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

NCT02508259 | Completed Phase 1 Results DMC

• 1 other identifier: 15-0134
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Autism Spectrum Disorders

Keywords

mitochondria; purinergic signaling; cell danger response

Outcome Measures

Primary Outcomes (2)

• Autism Diagnostic Observation Schedule, 2nd Edition (ADOS2)

  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.

  6 weeks compared to baseline

• Expressive Language

  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.

  6 weeks compared to baseline

Secondary Outcomes (4)

• Aberrant Behavior Checklist (ABC)

  6 weeks compared to baseline

• Autism Treatment Evaluation Checklist (ATEC)

  6 weeks

• The Clinical Global Impression - Improvement Scale (CGI-I)

  Overall ASD symptoms at 6 weeks

• Repetitive Behavior Questionnaire

  6 weeks compared to baseline

Study Arms (2)

Suramin

ACTIVE COMPARATOR

20 mg/kg suramin in 50 ml of saline by intravenous infusion over 30 minutes

Drug: Suramin

Saline

PLACEBO COMPARATOR

50 ml of saline by intravenous infusion over 30 minutes

Drug: Saline

Interventions

Suramin DRUG

20 mg/kg IV in 50 ml saline over 30 minutes

Also known as: Germanin

Suramin

Saline DRUG

50 ml IV over 30 minutes

Also known as: Normal saline

Saline

Eligibility Criteria

• Age: 4 Years - 17 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of California, San Diego: lead

Study Sites (1)

University of California, San Diego School of Medicine

La Jolla, California, 92093, United States

Location

Related Publications (5)

• Naviaux JC, Wang L, Li K, Bright AT, Alaynick WA, Williams KR, Powell SB, Naviaux RK. Antipurinergic therapy corrects the autism-like features in the Fragile X (Fmr1 knockout) mouse model. Mol Autism. 2015 Jan 13;6:1. doi: 10.1186/2040-2392-6-1. eCollection 2015.

  PMID: 25705365 BACKGROUND

• Naviaux JC, Schuchbauer MA, Li K, Wang L, Risbrough VB, Powell SB, Naviaux RK. Reversal of autism-like behaviors and metabolism in adult mice with single-dose antipurinergic therapy. Transl Psychiatry. 2014 Jun 17;4(6):e400. doi: 10.1038/tp.2014.33.

  PMID: 24937094 BACKGROUND

• Naviaux RK, Zolkipli Z, Wang L, Nakayama T, Naviaux JC, Le TP, Schuchbauer MA, Rogac M, Tang Q, Dugan LL, Powell SB. Antipurinergic therapy corrects the autism-like features in the poly(IC) mouse model. PLoS One. 2013;8(3):e57380. doi: 10.1371/journal.pone.0057380. Epub 2013 Mar 13.

  PMID: 23516405 BACKGROUND

• Naviaux RK. Metabolic features of the cell danger response. Mitochondrion. 2014 May;16:7-17. doi: 10.1016/j.mito.2013.08.006. Epub 2013 Aug 24.

  PMID: 23981537 BACKGROUND

• Naviaux RK, Curtis B, Li K, Naviaux JC, Bright AT, Reiner GE, Westerfield M, Goh S, Alaynick WA, Wang L, Capparelli EV, Adams C, Sun J, Jain S, He F, Arellano DA, Mash LE, Chukoskie L, Lincoln A, Townsend J. Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial. Ann Clin Transl Neurol. 2017 May 26;4(7):491-505. doi: 10.1002/acn3.424. eCollection 2017 Jul.

  PMID: 28695149 BACKGROUND

Related Links

• Resource materials from the PI's lab website

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Suramin; Sodium Chloride; Saline Solution

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Naphthalenesulfonates; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Arylsulfonates; Arylsulfonic Acids; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Polycyclic Compounds; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds; Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Results Point of Contact

• Title: Dr. Robert K. Naviaux, MD, PhD
• Organization: University of California, San Diego School of Medicine

naviaux@ucsd.edu

619-543-2904

Study Officials

• Robert K Naviaux, MD, PhD

  University of California, San Diego

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 22, 2015
• First Posted: July 24, 2015
• Study Start: May 1, 2015
• Primary Completion: March 1, 2016
• Study Completion: April 1, 2016
• Last Updated: July 16, 2019
• Results First Posted: July 16, 2019

Record last verified: 2019-06

Data Sharing

• IPD Sharing: Will share

Locations

US (1)