New drug tested for rare genetic disorder
NCT ID NCT06430385
Summary
This study is testing a new drug called ION440 for people with MECP2 Duplication Syndrome, a rare genetic disorder. The main goal is to see if the drug is safe and how the body handles it. The drug is given via a spinal injection, and the study will include both children and adults.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Baylor College of Medicine
RECRUITINGHouston, Texas, 77030, United States
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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CHU Dijon Bourgogne
RECRUITINGDijon, 21079, France
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Gillette Children's Specialty Healthcare
RECRUITINGSaint Paul, Minnesota, 55101, United States
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Kennedy Krieger
RECRUITINGBaltimore, Maryland, 21205, United States
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Kepler University Hospital
RECRUITINGLinz, 4040, Austria
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Rady Children's Hospital
RECRUITINGSan Diego, California, 92123, United States
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University of Colorado Hopsital - Anschutz Medical Campus
RECRUITINGAurora, Colorado, 80045, United States
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Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232, United States
Conditions
Explore the condition pages connected to this study.