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

Isaac Melamed

Status and phase: Unknown; Phase 4

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

Treatments: Drug: Gammaplex 5%

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02199925

GMXAUT01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 30 estimated patients

Sex: All

Ages: 4 to 12 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

Gammaplex 5% IGIV

Experimental group

Description:

Gammaplex 5% IGIV administered intravenously

Treatment:

Drug: Gammaplex 5%