September 13, 2010
Seaside Therapeutics released positive results from a phase II trial of STX209 for the treatment of autism spectrum disorders. This open label trial enrolled 32 pediatric subjects, 6-17 years of age. Dosing was conducted as a flexible titration over two weeks to the optimal titrated dose (OTD). The starting dose was 1 mg twice a day and was gradually increased up to 10 mg three times a day for subjects greater than 11 years of age. OTD was continued for the remainder of the eight-week treatment period. Data are from the per-protocol population of 25 subjects. The primary endpoint, an improvement on the Irritability subscale of the Aberrant Behavior Checklist, achieved statistical significance (p<0.001). STX209 also demonstrated statistically significant improvements across a number of other global and specific neurobehavioral outcomes, including improvements on the Social Withdrawal subscale of the Aberrant Behavior Checklist (p<0.001), which assesses a core symptom of ASD. STX209 was well tolerated.
January 12, 2004
Repligen Corporation reported negative results from a phase III trial investigating RG1068, a synthetic human secretin for the treatment of autism. Results showed the data failed to meet the dual primary endpoints of social interaction improvements, measured by the Autism Diagnostic Observation Schedule (ADOS) and the parental Clinical Global Impression of Change (CGI). Data demonstrated a statistically significant improvement with RG1068 versus placebo on ADOS but not on the CGI. The study also showed a higher placebo effect than was observed in an earlier study. The double-blind, placebo-controlled, study enrolled 132 children with moderate to severe symptoms of autism at 15 sites in the U.S. Subjects received six injections of RG1068 or placebo for 18 weeks.
October 21, 2002
Repligen reported positive results from an open label, extension study of a phase II clinical trial for RG1068, a synthetic secretin investigated in children with autism. The goal of the study was to examine long term safety over an 18-week period. There were no serious adverse events in either the RG1068 or placebo-treated groups and no difference in the number of adverse behaviors including hyperactivity, sleep disruption, increased irritability or aggressiveness between the two groups. Repligen is currently sponsoring a phase II clinical trial of RG1068 in young children with autism.