Trial Outcomes & Findings for University of California, San Diego (UCSD) Suramin Autism Treatment-1 (SAT1) Trial (NCT NCT02508259)
NCT ID: NCT02508259
Last Updated: 2019-07-16
Results Overview
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.
COMPLETED
PHASE1/PHASE2
10 participants
6 weeks compared to baseline
2019-07-16
Participant Flow
Participant milestones
| Measure |
Suramin
20 mg/kg IV x1
|
Saline (Placebo)
IV Infusion x 1
|
|---|---|---|
|
Overall Study
STARTED
|
5
|
5
|
|
Overall Study
COMPLETED
|
5
|
5
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
University of California, San Diego (UCSD) Suramin Autism Treatment-1 (SAT1) Trial
Baseline characteristics by cohort
| Measure |
Suramin
n=5 Participants
20 mg/kg IV x 1
|
Saline (Placebo)
n=5 Participants
IV infusion x 1
|
Total
n=10 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
8.9 years
STANDARD_DEVIATION 3.3 • n=5 Participants
|
9.2 years
STANDARD_DEVIATION 3.8 • n=7 Participants
|
9.1 years
STANDARD_DEVIATION 3.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
5 participants
n=5 Participants
|
5 participants
n=7 Participants
|
10 participants
n=5 Participants
|
|
Autism Diagnostic Observation Schedule, 2nd edition (ADOS2)
|
8.6 units on a scale
STANDARD_DEVIATION 0.9 • n=5 Participants
|
9.4 units on a scale
STANDARD_DEVIATION 1.3 • n=7 Participants
|
9.0 units on a scale
STANDARD_DEVIATION 1.2 • n=5 Participants
|
PRIMARY outcome
Timeframe: 6 weeks compared to baselinePopulation: Patients receiving either suramin or saline.
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.
Outcome measures
| Measure |
Suramin
n=5 Participants
20 mg/kg IV x 1
|
Saline (Placebo)
n=5 Participants
IV Infusion
|
|---|---|---|
|
Autism Diagnostic Observation Schedule, 2nd Edition (ADOS2)
|
-1.6 units on a scale
Standard Deviation 0.55
|
-0.4 units on a scale
Standard Deviation 0.55
|
PRIMARY outcome
Timeframe: 6 weeks compared to baselinePopulation: Subjects receiving either suramin or saline.
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.
Outcome measures
| Measure |
Suramin
n=5 Participants
20 mg/kg IV x 1
|
Saline (Placebo)
n=5 Participants
IV Infusion
|
|---|---|---|
|
Expressive Language
|
-4.2 units on a scale
Standard Deviation 8.3
|
2.0 units on a scale
Standard Deviation 4.6
|
SECONDARY outcome
Timeframe: 6 weeks compared to baselinePopulation: Subjects receiving either suramin or saline.
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.
Outcome measures
| Measure |
Suramin
n=5 Participants
20 mg/kg IV x 1
|
Saline (Placebo)
n=5 Participants
IV Infusion
|
|---|---|---|
|
Aberrant Behavior Checklist (ABC)
|
-4.0 units on a scale
Standard Deviation 2.3
|
1.0 units on a scale
Standard Deviation 4.3
|
SECONDARY outcome
Timeframe: 6 weeksPopulation: Subjects receiving either suramin or saline.
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.
Outcome measures
| Measure |
Suramin
n=5 Participants
20 mg/kg IV x 1
|
Saline (Placebo)
n=5 Participants
IV Infusion
|
|---|---|---|
|
Autism Treatment Evaluation Checklist (ATEC)
|
-2.0 units on a scale
Standard Deviation 1.4
|
-0.2 units on a scale
Standard Deviation 2.9
|
SECONDARY outcome
Timeframe: Overall ASD symptoms at 6 weeksPopulation: Subjects receiving either suramin or saline.
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.
Outcome measures
| Measure |
Suramin
n=5 Participants
20 mg/kg IV x 1
|
Saline (Placebo)
n=5 Participants
IV Infusion
|
|---|---|---|
|
The Clinical Global Impression - Improvement Scale (CGI-I)
|
-1.8 units on a scale
Standard Deviation 1.04
|
0.0 units on a scale
Standard Deviation 0.34
|
SECONDARY outcome
Timeframe: 6 weeks compared to baselinePopulation: Subjects receiving suramin or saline.
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.
Outcome measures
| Measure |
Suramin
n=5 Participants
20 mg/kg IV x 1
|
Saline (Placebo)
n=5 Participants
IV Infusion
|
|---|---|---|
|
Repetitive Behavior Questionnaire
|
-3.2 units on a scale
Standard Deviation 5.8
|
-0.8 units on a scale
Standard Deviation 3.3
|
Adverse Events
Suramin
Saline (Placebo)
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Suramin
n=5 participants at risk
20 mg/kg IV x 1
|
Saline (Placebo)
n=5 participants at risk
IV Infusion x 1
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Rash
|
100.0%
5/5 • Number of events 5 • 6 weeks
|
0.00%
0/5 • 6 weeks
|
|
Infections and infestations
Upper respiratory tract infection (URI)
|
40.0%
2/5 • Number of events 2 • 6 weeks
|
40.0%
2/5 • Number of events 2 • 6 weeks
|
|
Nervous system disorders
Headache
|
20.0%
1/5 • Number of events 1 • 6 weeks
|
0.00%
0/5 • 6 weeks
|
|
Gastrointestinal disorders
Vomiting
|
20.0%
1/5 • Number of events 1 • 6 weeks
|
20.0%
1/5 • Number of events 1 • 6 weeks
|
|
Nervous system disorders
Hyperactivity
|
40.0%
2/5 • Number of events 2 • 6 weeks
|
20.0%
1/5 • Number of events 1 • 6 weeks
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
20.0%
1/5 • Number of events 1 • 6 weeks
|
20.0%
1/5 • Number of events 1 • 6 weeks
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/5 • 6 weeks
|
20.0%
1/5 • Number of events 1 • 6 weeks
|
Additional Information
Dr. Robert K. Naviaux, MD, PhD
University of California, San Diego School of Medicine
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place