A global clinical trial involving South Florida children is studying a drug that — if proven safe and effective — would deliver a dose of hope to the autism community.
ConnectMe, an international initiative with more than 85 study centers in the United States and 185 worldwide, is investigating the effects of memantine on children with autism, Asperger’s and atypical autism (also called pervasive developmental disorder not otherwise specified, or PDD-NOS). The hope is it will help treat the communication and social limitations at the heart of these disorders.
Billed as the largest study of its kind, ConnectMe is now enrolling South Florida children, according to Forest Laboratories Inc., the New York City-based pharmaceutical company leading the study.
If it’s shown to convincingly and safely improve trial participants’ impairments in interacting and communicating with others, researchers would seek the FDA’s nod as the first drug approved for offsetting autism’s core effects, said Dr. Peter Ventre, co-owner and principal investigator for Research Centers of America in Oakland Park, which is leading the study in South Florida.
“We are super-excited and super-optimistic,” said Ventre, whose research firm specializes in behavioral science. “We do a lot of autism studies, but this is nothing like we’ve seen before. Families are enthusiastic about this.”
Though there are medications approved for treating irritability, anxiety and aggressive behavior in children with autism, Ventre said, none have been cleared for mitigating impaired social interaction and communication skills.
Right now, memantine is approved only for the treatment of Alzheimer’s symptoms in the elderly, and the doses used in this study would be adjusted to levels appropriate for pediatric patients.
Children ages 6 to 12 with a confirmed diagnosis of autism, Asperger’s and PDD-NOS from throughout Palm Beach, Broward and Miami-Dade counties are invited to participate. The study would last 50 weeks and require up to 13 visits to Research Centers of America offices, according to the ConnectMe website, ConnectMeTrial.com.
Participating in the study is free and voluntary, and parents can decide to remove their children at any time.
Though Research Centers of America is the only South Florida location currently leading the study, other sites are expected to be added soon.
“Medications that target the core symptoms of communication and social interaction problems could have great impact on these individuals,” said Dr. Daniel L. Coury, medical director of the Autism Speaks Autism Treatment Network. “As improvement in these areas would decrease the need for some current treatments and increase the options and perhaps effectiveness for other non-medical treatments such as speech therapy.”
Research psychologist Judy Montero Dominguez of Sunrise said those limitations are her 12-year-old son’s “biggest handicap.”
“One of my biggest frustrations as a parent is that there’s really nothing that’s been proven effective for that,” Dominguez said, adding that finding such a drug “would give us hope that his quality of life would improve for him.”
Several local children have enrolled already in the study, Ventre said. The trial will remain open for enrollment for at least another year.
Overseen by the FDA and independent ethics committees, the ConnectMe trials would give memantine to about 192 children for at least 12 weeks. Those who show improvement may participate in a randomized withdrawal study with full or reduced doses or a placebo, the ConnectMe site says. Children whose symptoms worsen would stop taking the drug.
For more information about the study, or to find other trial sites, go to ConnectMeTrial.com or call 877-900-8735.