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

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ClinicalTrials.gov Identifier: NCT02199925
Recruitment Status : Unknown
Verified March 2015 by Isaac Melamed, IMMUNOe Research Centers.
Recruitment status was:  Active, not recruiting
First Posted : July 25, 2014
Last Update Posted : April 16, 2015
Sponsor:
Collaborator:
Bio Products Laboratory
Information provided by (Responsible Party):
Isaac Melamed, IMMUNOe Research Centers

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.

Condition or disease Intervention/treatment Phase
Autism Autistic Disorder Asperger's Disorder PDD Pervasive Developmental Disorder Drug: Gammaplex 5% Phase 4

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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : June 2013
Estimated Primary Completion Date : April 2015
Estimated Study Completion Date : July 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Rhophylac

Arm Intervention/treatment
Experimental: Gammaplex 5% IGIV
Gammaplex 5% IGIV administered intravenously
Drug: Gammaplex 5%



Primary Outcome Measures :
  1. The primary objective is to assess the efficacy of Gammaplex as an immunomodulatory therapy in autistic children with evidence of immune dysfunction. [ Time Frame: 1 year ]

    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


Secondary Outcome Measures :
  1. The modulatory effect of IVIG on neuroinflammation will be assessed by laboratory measurements which we believe are indicative of immune dysregulation. [ Time Frame: 1 year ]

Other Outcome Measures:
  1. To improve development in autistic spectrum [ Time Frame: 1 year ]


Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02199925


Locations
United States, Colorado
IMMUNOe International Research Centers
Centennial, Colorado, United States, 80112
Sponsors and Collaborators
Isaac Melamed
Bio Products Laboratory
Investigators
Principal Investigator: Isaac Melamed, MD IMMUNOe International Clinical Research Centers

Responsible Party: Isaac Melamed, Principal Investigator, IMMUNOe Research Centers
ClinicalTrials.gov Identifier: NCT02199925     History of Changes
Other Study ID Numbers: GMXAUT01
First Posted: July 25, 2014    Key Record Dates
Last Update Posted: April 16, 2015
Last Verified: March 2015

Keywords provided by Isaac Melamed, IMMUNOe Research Centers:
Autism
Autism spectrum
Autism children

Additional relevant MeSH terms:
Disease
Autistic Disorder
Developmental Disabilities
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Asperger Syndrome
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders
gamma-Globulins
Immunoglobulins, Intravenous
Rho(D) Immune Globulin
Immunologic Factors
Physiological Effects of Drugs