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Trial record 2 of 21 for:    suramin

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Autism Spectrum Disorders
Interventions Drug: Suramin
Drug: Saline
Enrollment 10
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Suramin Saline (Placebo)
Hide Arm/Group Description 20 mg/kg IV x1 IV Infusion x 1
Period Title: Overall Study
Started 5 5
Completed 5 5
Not Completed 0 0
Arm/Group Title Suramin Saline (Placebo) Total
Hide Arm/Group Description 20 mg/kg IV x 1 IV infusion x 1 Total of all reporting groups
Overall Number of Baseline Participants 5 5 10
Hide Baseline Analysis Population Description
Male subjects with autism spectrum disorder (ASD), ages 4-17 years
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 5 participants 5 participants 10 participants
8.9  (3.3) 9.2  (3.8) 9.1  (3.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 5 participants 10 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
5
 100.0%
5
 100.0%
10
 100.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 5 participants 10 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
  20.0%
1
  20.0%
2
  20.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
4
  80.0%
4
  80.0%
8
  80.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 5 participants 5 participants 10 participants
5 5 10
Autism Diagnostic Observation Schedule, 2nd edition (ADOS2)   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 5 participants 5 participants 10 participants
8.6  (0.9) 9.4  (1.3) 9.0  (1.2)
[1]
Measure Description: 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.
1.Primary Outcome
Title Autism Diagnostic Observation Schedule, 2nd Edition (ADOS2)
Hide Description 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.
Time Frame 6 weeks compared to baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Patients receiving either suramin or saline.
Arm/Group Title Suramin Saline (Placebo)
Hide Arm/Group Description:
20 mg/kg IV x 1
IV Infusion
Overall Number of Participants Analyzed 5 5
Mean (Standard Deviation)
Unit of Measure: units on a scale
-1.6  (0.55) -0.4  (0.55)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Suramin
Comments In this analysis, each child was used as their own control to examine before and after suramin treatment effects in a paired t-test design. This study was a small pilot study of safety and activity of suramin. The small subject numbers of just 5 per group make it impossible to draw any strong conclusions about efficacy. Future studies are required.
Type of Statistical Test Equivalence
Comments The null hypothesis was that before and after treatment ADOS scores were equivalent.
Statistical Test of Hypothesis P-Value 0.0028
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.6
Confidence Interval (2-Sided) 95%
-2.3 to -0.9
Parameter Dispersion
Type: Standard Deviation
Value: 0.55
Estimation Comments [Not Specified]
2.Primary Outcome
Title Expressive Language
Hide Description 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.
Time Frame 6 weeks compared to baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects receiving either suramin or saline.
Arm/Group Title Suramin Saline (Placebo)
Hide Arm/Group Description:
20 mg/kg IV x 1
IV Infusion
Overall Number of Participants Analyzed 5 5
Mean (Standard Deviation)
Unit of Measure: units on a scale
-4.2  (8.3) 2.0  (4.6)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Suramin
Comments In this analysis, each child was used as their own control to examine before and after suramin treatment effects in a paired t-test design. This study was a small pilot study of safety and activity of suramin. The small subject numbers of just 5 per group make it impossible to draw any strong conclusions about efficacy. Future studies are required.
Type of Statistical Test Equivalence
Comments The child-specific difference in EOWPVT scores were compared for equivalence before and 6-weeks after suramin treatment
Statistical Test of Hypothesis P-Value 0.32
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -4.2
Confidence Interval (2-Sided) 95%
-14.5 to 6.1
Parameter Dispersion
Type: Standard Deviation
Value: 8.3
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Aberrant Behavior Checklist (ABC)
Hide Description 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.
Time Frame 6 weeks compared to baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects receiving either suramin or saline.
Arm/Group Title Suramin Saline (Placebo)
Hide Arm/Group Description:
20 mg/kg IV x 1
IV Infusion
Overall Number of Participants Analyzed 5 5
Mean (Standard Deviation)
Unit of Measure: units on a scale
-4.0  (2.3) 1.0  (4.3)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Suramin
Comments In this analysis, each child was used as their own control to examine before and after suramin treatment effects in a paired t-test design. This study was a small pilot study of safety and activity of suramin. The small subject numbers of just 5 per group make it impossible to draw any strong conclusions about efficacy. Future studies are required.
Type of Statistical Test Equivalence
Comments In this analysis, the null hypothesis was tested that the child-specific ABC subscores for stereotypy were unchanged before and after suramin treatment.
Statistical Test of Hypothesis P-Value 0.019
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -4.0
Confidence Interval (2-Sided) 95%
-6.9 to -1.1
Parameter Dispersion
Type: Standard Deviation
Value: 2.3
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Autism Treatment Evaluation Checklist (ATEC)
Hide Description 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.
Time Frame 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects receiving either suramin or saline.
Arm/Group Title Suramin Saline (Placebo)
Hide Arm/Group Description:
20 mg/kg IV x 1
IV Infusion
Overall Number of Participants Analyzed 5 5
Mean (Standard Deviation)
Unit of Measure: units on a scale
-2.0  (1.4) -0.2  (2.9)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Suramin
Comments In this analysis, each child was used as their own control to examine before and after suramin treatment effects in a paired t-test design. This study was a small pilot study of safety and activity of suramin. The small subject numbers of just 5 per group make it impossible to draw any strong conclusions about efficacy. Future studies are required.
Type of Statistical Test Equivalence
Comments In this analysis, we tested the equivalence of the child-specific ATEC subscore for language before and 6-weeks after treatment with suramin.
Statistical Test of Hypothesis P-Value 0.034
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -2.0
Confidence Interval (2-Sided) 95%
-2.7 to -0.49
Parameter Dispersion
Type: Standard Deviation
Value: 1.4
Estimation Comments [Not Specified]
5.Secondary Outcome
Title The Clinical Global Impression - Improvement Scale (CGI-I)
Hide Description 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.
Time Frame Overall ASD symptoms at 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects receiving either suramin or saline.
Arm/Group Title Suramin Saline (Placebo)
Hide Arm/Group Description:
20 mg/kg IV x 1
IV Infusion
Overall Number of Participants Analyzed 5 5
Mean (Standard Deviation)
Unit of Measure: units on a scale
-1.8  (1.04) 0.0  (0.34)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Suramin, Saline (Placebo)
Comments In this analysis, each child was used as their own control to examine before and after suramin treatment effects in a paired t-test design. This study was a small pilot study of safety and activity of suramin. The small subject numbers of just 5 per group make it impossible to draw any strong conclusions about efficacy. Future studies are required.
Type of Statistical Test Equivalence
Comments In this analysis, overall ASD symptom scores, measured by CGI were compared between suramin and placebo groups.
Statistical Test of Hypothesis P-Value 0.05
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -1.8
Confidence Interval (2-Sided) 95%
-3.4 to -0.15
Parameter Dispersion
Type: Standard Deviation
Value: 1.04
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Repetitive Behavior Questionnaire
Hide Description 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.
Time Frame 6 weeks compared to baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects receiving suramin or saline.
Arm/Group Title Suramin Saline (Placebo)
Hide Arm/Group Description:
20 mg/kg IV x 1
IV Infusion
Overall Number of Participants Analyzed 5 5
Mean (Standard Deviation)
Unit of Measure: units on a scale
-3.2  (5.8) -0.8  (3.3)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Suramin
Comments In this analysis, each child was used as their own control to examine before and after suramin treatment effects in a paired t-test design. This study was a small pilot study of safety and activity of suramin. The small subject numbers of just 5 per group make it impossible to draw any strong conclusions about efficacy. Future studies are required.
Type of Statistical Test Equivalence
Comments In this analysis, the child-specific RBQ score was tested for equivalence before and 6-weeks after suramin treatment.
Statistical Test of Hypothesis P-Value 0.28
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -3.2
Confidence Interval (2-Sided) 95%
-10.4 to 4.0
Parameter Dispersion
Type: Standard Deviation
Value: 5.8
Estimation Comments [Not Specified]
Time Frame 6 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Suramin Saline (Placebo)
Hide Arm/Group Description 20 mg/kg IV x 1 IV Infusion x 1
All-Cause Mortality
Suramin Saline (Placebo)
Affected / at Risk (%) Affected / at Risk (%)
Total   0/5 (0.00%)      0/5 (0.00%)    
Hide Serious Adverse Events
Suramin Saline (Placebo)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/5 (0.00%)      0/5 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Suramin Saline (Placebo)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/5 (100.00%)      4/5 (80.00%)    
Blood and lymphatic system disorders     
Leukocytosis * 1  0/5 (0.00%)  0 1/5 (20.00%)  1
Gastrointestinal disorders     
Vomiting * 1 [1]  1/5 (20.00%)  1 1/5 (20.00%)  1
Infections and infestations     
Upper respiratory tract infection (URI) * 1  2/5 (40.00%)  2 2/5 (40.00%)  2
Metabolism and nutrition disorders     
Hypoglycemia * 1 [2]  1/5 (20.00%)  1 1/5 (20.00%)  1
Nervous system disorders     
Headache * 1  1/5 (20.00%)  1 0/5 (0.00%)  0
Hyperactivity * 1  2/5 (40.00%)  2 1/5 (20.00%)  1
Skin and subcutaneous tissue disorders     
Rash * 1  5/5 (100.00%)  5 0/5 (0.00%)  0
1
Term from vocabulary, CTCAE (4.0)
*
Indicates events were collected by non-systematic assessment
[1]
In 7-year old after pizza and slushee consumption after playing youth league basketball, and in a 6-year old after a car ride.
[2]
Both cases occurred 6-weeks after the infusion, after several days of a URI and fasting before lunch for the blood draw. Hypoglycemia was asymptomatic and corrected after a normal lunch.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Robert K. Naviaux, MD, PhD
Organization: University of California, San Diego School of Medicine
Phone: 619-543-2904
EMail: naviaux@ucsd.edu
Layout table for additonal information
Responsible Party: Robert K. Naviaux, University of California, San Diego
ClinicalTrials.gov Identifier: NCT02508259    
Other Study ID Numbers: 15-0134
First Submitted: July 22, 2015
First Posted: July 24, 2015
Results First Submitted: May 1, 2017
Results First Posted: July 16, 2019
Last Update Posted: July 16, 2019