Clinical trial NCT02199925: An Open-Label Study to Evaluate the Efficacy of High-Dose Gammaplex in Children on the Autism Spectrum
Official title: A Phase IV, Single-site, Open-Label Study to Evaluate the Efficacy of High-Dose Gammaplex in Children on the Autism Spectrum
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Autism
Autistic Disorder
Asperger's Disorder
PDD
Pervasive Developmental Disorder
Keywords: Autism
Autism spectrum
Autism children
Lead sponsor: IMMUNOe Research Centers
Sponsor class: INDUSTRY
Collaborators: name: Bio Products Laboratory
First posted: 2014-07-25
Enrollment: 30
Sex: ALL
Minimum age (years): 4
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: If autism is the consequence of a chronic inflammatory process preventing the infant brain from forming the proper neural connections, then treatment that reduces inflammation might have a positive impact on autism. IGIV treatment has been known to suppress inflammation and has been used in the treatment of inflammatory conditions and autoimmune diseases.

Detailed description: We have come to believe that an immature immune system with abnormal signaling and altered apoptotic pathways may result in a process of neuroinflammation with a clinical presentation of Autism Spectrum Disorder. If Autism Spectrum Disorders are the consequence of a chronic inflammatory process that prevents the infant brain from forming the proper neural connections, then treatment that reduces inflammation might have a positive impact on autism. We believe that there is a correlation between immunological abnormalities and the development of the autism.

Eligibility criteria: Inclusion Criteria:

* The subject is between the ages of or equal to 4 and 12 years of age, of either gender, and belonging to any ethnic group.
* The subject has a diagnosis, for a minimum of 6 months prior to screening, of either autistic disorder, Asperger disorder, or pervasive developmental disorder (PDD).
* Normal physical test results.
* Immunological lab results showing reduced levels of activated CD40L or abnormal lymphocyte stimulation, or T or B cell dysfunction (hypgammaglobulinemia, primary immunodeficiency (PIDD), or common variable immune deficiency (CVID).

Exclusion Criteria:

* A diagnosis of isolated IGA deficiency
* Allergic reactions to blood products

Primary outcomes:
measure: The primary objective is to assess the efficacy of Gammaplex as an immunomodulatory therapy in autistic children with evidence of immune dysfunction.
timeFrame: 1 year
Secondary outcomes:
measure: The modulatory effect of IVIG on neuroinflammation will be assessed by laboratory measurements which we believe are indicative of immune dysregulation.
timeFrame: 1 year
Other outcomes:
measure: To improve development in autistic spectrum
timeFrame: 1 year
Locations:
city: Centennial
country: United States
facility: IMMUNOe International Research Centers
state: Colorado
Study officials:
affiliation: IMMUNOe International Clinical Research Centers
name: Isaac Melamed, MD
role: PRINCIPAL_INVESTIGATOR
References: