Clinical trial NCT00110708: Safety and Efficacy Study in the Treatment of Intestinal Problems Associated With Autism
Official title: A Randomized, Placebo-Controlled Phase II Trial Evaluating Safety and Efficacy of Oral Human Immunoglobulin in the Treatment of Gastrointestinal Dysfunction Associated With Autistic Disorder in Pediatric Patients From 2 to 18 Years of Age
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Autism
Autistic Disorder
Child Development Disorders, Pervasive
Gastrointestinal Diseases
Signs and Symptoms, Digestive
Keywords: autism
gastrointestinal dysfunction
autistic disorder
diarrhea
constipation
abdominal pain
gastrointestinal problems associated with autism
immune globulin
immunoglobulin
Immunoglobulins, Intravenous
Intestinal Mucosa/immunology/pathology
Administration, Oral
Autistic Disorder/*drug therapy
Child Behavior/*drug effects
Constipation/drug therapy
Diarrhea/drug therapy
Gastrointestinal Agents/*therapeutic use
Lead sponsor: PediaMed Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2005-05-13
Enrollment: 120
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine if human immunoglobulin given by mouth twice a day is effective in treating the persistent gastrointestinal (GI) problems such as diarrhea, constipation, abdominal pain, and bloating, in children with autism.

Detailed description: Autistic GI Dysfunction (AGID) is a term that describes a constellation of GI signs and symptoms often found in children with autistic disorder, including abdominal pain, constipation, chronic diarrhea, alternating constipation and diarrhea, gaseousness, bloating, and reflux.

The objective of this study is to assess the potential efficacy of oral immunoglobulin in reducing a wide range of GI symptoms in children and adolescents diagnosed with autistic disorder.

Eligibility criteria: Inclusion Criteria:

* Male or female from 2 years to 18 years of age (up to, but not inclusive of the 18th birthday)
* Diagnosis of autistic disorder corroborated by an Autism Diagnostic Interview - Revised (ADI-R) assessment performed by a certified investigator
* Physician Clinical Global Impression of Severity (of Autistic Disorder)
* History of chronic, persistent gastrointestinal disturbance
* No elective changes in medication, diet intervention, or behavioral therapy during the study (18 weeks total)

Exclusion Criteria:

* Evidence of a gastrointestinal infection or GI abnormality
* A known diagnosis of other gastrointestinal pathology
* Antibiotic and/or antifungal (e.g. nystatin) medication
* Chelation therapy
* Medication affecting gastrointestinal transit
* Planned use of prohibited drugs or agents that could affect GI transit
* Changes in diet intervention within 30 days prior to the screening visit
* Changes in alternative medical therapies or dietary supplements within 30 days prior to the screening visit
* Adding and/or changing behavior modification or psychotherapy during participation in the study
* Adding or changing psychotropic medication during participation in the study
* DSM-IV diagnosis of a pervasive developmental disorder other than autistic disorder
* Evidence of a seizure disorder, diagnosis of fragile X syndrome, tuberous sclerosis complex, liver disease, pancreatic disease, cystic fibrosis, or chronic infection
* Previous gastrointestinal surgery
* Pregnancy
* Participation in another investigational study
* Significant deviation from normal laboratory test values at baseline
* IgA deficiency (serum IgA \< 5 mg/dL)
* A history of severe hypersensitivity to human immunoglobulin
* Treatment with any human immunoglobulin and/or immunoglobulin products
* Any concurrent medication that would compromise subject's tolerance of drug or compliance with the protocol

Primary outcomes:
measure: Global improvement in gastrointestinal function
Secondary outcomes:
measure: Assessment of behavior (improvement and severity); additional assessments of gastrointestinal conditions
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Southwest Autism Research and Resource Center
state: Arizona
city: Phoenix
country: United States
facility: Center for Autism Research and Education
state: Arizona
city: Sacramento
country: United States
facility: University of California Davis, MIND Institute
state: California
city: Gainesville
country: United States
facility: Sarkis Clinical Trials
state: Florida
city: Gainesville
country: United States
facility: University of Florida HSC
state: Florida
city: Melbourne
country: United States
facility: International Child Development Resource Center
state: Florida
city: Orange City
country: United States
facility: Medical Research Group of Central Florida
state: Florida
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Kansas City
country: United States
facility: University of Kansas Medical Center, Developmental Disabilities Center
state: Kansas
city: Louisville
country: United States
facility: Bluegrass Clinical Research
state: Kentucky
city: Baton Rouge
country: United States
facility: Gulf Coast Research, LLC
state: Louisiana
city: Hingham
country: United States
facility: Hardy Health Associates
state: Massachusetts
city: Royal Oak
country: United States
facility: Pivotal Research Centers - Detroit
state: Michigan
city: St. Louis
country: United States
facility: Mercy Health Research
state: Missouri
city: Piscataway
country: United States
facility: Robert Wood Johnson Medical School
state: New Jersey
city: Rochester
country: United States
facility: Strong Center for Developmental Disabilities, University of Rochester Medical Center
state: New York
city: Akron
country: United States
facility: Children's Hospital Medical Center of Akron
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: Nisonger Center Ohio State University
state: Ohio
city: Pittsburgh
country: United States
facility: Merck Child Outpatient Clinic
state: Pennsylvania
city: San Antonio
country: United States
facility: North San Antonio Healthcare Associates
state: Texas
city: Seattle
country: United States
facility: Autism Spectrum Treatment and Research Center
state: Washington
Study officials:
References:
citation: Handen BL, Melmed RD, Hansen RL, Aman MG, Burnham DL, Bruss JB, McDougle CJ. A double-blind, placebo-controlled trial of oral human immunoglobulin for gastrointestinal dysfunction in children with autistic disorder. J Autism Dev Disord. 2009 May;39(5):796-805. doi: 10.1007/s10803-008-0687-y. Epub 2009 Jan 16.