Secretin for the Treatment of Autism
Condition: Autism
MeSH terms: Autistic Disorder
Study type: Interventional
Phase: Phase 3

Summary: 
      Many drugs used to treat autism target specific symptoms, such as hyperactivity and
      aggressiveness. Few drugs target the core autistic symptoms of impaired social interaction
      and communication. This study will evaluate two forms of the drug secretin for the treatment
      of core autistic symptoms.
    

Detailed description: 
      Autism is a disorder characterized by impairments of social interactions, verbal and
      nonverbal communication, and preoccupation with unusual activities or interests, particularly
      stereotyped or repetitive movements. This debilitating disorder is estimated to occur in 2 to
      10 of every 10,000 births. A primary focus in pyschopharmacological intervention has been to
      treat specific associated symptoms, such as hyperactivity, aggressiveness, and temper
      tantrums; there are few pharmacologic treatments directed toward core autistic symptoms.

      Secretin is a gut hormone with binding sites in the brain. Previous research has described
      three patients with autism who underwent diagnostic endoscopy for gastrointestinal complaints
      and experienced dramatic improvement in autistic symptoms following the administration of
      intravenous secretin given as part of endoscopy. Though the results of this single,
      uncontrolled study have limited interpretive value, many autistic children have been exposed
      and continue to be exposed to this potential treatment in an uncontrolled manner. This double
      blind, placebo-controlled trial will evaluate the safety and efficacy of secretin for the
      treatment of autism.

      Participants will be randomly assigned to one of three treatment groups: synthetic porcine
      secretin (sPS), biologically derived porcine secretin (bPS), or placebo. Each participant
      will be given an initial intravenous dose of 0.1 ml of their assigned treatment at the
      appropriate dose (0.2 ug for sPS and 1 CU for bPS). If no allergic reaction occurs within one
      minute, the participants will continue in the study and receive the full remaining dose over
      one minute. Participants will be evaluated one week before and four weeks after infusion for
      social, communication, and behavioral functioning as measured by Autistic Diagnostic
      Observation Schedule Generic (by blinded raters); Rimland Questionnaires (by parents and
      teachers); Expressive Vocabulary Test; MacArthur Communication Inventory (by parents and
      teachers); and Aberrant Behavior Checklist (by parents and teachers). Participants will also
      have a physical exam and blood and urine tests. After completion of preliminary data
      analysis, placebo patients will be offered open label therapy if appropriate.
    

Intervention name(s): secretin, synthetic porcine; secretin, biologically derived porcine
Intervention type(s): Drug; Drug
Intervention MeSH terms: Secretin
Allocation: Randomized
Intervention model: Factorial Assignment
Masking: Double
Primary purpose: Treatment
Keywords: Pervasive Developmental Disorders; Secretin, synthetic porcine; Secretin, biological porcine; Autism Diagnostic Observation Schedule - Generic (ADOS-G); Rimland Questionnaire; Expressive Vocabulary Test; MacArthur Communication Inventory; Aberrant Behavior Checklist