Aevi Genomic Medicine Provides Update on Sample Size Re-estimation for Phase 2 ASCEND Trial in ADHD
Published: Jun 12, 2018
Aevi Genomic Medicine, Inc. (NASDAQ: GNMX) (the Company) today announced that it will increase the sample size for Part A of its Phase 2 ASCEND clinical trial, which is enrolling a mGluR mutation positive genetic subset of pediatric and adolescent patients with Attention Deficit Hyperactivity Disorder (ADHD), to confirm response to AEVI-001 in these patients.
The decision to increase the sample size was made according to the protocol defined sample size re-estimatation design, which allows an adjustment in the sample size after an interim analysis of the placebo arm to ensure the trial is appropriately powered. As a result of the interim analysis performed by an independent unblinded statistician, the sample size will be increased from 42 to 64 patients.
"The prespecified, interim analysis and sample size re-estimation was planned as part of the study, allowing us to evaluate the placebo effect," said Garry A. Neil, M.D., Chief Scientific Officer of Aevi Genomic Medicine. "While we saw a high placebo effect in the overall SAGA trial, we observed a considerably lower placebo effect in the 8-gene subset. Sample size re-estimation allowed us to efficiently assess the placebo response assumption in a prospective trial and adapt the sample size as needed. Based on this analysis, we will enroll 64 patients to ensure sufficient power to detect a robust treatment effect. We have continued recruitment activities based on the current design to maintain momentum, surpassing 42 patients, and we look forward to completing enrollment and announcing topline results in 4Q18."
ASCEND is an adaptive, 6-week, double-blind parallel-group study in children and adolescents (ages 6-17 years) with ADHD with and without copy number variants (CNVs) in specific genes implicated in glutamatergic signaling and neuronal connectivity. Part A includes subjects determined to have one of 8 specific gene mutation(s) implicated in glutamatergic signaling and neuronal connectivity. Part B will assess subjects who do not have CNVs in any of the specific gene mutation(s) implicated in glutamatergic signaling and neuronal connectivity. Once subjects are confirmed as eligible for each part of the study, they are randomized to one of two treatment groups (AEVI-001 or placebo) in a 1:1 ratio.
AEVI-001 is an oral non-stimulant pan selective activator/modulator of mGluRs. The molecule has excellent pharmacokinetic and metabolic profiles and crosses the blood brain barrier.
Aevi Genomic Medicine remains committed to the development of AEVI-001 as a potential treatment for a sub-population of Attention Deficit Hyperactivity Disorder (ADHD) patients with genetic mutations that disrupt the mGluR network. In the US, the CDC estimates that 6.4 million children 4-17 years of age (11%) have ever been diagnosed with ADHD. Many ADHD patients remain unsatisfied with existing therapies, particularly with respect to safety, tolerability and treatment of comorbidities.
AEVI-001 is an investigational agent that has not been approved by the US FDA or any other regulatory agencies.
About Aevi Genomic Medicine, Inc.
Aevi Genomic Medicine, Inc. is dedicated to unlocking the potential of genomic medicine to translate genetic discoveries into novel therapies. Driven by a commitment to patients with pediatric onset life-altering diseases, the Company's research and development efforts leverage an internal genomics platform and an ongoing collaboration with the Center for Applied Genomics at The Children's Hospital of Philadelphia. Based on discoveries from the genomics collaboration, the Company currently has two programs in clinical development, AEVI-001 for mGLuR+ ADHD and AEVI-002 for severe pediatric onset Crohn's disease.
More information on the Company and pipeline is located on its website www.aevigenomics.com.
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SOURCE Aevi Genomic Medicine, Inc.