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

Primary Purpose

Autism, Autistic Disorder, Asperger's Disorder

Status
Unknown status
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Gammaplex 5%
Sponsored by
Isaac Melamed
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autism focused on measuring Autism, Autism spectrum, Autism children

Eligibility Criteria

4 Years - 12 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • IMMUNOe International Research Centers

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Gammaplex 5% IGIV

Arm Description

Gammaplex 5% IGIV administered intravenously

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
July 21, 2014
Last Updated
April 15, 2015
Sponsor
Isaac Melamed
Collaborators
Bio Products Laboratory
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1. Study Identification

Unique Protocol Identification Number
NCT02199925
Brief Title
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

2. Study Status

Record Verification Date
March 2015
Overall Recruitment Status
Unknown status
Study Start Date
June 2013 (undefined)
Primary Completion Date
April 2015 (Anticipated)
Study Completion Date
July 2015 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Isaac Melamed
Collaborators
Bio Products Laboratory

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism, Autistic Disorder, Asperger's Disorder, PDD, Pervasive Developmental Disorder
Keywords
Autism, Autism spectrum, Autism children

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Gammaplex 5% IGIV
Arm Type
Experimental
Arm Description
Gammaplex 5% IGIV administered intravenously
Intervention Type
Drug
Intervention Name(s)
Gammaplex 5%
Primary Outcome Measure Information:
Title
The primary objective is to assess the efficacy of Gammaplex as an immunomodulatory therapy in autistic children with evidence of immune dysfunction.
Description
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
Time Frame
1 year
Secondary Outcome Measure Information:
Title
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 Pre-specified Outcome Measures:
Title
To improve development in autistic spectrum
Time Frame
1 year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Isaac Melamed, MD
Organizational Affiliation
IMMUNOe International Clinical Research Centers
Official's Role
Principal Investigator
Facility Information:
Facility Name
IMMUNOe International Research Centers
City
Centennial
State/Province
Colorado
ZIP/Postal Code
80112
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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