An Open-Label Study to Evaluate the Efficacy of High-Dose Gammaplex in Children on the Autism Spectrum

Autism Clinical Trial

Official title:

A Phase IV, Single-site, Open-Label Study to Evaluate the Efficacy of High-Dose Gammaplex in Children on the Autism Spectrum

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02199925
• Other study ID #: GMXAUT01
• Status: Active, not recruiting
• Phase: Phase 4
• First received: July 21, 2014
• Last updated: April 15, 2015
• Start date: June 2013
• Est. completion date: July 2015

Study information

• Verified date: March 2015
• Source: IMMUNOe International Research Centers
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 30
• Est. completion date: July 2015
• Est. primary completion date: April 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 4 Years to 12 Years

Eligibility

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

Study Design

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Asperger Syndrome
• Asperger's Disorder
• Autism
• Autistic Disorder
• Child Development Disorders, Pervasive
• Developmental Disabilities
• Disease
• PDD
• Pervasive Developmental Disorder

Intervention

Drug:
• Gammaplex 5%

Locations

Country	Name	City	State
United States	IMMUNOe International Research Centers	Centennial	Colorado

Sponsors (2)

Lead Sponsor	Collaborator
Isaac Melamed	Bio Products Laboratory

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	To improve development in autistic spectrum		1 year	No
Primary	The primary objective is to assess the efficacy of Gammaplex as an immunomodulatory therapy in autistic children with evidence of immune dysfunction.	The primary endpoint is disease improvement, as evidenced by standardized test measures across 3 intervals: screening for baseline measurements, visit 5, and end of study visit measurements. Primary efficacy variables will be measured using the following standardized tests; Caregiver Scales (SRS, CCC-2, ABC); CGI-S and CGI - I; PPVT; ADOS	1 year	No
Secondary	The modulatory effect of IVIG on neuroinflammation will be assessed by laboratory measurements which we believe are indicative of immune dysregulation.		1 year	No