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

NCT02199925 | Unknown Phase 4 DMC

• 1 other identifier: GMXAUT01
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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.

Conditions

Autism; Autistic Disorder; Asperger's Disorder; PDD; Pervasive Developmental Disorder

Keywords

Autism; Autism spectrum; Autism children

Outcome Measures

Primary Outcomes (1)

• 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 1. Caregiver Scales (SRS, CCC-2, ABC) 2. CGI-S and CGI - I 3. PPVT 4. ADOS

  1 year

Secondary Outcomes (1)

• The modulatory effect of IVIG on neuroinflammation will be assessed by laboratory measurements which we believe are indicative of immune dysregulation.

  1 year

Other Outcomes (1)

• To improve development in autistic spectrum

  1 year

Study Arms (1)

Gammaplex 5% IGIV

EXPERIMENTAL

Gammaplex 5% IGIV administered intravenously

Drug: Gammaplex 5%

Interventions

Gammaplex 5% DRUG

Gammaplex 5% IGIV

Eligibility Criteria

• Age: 4 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).

You may not qualify if:

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

Sponsors & Collaborators

• Isaac Melamed: lead
• Bio Products Laboratory: collaborator

Study Sites (1)

IMMUNOe International Research Centers

Centennial, Colorado, 80112, United States

Location

MeSH Terms

Conditions

Autistic Disorder; Asperger Syndrome; Child Development Disorders, Pervasive

Condition Hierarchy (Ancestors)

Autism Spectrum Disorder; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Isaac Melamed, MD

  IMMUNOe International Clinical Research Centers

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 21, 2014
• First Posted: July 25, 2014
• Study Start: June 1, 2013
• Primary Completion: April 1, 2015
• Study Completion: July 1, 2015
• Last Updated: April 16, 2015

Record last verified: 2015-03

Locations

US (1)