NCT02508259: SAT1

The UCSD Suramin Autism Treatment-1 (SAT1) Trial

Completed Phase 1/Phase 2 Results posted Last updated 21 June 2019

What this trial tests

Phase 1/Phase 2 trial testing Suramin in Autism Spectrum Disorders in 10 participants. Completed in 1 April 2016.

Timeline

1 May 2015

Primary endpoint
1 March 2016

1 April 2016

Quick facts

Lead sponsor	University of California, San Diego
Phase	Phase 1/Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	10
Start date	1 May 2015
Primary completion	1 March 2016
Estimated completion	1 April 2016
Sites	1 location across United States

Drugs / interventions tested

• Suramin — full drug profile →
• Saline

Conditions studied

• Autism Spectrum Disorders — all drugs for Autism Spectrum Disorders →

Sponsor

University of California, San Diego

Who can join

Adults 4 to 17, male only, with Autism Spectrum Disorders. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• 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.
• 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

Sponsor's own description

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

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. 100 Years of Suramin.
   Wiedemar N, Hauser DA, Mäser P. · · 2020 · cited 135× · PMID 31844000 · DOI 10.1128/aac.01168-19
2. GABAergic inhibitory neurons as therapeutic targets for cognitive impairment in schizophrenia.
   Xu MY, Wong AHC. · · 2018 · cited 103× · PMID 29565038 · DOI 10.1038/aps.2017.172
3. Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial.
   Naviaux RK, Curtis B, Li K, Naviaux JC, et al · · 2017 · cited 89× · PMID 28695149 · DOI 10.1002/acn3.424
4. Purinergic signaling triggers endfoot high-amplitude Ca2+ signals and causes inversion of neurovascular coupling after subarachnoid hemorrhage.
   Pappas AC, Koide M, Wellman GC. · · 2016 · cited 10× · PMID 27207166 · DOI 10.1177/0271678x16650911
5. Altered Purinergic Signaling in Neurodevelopmental Disorders: Focus on P2 Receptors.
   Boccazzi M, Raffaele S, Zanettin T, Abbracchio MP, et al · · 2023 · cited 5× · PMID 37238724 · DOI 10.3390/biom13050856
6. Suramin Interactions Across Biological Systems: From Molecular Targets to Therapeutic Implications.
   Catalano A, Scaglione V, Sgobba MN, Ferrone L, et al · · 2026 · PMID 42072648 · DOI 10.3390/biom16040527
7. Autism spectrum disorder and the role of nuclear hormone receptors: insights and therapeutic implications.
   Chintalapally S, Rajanala K, Upadhyay A. · · 2025 · PMID 41245836 · DOI 10.3389/fnins.2025.1655348