New hope for kids with rare Muscle-Weakening disease

NCT ID NCT06607627

Recruiting now Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a drug called gefurulimab in 12 children aged 6 to 18 with generalized myasthenia gravis, a condition that causes muscle weakness. The drug works by blocking a part of the immune system to reduce symptoms. Participants receive weekly injections, and researchers will check how the drug moves through the body, its safety, and whether it improves muscle strength.

What this could mean

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

Active substance: gefurulimab (a drug that blocks a part of the immune system called complement C5)
What this could lead to: If successful, this could provide a new treatment option to control muscle weakness in children with generalized myasthenia gravis.
What could go wrong: This is a small, early-phase study with only 12 participants, so results may not apply to all patients. The drug requires lifelong use and carries infection risks, including meningococcal infection.

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Conditions

The condition(s) this trial relates to.

myasthenia gravis

As listed by the trial registrant

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

GENERALIZED MYASTHENIA GRAVIS GMG

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Research Site
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Research Site
RECRUITING

Norfolk, Virginia, 23507, United States
Research Site
RECRUITING

Joinville, 89202-451, Brazil
Research Site
RECRUITING

Salvador, 41253-190, Brazil
Research Site
RECRUITING

São José do Rio Preto, 15090-000, Brazil
Research Site
RECRUITING

São Paulo, 0438-002, Brazil
Research Site
RECRUITING

São Paulo, 05403-000, Brazil
Research Site
RECRUITING

Saitama-Shi, 330-8777, Japan
Research Site
WITHDRAWN

Bydgoszcz, 85-065, Poland
Research Site
WITHDRAWN

Lodz, 93-338, Poland
Research Site
RECRUITING

Warsaw, 02-097, Poland
Research Site
RECRUITING

New Taipei City, 23561, Taiwan
Research Site
RECRUITING

Taipei, 11101, Taiwan

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