Promising drug for kids with rare muscle disease moves to next phase
NCT ID NCT06055959
First seen Jun 27, 2026 · Last updated Jul 31, 2026 · Updated 4 times
Summary
This study is testing a drug called zilucoplan in children aged 2 to 17 with generalized myasthenia gravis, a disease that causes muscle weakness. The goal is to see how the drug works in the body, if it is safe, and if it helps control the disease. Only 8 children will take part, and they will receive the drug without a placebo.
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
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Phase 2/3
Runs two stages together: whether the treatment works, then large-scale confirmation.
- Participants
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About 8 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Oct 2024
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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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
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12 to 17 years
- Sex
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Anyone
- Healthy volunteers
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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: United States of America (USA) specific inclusion criterion: \- Participant must be 12 to \<18 years of age at the time of signing the Informed consent/assent according to local regulation Rest of world (ROW) specific inclusion criterion: \- Participant must be 2 to \<18 years of age at the time of signing the Informed consent/assent according to local regulation Global inclusion criteria: * Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening * Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening * Participants with gMG, including: * An MG-activities of daily living (MG-ADL) total score of 6 or more in adolescents from 12 years to \<18 years of age at Screening * Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2 years to \<12 years of age at Screening (does not apply to US) * Documented vaccination against meningococcal infections within 3 years prior to study start. If not fully vaccinated, participants must receive appropriate prophylactic antibiotic treatment until at least 2 weeks after the initial dose of vaccine(s) Exclusion Criteria: * Participant has known positive serology for muscle-specific kinase * 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 had a thymectomy within 6 months prior to Baseline * Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator * Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
5 sites in 4 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Mg0014 20104
WITHDRAWNSeoul, South Korea
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Mg0014 20220
RECRUITINGSeoul, South Korea
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Mg0014 40144
RECRUITINGMilan, Italy
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Mg0014 40218
ACTIVE_NOT_RECRUITINGWarsaw, Poland
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Mg0014 40735
RECRUITINGGlasgow, United Kingdom
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Mg0014 40736
RECRUITINGLondon, United Kingdom
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Mg0014 40774
RECRUITINGKatowice, Poland
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Mg0014 50168
WITHDRAWNChicago, Illinois, 60611, United States
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Mg0014 50574
WITHDRAWNFlower Mound, Texas, 75028, 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