New pill tested to halt debilitating 'Second Skeleton' disease
NCT ID NCT05039515
Summary
This study is testing whether an oral medication called fidrisertib can slow the formation of new, abnormal bone in people with Fibrodysplasia Ossificans Progressiva (FOP), a rare and severely disabling genetic disease. Researchers will compare two different doses of the drug against a placebo in about 113 participants, including adults and children as young as 5 years old. The main goal is to see if the drug reduces the volume of new bone growth measured by whole-body scans over a year.
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Contacts and locations
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Locations
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Aichi Children's Health and Medical Center
Ōbu, Japan
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Children's Hospital Capital Institute of Pediatrics (CIP)
Beijing, China
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Groupe Hospitalier Necker Enfants Malades
Paris, 75015, France
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Hopital Lariboisiere
Paris, France
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Hospital Center Lisbon North, E.P.E- Hospital Santa Maria
Lisbon, Portugal
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Hospital Italiano de Buenos Aires
Buenos Aires, Argentina
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Hospital Universitario Ramon y Cajal
Pozuelo de Alarcón, 28224, Spain
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Hospital Universitario Y Politecnico La Femerge
Valencia, 46026, Spain
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Instituto Nacional De Rehabilitacion
Mexico City, Mexico
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Irccs Gaslini Institute
Genoa, 16147, Italy
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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Nagoya University Hospital
Nagoya, Japan
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Norrlands Universitetssjukhus
Umeå, Sweden
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Peking Union Medical College Hospital
Beijing, China
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Royal North Shore Hospital - New South Wales
Sydney, Australia
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Seoul National University Hospital
Seoul, South Korea
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Shangai Children Medical Center
Shanghai, China
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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The Perelman School of Medicine - The University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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The University of Tokyo Hospital
Tokyo, Japan
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Tongi University - Tongi Hospital
Shanghai, China
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University Health Network (UHN), Toronto General Hospital (TGH)
Toronto, Canada
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University Hospitals Leuven
Leuven, Belgium
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University of Alberta, Alberta Health Services (AHS)
Edmonton, Canada
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University of California San Francisco (UCSF)
San Francisco, California, 94143, United States
Conditions
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