Clinical Trials Resource Center


October 16, 2017

Neurim Pharmaceuticals presented results of the pivotal placebo-controlled phase III study of pediatric prolonged-release melatonin formulation (PedPRM) in Autism Spectrum Disorder (ASD) children (age 2-18 years) who suffer from insomnia. The phase III study was a randomized, double-blind, parallel group, multicenter (EU and U.S.) study in children with ASD or neurogenetic diseases and sleep disorders. One hundred and twenty-five patients who had not shown improvement after standard sleep behavioral intervention, received two weeks placebo run-in, and were then randomized to PedPRM (2mg with optional increase to 5mg) or placebo in the evening, for 13 weeks. Completers received PedPRM open-label for additional 13 weeks. The study’s primary endpoint showed a clinically meaningful and statistically significant increase in total sleep time (p=0.034). Participants slept on average 57.5 minutes longer at night with PedPRM compared to 9.14 with placebo. Sleep latency (SL) decreased by 39.6 minutes on average with PedPRM compared to 12.5 with placebo (p=0.011) without causing earlier wakeup time. Sleep maintenance, parents’ quality of life and satisfaction of child’s sleep also improved significantly. The beneficial effects of PedPRM were maintained over the long term. Furthermore, compliance with the age-appropriate PedPRM formulation was good and it had a favorable safety profile over the short and long term period.

November 14, 2016

Neurim Pharmaceuticals announced results from its NEU_CH_7911 phase III study of PedPRM for prolonged-release melatonin (PedPRM) for sleep disturbances in children with Autism spectrum disorders (ASD). This was a randomized, double-blind, placebo-controlled, parallel group, multicenter (EU and U.S.) study in children with ASD or neurogenetic diseases and sleep disorders. Patients (125), who had not shown improvement practicing sleep hygiene, received two weeks placebo run-in, and then randomized to PedPRM (2mg with optional increase to 5mg) or placebo in the evening, for 13 weeks. Completers received PedPRM open-label for additional 13 weeks. Primary efficacy endpoint was defined as the difference between PedPRM and placebo in mean change from run-in to the end of double-blind treatment period, in parent-reported TST (Daily Sleep and Nap Diary). PedPRM met the primary efficacy endpoint demonstrating statistically significant improvement in total sleep time (TST) compared to placebo. In addition to TST, secondary efficacy endpoints demonstrating improvements in sleep initiation and maintenance were also met. The safety profile was similar between PedPRM and placebo-treated groups.

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.

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