New shot could replace IV drips for rare muscle disease
NCT ID NCT05514873
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 Hide 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
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Generalized myasthenia gravis are added.
Genom att skicka in godkänner du våra Användarvillkor
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a new drug offer Long-Term relief for muscle weakness?
- Can a new drug ease the muscle weakness of myasthenia gravis?
- Engineered immune cells take aim at debilitating muscle weakness
- New hope for myasthenia gravis: experimental drug CNP-106 enters human trials
- New hope for myasthenia gravis patients: experimental drug enters phase 2 trial
- New drug trial aims to ease muscle weakness in rare disease