AutismPhase 4NCT02199925
An Open-Label Study to Evaluate the Efficacy of High-Dose Gammaplex in Children on the Autism Spectrum
A Phase 4 study testing Gammaplex 5% in people with Autism and Autistic Disorder, run by Isaac Melamed at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 12 years
- Conditions the study is for
- Autism, Autistic Disorder, Asperger's Disorder
- What is being tested?
- Gammaplex 5%
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2013to Jul 2015
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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).
Who cannot join
- A diagnosis of isolated IGA deficiency
- Allergic reactions to blood products
Trial locations and contacts
1 site in 1 country.
United States1 site
IMMUNOe International Research Centers
Centennial, Colorado, 80112
About this study
Official title: 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.
Read the 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.
- Being tested
- Gammaplex 5%
- Keywords
- Autism, Autism spectrum, Autism children
- Medical subject headings
- Autistic Disorder, Asperger Syndrome, Child Development Disorders, Pervasive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gammaplex 5%Drug
| Study group | Role | What they receive |
|---|---|---|
| Gammaplex 5% IGIV | Experimental | Gammaplex 5% IGIV administered intravenously Receives: Drug: Gammaplex 5% |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2014 (estimated)
- Study startJun 2013
- Primary completionApr 2015 (estimated)
- Study completionJul 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AutismStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Isaac Melamed
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Bio Products Laboratory
- Responsible party
- Sponsor-investigator, Isaac Melamed, IMMUNOe Research Centers
- Investigators
- Isaac Melamed, MD, Principal investigator, IMMUNOe International Clinical Research Centers
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02199925
- Sponsor's protocol ID
- GMXAUT01
- Status last verified
- Mar 2015
- First submitted
- 21 Jul 2014
- Last updated
- 16 Apr 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IV, Single-site, Open-Label Study to Evaluate the Efficacy of High-Dose Gammaplex in Children on the Autism Spectrum
- ClinicalTrials.gov ID
- NCT02199925
- Acronym / sponsor ID
- GMXAUT01
- Keywords
- Autism, Autism spectrum, Autism children
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Isaac MelamedIndustry (pharma, biotech, devices)
- Collaborators
- Bio Products Laboratory
- Study start
- Jun 2013
- Primary completion
- Apr 2015 (estimated)
- Study completion
- Jul 2015 (estimated)
- First posted
- 25 Jul 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gammaplex 5% IGIV | Experimental | Drug: Gammaplex 5%Gammaplex 5% IGIV administered intravenously |
Who may qualify
- Ages
- 4 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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).
Key exclusion criteria (2 listed)
- A diagnosis of isolated IGA deficiency
- Allergic reactions to blood products
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Gammaplex 5% | Drug | Gammaplex 5% IGIV |
Primary outcome
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 secondary outcome
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Isaac Melamed, MD principal investigator
IMMUNOe International Clinical Research Centers
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Gammaplex 5% in people with Autism and Autistic Disorder, run by Isaac Melamed at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2015.