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

April 15, 2015 updated by: Isaac Melamed

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

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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

Enrollment (Anticipated)

30

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Colorado
      • Centennial, Colorado, United States, 80112
        • IMMUNOe International Research Centers

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years to 12 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Gammaplex 5% IGIV
Gammaplex 5% IGIV administered intravenously
Drug: Gammaplex 5%

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
1 year

Secondary Outcome Measures

Outcome Measure
Time Frame
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
1 year

Other Outcome Measures

Outcome Measure
Time Frame
To improve development in autistic spectrum
Time Frame: 1 year
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Isaac Melamed

Collaborators

Bio Products Laboratory

Investigators

  • Principal Investigator: Isaac Melamed, MD, IMMUNOe International Clinical Research Centers

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2013

Primary Completion (Anticipated)

April 1, 2015

Study Completion (Anticipated)

July 1, 2015

Study Registration Dates

First Submitted

July 21, 2014

First Submitted That Met QC Criteria

July 23, 2014

First Posted (Estimate)

July 25, 2014

Study Record Updates

Last Update Posted (Estimate)

April 16, 2015

Last Update Submitted That Met QC Criteria

April 15, 2015

Last Verified

March 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GMXAUT01

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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