New hope for kids with rare muscle disease: drug trial launches

NCT ID NCT06392386

Recruiting now Disease control Sponsor: argenx Source: ClinicalTrials.gov ↗

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

Summary

This study tests a drug called efgartigimod in 12 children aged 2 to 18 with generalized myasthenia gravis, a condition that causes muscle weakness. The goal is to find the right dose and see if it is safe. Participants receive injections and are monitored for up to 14 weeks.

What this could mean

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

Active substance: efgartigimod
What this could lead to: If successful, this could provide a new treatment option to control symptoms 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 children. The drug may cause side effects or not work as well as hoped.

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

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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

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

AP-HM- Hôpital de La Timone
RECRUITING

Marseille, 13385, France
Carolinas HealthCare System Neurosciences Institute - Neurology
RECRUITING

Charlotte, North Carolina, 28207, United States

Contact Email: •••••@•••••
Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM
RECRUITING

Warsaw, 02-097, Poland
Childrens Hospital of Eastern Ontario
RECRUITING

Ottawa, K1H 8L1, Canada

Contact Email: •••••@•••••
Fakultni nemocnice Brno
RECRUITING

Brno, 613 00, Czechia
Fakultni nemocnice Ostrava
RECRUITING

Ostrava, 708 00, Czechia
Hadassah Medical Center- Ein Kerem
RECRUITING

Jerusalem, 9112001, Israel

Contact Email: •••••@•••••
Hospital Universitari i Politecnic La Fe de Valencia
RECRUITING

Valencia, 46026, Spain
Hôpital Nestlé
RECRUITING

Lausanne, 1011, Switzerland

Contact Email: •••••@•••••
Istituto G Gaslini Ospedale Pediatrico IRCCS
RECRUITING

Genova, 16147, Italy
Leiden University Medical Center
RECRUITING

Leiden, 2333 ZA, Netherlands
Neurologia Śląska Centrum Medyczne
RECRUITING

Katowice, 40-689, Poland
Neurology Rare Disease Center
RECRUITING

Denton, Texas, 76208, United States

Contact Email: •••••@•••••
Oxford Children's Hospital
RECRUITING

Oxford, OX3 9DU, United Kingdom

Contact Email: •••••@•••••
Schneider Children's Medical Center of Israel
RECRUITING

Petah Tikvah, 4920235, Israel

Contact Email: •••••@•••••
Tel Aviv Sourasky Medical Center Ichilov
RECRUITING

Tel Aviv, 6423906, Israel

Contact Email: •••••@•••••
UZ Gent
RECRUITING

Ghent, 9000, Belgium
University of Virginia
RECRUITING

Charlottesville, Virginia, 22903, United States

Contact Email: •••••@•••••
Universitätsklinikum Essen
RECRUITING

Essen, 45147, Germany
Uniwersyteckie Centrum Kliniczne w Gdansku
RECRUITING

Gdansk, 80-211, Poland

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