FRAGILE X SYNDROME
Clinical trials for FRAGILE X SYNDROME explained in plain language.
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Brain drug tested at home for genetic condition
Disease control CompletedThis study tested a single dose of an experimental drug called gaboxadol in 10 adult men with Fragile X Syndrome, a genetic condition that causes intellectual disability. The main goal was to see how the drug affects brain wave patterns measured by a special cap (EEG). Researcher…
Matched conditions: FRAGILE X SYNDROME
Phase: PHASE2 • Sponsor: Craig Erickson • Aim: Disease control
Last updated Mar 30, 2026 14:30 UTC
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New hope for teens with fragile x: drug trial targets learning and behavior
Disease control CompletedThis study tested an investigational drug, BPN14770, to see if it could improve thinking skills, language, and daily functioning in male adolescents with Fragile X Syndrome. It involved 163 boys aged 9 to 18 and compared the drug's effects to a placebo over 13 weeks. The main goa…
Matched conditions: FRAGILE X SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Tetra Discovery Partners • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC
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Diabetes drug tested as potential treatment for genetic disorder
Disease control CompletedThis study tested whether metformin, a common diabetes medication, could help people with Fragile X syndrome. For four months, 125 participants aged 6 to 35 took either metformin or a placebo pill. Researchers measured changes in language skills, behavior problems, and weight to …
Matched conditions: FRAGILE X SYNDROME
Phase: PHASE2 • Sponsor: University of Alberta • Aim: Disease control
Last updated Mar 23, 2026 15:14 UTC
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Skin gel trial offers hope for easing fragile x symptoms
Symptom relief CompletedThis study tested whether a cannabidiol (CBD) gel applied to the skin could help reduce behavioral challenges in children and young adults with Fragile X Syndrome. Over 200 participants, aged 3 to under 30, were randomly assigned to receive either the active gel or a placebo gel …
Matched conditions: FRAGILE X SYNDROME
Phase: PHASE3 • Sponsor: Zynerba Pharmaceuticals, Inc. • Aim: Symptom relief
Last updated Mar 12, 2026 13:50 UTC
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NIH opens doors to children with rare genetic conditions for staff training and research
Knowledge-focused CompletedThis study aimed to evaluate children with known or suspected genetic disorders while providing hands-on training for medical staff. Researchers enrolled 96 children of any age to perform medical exams, genetic testing, and other evaluations to gather data and improve research me…
Matched conditions: FRAGILE X SYNDROME
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:41 UTC