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University of California, San Diego (UCSD) Suramin Treatment Trial for Autism

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Robert K. Naviaux, University of California, San Diego
ClinicalTrials.gov Identifier:
NCT02508259
First received: July 22, 2015
Last updated: April 25, 2016
Last verified: April 2016
  Purpose
This study is designed to test the safety and efficacy of a single, intravenous dose of suramin in autism spectrum disorders (ASD).

Condition Intervention Phase
Autism Spectrum Disorders
Drug: Suramin
Drug: Saline
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: The UCSD Suramin Autism Treatment Trial

Resource links provided by NLM:


Further study details as provided by Robert K. Naviaux, University of California, San Diego:

Primary Outcome Measures:
  • Joint social attention [ Time Frame: 2 days to 6 weeks ]
    ADOS (autism diagnostic observation schedule) reciprocal social interaction domain scores

  • Receptive language [ Time Frame: 2 days to 6 weeks ]
    Peabody picture vocabulary test (PPVT)


Secondary Outcome Measures:
  • Social interaction [ Time Frame: 2 days to 6 weeks ]
    Blinded examiner scoring of video recorded ADOS evaluations using elements of the Communication and Symbolic Behavior Scales (CSBS)

  • Expressive language [ Time Frame: 2 days to 6 weeks ]
    Expressive one word picture vocabulary test (EOWPVT)

  • Cortical connectivity [ Time Frame: 2 days to 6 weeks ]
    EEG power and coherence analysis

  • Autonomic nervous system balance [ Time Frame: 2 days to 6 weeks ]
    EKG analysis of heart rate variability

  • Praxia and Motor coordination [ Time Frame: 2 days to 6 weeks ]
    Fine motor and gross motor digital balance board and dynamic gait analysis

  • Aberrant behaviors [ Time Frame: 2 days to 6 weeks ]
    Aberrant behavior checklist (ABC)

  • Autism core and secondary symptoms [ Time Frame: 2 days to 6 weeks ]
    Autism treatment evaluation checklist (ATEC)

  • The Clinical Global Impression - improvement scale (CGI-I) [ Time Frame: 2 days to 6 weeks ]
    7-point scale reflecting the change in core autism behaviors

  • Global assessment of child developmental behaviors [ Time Frame: 2 days to 6 weeks ]
    Child behavior checklist (CBCL)

  • Repetitive Behaviors [ Time Frame: 2 days to 6 weeks ]
    Repetitive behavior questionnaire (RBQ)


Enrollment: 10
Study Start Date: May 2015
Study Completion Date: April 2016
Primary Completion Date: March 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Suramin
20 mg/kg suramin in 50 ml of saline by intravenous infusion over 30 minutes
Drug: Suramin
20 mg/kg IV in 50 ml saline over 30 minutes
Other Name: Germanin
Placebo Comparator: Saline
50 ml of saline by intravenous infusion over 30 minutes
Drug: Saline
50 ml IV over 30 minutes
Other Name: Normal saline

Detailed Description:
This study is designed to test a new theory of the origin and treatment of ASD. In this theory, ASD is caused by both genes and environment interacting to produce a persistent cell danger response (CDR; Naviaux RK, 2014) that interferes with and alters normal child brain development. Gut microbiome and immune systems are also affected. In this theory, the pathological persistence of the cell danger response is traceable to mitochondria, and maintained by purinergic signaling mediated by the release of extracellular nucleotides like adenosine triphosphate (ATP), adenosine diphosphate (ADP), uridine triphosphate (UTP), and uridine diphosphate (UDP). Suramin inhibits excess purinergic signaling by acting as a competitive inhibitor of nucleotide signaling at both ionotropic purinergic (P2X) receptors, and G-protein coupled, metabotropic purinergic (P2Y) receptors. Suramin has been found to correct the symptoms, metabolism, and brain synaptic abnormalities in two classical genetic and environmental mouse models of autism (Naviaux JC, et al. 2015; Naviaux JC, et al. 2014; Naviaux RK, et al. 2013). This study will test the safety and efficacy of a single dose of suramin in children with ASD. While it is not anticipated that a single dose will produce benefits for more than a few weeks, if successful, this study may lead to the development of newer and safer drugs for autism treatment.
  Eligibility

Ages Eligible for Study:   4 Years to 17 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Autism diagnostic observation schedule (ADOS) score of ≥ 7
  • Diagnosis of autism spectrum disorder by Diagnostic and Statistical Manual, 5th edition (DSM-V)
  • Stable treatment and diet regimen for ≥ 2 months
  • Resident of San Diego region

Exclusion Criteria:

  • Any prescription medications
  • Hospitalization within the previous 2 months
  • Active medical problem such as seizures, heart, liver, kidney, or adrenal disease
  • Planning to start a new drug, diet, or behavioral intervention during the study
  • Weight under the 5th percentile for age
  • Unable to tolerate venipuncture, urine collection, or an indwelling intravenous catheter for 3-4 hours
  • Plasma creatinine ≥ 1.4 mg/dl
  • Liver function alanine amino transferase (ALT) or aspartate amino transferase (AST) ≥ 1.5-fold above the upper limit of normal
  • Known intolerance to suramin or other antipurinergic drugs
  • Unable to perform or cooperate with study requirements
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02508259

Locations
United States, California
University of California, San Diego School of Medicine
La Jolla, California, United States, 92093
Sponsors and Collaborators
University of California, San Diego
Investigators
Principal Investigator: Robert K Naviaux, MD, PhD University of California, San Diego
  More Information

Publications:
Responsible Party: Robert K. Naviaux, Principal Investigator, University of California, San Diego
ClinicalTrials.gov Identifier: NCT02508259     History of Changes
Other Study ID Numbers: 15-0134
Study First Received: July 22, 2015
Last Updated: April 25, 2016
Individual Participant Data  
Plan to Share IPD: Yes
Plan Description: Safety, metabolomic, and completed outcome data will be made available to qualified institutional groups after peer review and publication.

Keywords provided by Robert K. Naviaux, University of California, San Diego:
mitochondria
purinergic signaling
cell danger response

Additional relevant MeSH terms:
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Suramin
Antinematodal Agents
Anthelmintics
Antiparasitic Agents
Anti-Infective Agents
Antineoplastic Agents
Trypanocidal Agents
Antiprotozoal Agents

ClinicalTrials.gov processed this record on May 25, 2017