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Trial record 2 of 21 for:    suramin

University of California, San Diego (UCSD) Suramin Autism Treatment-1 (SAT1) Trial (SAT1)

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ClinicalTrials.gov Identifier: NCT02508259
Recruitment Status : Completed
First Posted : July 24, 2015
Results First Posted : July 16, 2019
Last Update Posted : July 16, 2019
Sponsor:
Information provided by (Responsible Party):
Robert K. Naviaux, University of California, San Diego

Brief Summary:
This study is designed to test the safety and efficacy of a single, intravenous dose of suramin in autism spectrum disorders (ASD).

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorders Drug: Suramin Drug: Saline Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: The UCSD Suramin Autism Treatment-1 (SAT1) Trial
Study Start Date : May 2015
Actual Primary Completion Date : March 2016
Actual Study Completion Date : April 2016

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. Autism Diagnostic Observation Schedule, 2nd Edition (ADOS2) [ Time Frame: 6 weeks compared to baseline ]
    ADOS2 comparison scores are units on a scale of 0-10. A score of 7-10 was required for enrollment. A score of 7-10 is diagnostic for autism spectrum disorder (ASD). The higher the score, the more severe the core symptoms of autism spectrum disorder. Scores of 6 and below are considered off the ASD spectrum.

  2. Expressive Language [ Time Frame: 6 weeks compared to baseline ]
    Expressive One Word Picture Vocabulary Test (EOWPVT) scores are normalized for age. Typical language development produces a mean score of 100 with a standard deviation of 15. Outcomes for EOWPVT were expressed as the mean of the child-specific difference before and 6-weeks after treatment. For example, if the 6-week standard EOWPVT score was 59.6 and the baseline score was 63.8, the difference is -4.2 (= 59.6 - 63.8). A decrease in score at 6 weeks would corresponds to a decrease in language performance, while an increase, a positive difference, would reflect an increase.


Secondary Outcome Measures :
  1. Aberrant Behavior Checklist (ABC) [ Time Frame: 6 weeks compared to baseline ]
    The full ABC is a 58-item parent rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. Stereotypy is reported, and scores range from 0 to 21, with higher scores indicating worse behavior. A negative difference corresponds to decreased symptoms after treatment. A positive difference corresponds to increased symptoms after treatment.

  2. Autism Treatment Evaluation Checklist (ATEC) [ Time Frame: 6 weeks ]
    The reported value is the Language sub-score of the ATEC, and the range for the language sub-score is 0-20. The higher the score, the worse the disability. Outcomes were measured at 6 weeks after treatment compared to baseline. A negative difference corresponds to a decrease in language disability, i.e an improvement in speech and language. A positive difference reflects an increase in language disability, i.e. a decrease in speech and language.

  3. The Clinical Global Impression - Improvement Scale (CGI-I) [ Time Frame: Overall ASD symptoms at 6 weeks ]
    The CGI-I is scale that ranges from 1-7, reflecting the change in core autism behaviors after treatment. 1 is much improved, 4 is unchanged, and 7 is much worse.

  4. Repetitive Behavior Questionnaire [ Time Frame: 6 weeks compared to baseline ]
    Total repetitive behavior was assessed using the Repetitive behavior questionnaire (RBQ), which has a scale from 0-87. Higher scores correspond to more severe repetitive behavior. Outcomes were analyzed as the difference in the score 6 weeks after treatment compared to baseline. A negative difference corresponds to improved behavior compared to baseline. A positive difference corresponds to worse behavior.



Information from the National Library of Medicine

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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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02508259


Locations
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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
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Principal Investigator: Robert K Naviaux, MD, PhD University of California, San Diego
Additional Information:
Publications:
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Responsible Party: Robert K. Naviaux, Principal Investigator, University of California, San Diego
ClinicalTrials.gov Identifier: NCT02508259    
Other Study ID Numbers: 15-0134
First Posted: July 24, 2015    Key Record Dates
Results First Posted: July 16, 2019
Last Update Posted: July 16, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
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:
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Suramin
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Antinematodal Agents
Anthelmintics
Antiparasitic Agents
Anti-Infective Agents
Antineoplastic Agents
Trypanocidal Agents
Antiprotozoal Agents