bundle branch block * Participant has had recent surgery requiring general anesthesia within 2 weeks prior to Screening or is expected to have surgery requiring general anesthesia during the 12-week Main Treatment Period * Participant has received a treatment with an experimental drug within 30 days or 5 half lives of the experimental drug (whichever is longer) prior to Baseline * Participant has received treatment with rituximab within 6 months prior to Baseline or treatment is planned to occur during the study * Participant has received treatment with intravenous immunoglobulin G (IVIG), SC immunoglobulin, or plasma exchange PLEX 4 weeks prior to Baseline or participant is on chronic IVIG, SC immunoglobulin, or PLEX * Participant has previously participated in this study or participant has previously been assigned to treatment in a study of the medication under investigation in this study * Participant has participated in another study of an investigational study medication (and/or an investigational device) within the previous 30 days or is currently participating in another study of an investigational study medication (and/or an investigational device) * Participant has known positive serology for muscle-specific kinase

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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

  • Mg0017 50076

    Columbus, Ohio, 43221, United States

  • Mg0017 50086

    Charlotte, North Carolina, 28207, United States

  • Mg0017 50099

    San Francisco, California, 94143, United States

  • Mg0017 50111

    Seattle, Washington, 98195-0001, United States

  • Mg0017 50304

    Dallas, Texas, 75390-8866, United States

  • Mg0017 50555

    Austin, Texas, 78759, United States

  • Mg0017 50556

    Chapel Hill, North Carolina, 27599, United States

  • Mg0017 50557

    O'Fallon, Illinois, 62269, United States

  • Mg0017 50559

    Los Angeles, California, 90095, United States

  • Mg0017 50564

    Scottsdale, Arizona, 85251, United States

  • Mg0017 50569

    Greenfield, Wisconsin, 53228, United States

  • Mg0017 50593

    Rancho Mirage, California, 92270, United States

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