Methods of treating developmental disorders with gaboxadol

Abstract

Methods of treating developmental disorders such as Angelman syndrome, Fragile X syndrome, Fragile X-associated tremor/ataxia syndrome (FXTAS), Autistic Spectrum Disorder, Autism, Asperger's syndrome, pervasive developmental disorder, Childhood Disintegrative Disorder, Rett syndrome, Lanau-Kleffner Syndrome, Prader-Willi Syndrome, Tardive Dyskinesia, and/or Williams Syndrome with gaboxadol or a pharmaceutically acceptable salt thereof are provided. The methods provide therapeutic compositions that may be used to improve one or more symptoms of the developmental disorder.

What is claimed is: 
     
         1 . A method of treating a developmental disorder comprising administering to a patient in need thereof about 0.05 mg to about 30 mg gaboxadol or a pharmaceutically acceptable salt thereof wherein the method provides improvement in the patient for more than 6 hours after administration. 
     
     
         2 . The method of  claim 1 , wherein the developmental disorder is selected from the group consisting of an Autistic Spectrum Disorder, pervasive developmental disorder, Autism, Angelman syndrome, Fragile X syndrome, Fragile X-associated tremor/ataxia syndrome (FXTAS), Rett syndrome, Asperger's syndrome, Childhood Disintegrative Disorder, Lanau-Kleffner Syndrome, Prader-Willi Syndrome, Tardive Dyskinesia, and Williams Syndrome. 
     
     
         3 . The method of  claim 1 , wherein the developmental disorder is Angelman syndrome. 
     
     
         4 . The method of  claim 1 , wherein the developmental disorder is Fragile X syndrome. 
     
     
         5 . The method of  claim 1 , wherein the developmental disorder is Fragile X-associated tremor/ataxia syndrome (FXTAS). 
     
     
         6 . The method of  claim 1 , wherein the patient is administered a composition comprising about 1 mg to about 15 mg gaboxadol or a pharmaceutically acceptable salt thereof. 
     
     
         7 . The method of  claim 1 , wherein the patient is administered a composition comprising about 1 mg to about 10 mg gaboxadol or a pharmaceutically acceptable salt thereof. 
     
     
         8 . The method of  claim 1 , wherein the patient is administered a composition comprising about 1 mg to about 5 mg gaboxadol or a pharmaceutically acceptable salt thereof. 
     
     
         9 . The method of  claim 1 , wherein the in vivo plasma profile of the patient 6 hours after administration of the gaboxadol or pharmaceutically acceptable salt thereof is reduced by more than 50%. 
     
     
         10 . The method of  claim 1 , wherein the AUC 6-12  of the patient 6 hours after administration of the gaboxadol or pharmaceutically acceptable salt thereof is less than 75% of the administered dose. 
     
     
         11 . The method of  claim 1 , wherein the method provides improvement in at least one symptom selected from the group consisting of ataxia, gait, speech impairment, vocalization, cognition, motor activity, clinical seizure, hypotonia, hypertonia, feeding difficulty, drooling, mouthing behavior, sleep difficulties, hand flapping, easily provoked laughter and short attention span. 
     
     
         12 . The method of  claim 1 , wherein the method provides improvement in the patient for more than 6 hours. 
     
     
         13 . The method of  claim 1 , wherein the method provides improvement in the patient for more than 8 hours. 
     
     
         14 . The method of  claim 1 , wherein the composition provides improvement in the patient for at least 12 hours. 
     
     
         15 . A method of treating a developmental disorder comprising administering to a patient in need thereof gaboxadol or a pharmaceutically acceptable salt thereof wherein the method provides an in vivo plasma profile comprising a C max  less than about 400 ng/ml and wherein the method provides improvement in the patient for more than 6 hours after administration of the gaboxadol or a pharmaceutically acceptable salt thereof. 
     
     
         16 . A method of treating a developmental disorder comprising administering to a patient in need thereof gaboxadol or a pharmaceutically acceptable salt thereof wherein the method provides an in vivo plasma profile comprising a AUC 6-12  of less than about 900 ng·hr/ml and wherein the method provides improvement in the patient for more than 6 hours after administration of the gaboxadol or a pharmaceutically acceptable salt thereof. 
     
     
         17 . A method of treating a developmental disorder comprising administering to a patient in need thereof a first pharmaceutical composition comprising gaboxadol or a pharmaceutically acceptable salt thereof and a second pharmaceutical composition comprising gaboxadol or a pharmaceutically acceptable salt thereof wherein the second pharmaceutical composition provides an in vivo plasma profile comprising a mean AUC 0-∞  of at least 20% less than the first pharmaceutical composition. 
     
     
         18 . The method of  claim 17 , wherein the second pharmaceutical composition provides an in vivo plasma profile comprising a mean AUC 0-∞  of less than about 900 ng·hr/ml. 
     
     
         19 . The method of  claim 17 , wherein the C max  of the first pharmaceutical composition is more than 20% greater than the C max  of the second pharmaceutical composition. 
     
     
         20 . The method of  claim 17 , wherein the T max  of the first pharmaceutical composition is less than 2 hours. 
     
     
         21 . The method of  claim 17 , wherein the first pharmaceutical composition comprises a dissolution of at least 80% within the first 10 minutes. 
     
     
         22 . The method of  claim 17 , wherein the first pharmaceutical composition and the second pharmaceutical composition are provided in a single dosage form. 
     
     
         23 . The method of  claim 17 , wherein the patient is provided about 0.05 mg/kg/day to about 5 mg/kg/day gaboxadol or a pharmaceutically acceptable salt thereof in the first pharmaceutical composition. 
     
     
         24 . The method of  claim 17 , wherein the first pharmaceutical composition comprises about 0.05 mg to about 30 mg gaboxadol or a pharmaceutically acceptable salt thereof. 
     
     
         25 . The method of  claim 17 , wherein the second pharmaceutical composition comprises about 0.05 mg to about 30 mg gaboxadol or a pharmaceutically acceptable salt thereof.