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MEG Study of Acute STX209 Effects in ASD

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ClinicalTrials.gov Identifier: NCT02278328
Recruitment Status : Completed
First Posted : October 30, 2014
Results First Posted : October 21, 2019
Last Update Posted : October 21, 2019
Sponsor:
Collaborators:
Simons Foundation
Clinical Research Associates, LLC
Information provided by (Responsible Party):
Timothy Roberts, Children's Hospital of Philadelphia

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: None (Open Label);   Primary Purpose: Basic Science
Condition Autism Disorder
Interventions Drug: STX209 (15mg)
Drug: placebo
Drug: STX209 (30mg)
Enrollment 25
Recruitment Details  
Pre-assignment Details  
Arm/Group Title A. Placebo Then 15mg Then 30mg B. 15mg Then Placebo Then 30mg C. 15mg Then 30mg Then Placebo
Hide Arm/Group Description

Subjects will receive a single dose of placebo, then (a week later) 15mg STX209 then (a week later) 30mg STX209. Subjects will receive STX209 via oral disintegrating tablets, administered in individual 15mg tablets.

A randomized acute dose-response design will be employed with a total study duration of 3 weeks. At each visit, baseline MEG will be obtained followed by acute single-dose drug/placebo administration, followed 60 minutes later by repeat MEG. Over the course of the three weeks, each participant will receive a single dose of placebo and a single dose of STX209 from smallest to largest (15mg, and 30mg). On each occasion, separated by 1 week, participants will receive either placebo or 15mg STX209 or 30mg STX209. MRI and MRS will be performed immediately following MEG to provide an anatomic basis for source localization as well as to assess acute effects of STX209 administration on MRS estimates of GABA and glutamate.

Subjects will receive a single dose of 15mg STX209, then (a week later) placebo then (a week later) 30mg STX209. Subjects will receive STX209 via oral disintegrating tablets, administered in individual 15mg tablets.

A randomized acute dose-response design will be employed with a total study duration of 3 weeks. At each visit, baseline MEG will be obtained followed by acute single-dose drug/placebo administration, followed 60 minutes later by repeat MEG. Over the course of the three weeks, each participant will receive a single dose of placebo and a single dose of STX209 from smallest to largest (15mg, and 30mg). On each occasion, separated by 1 week, participants will receive either placebo or 15mg STX209 or 30mg STX209. MRI and MRS will be performed immediately following MEG to provide an anatomic basis for source localization as well as to assess acute effects of STX209 administration on MRS estimates of GABA and glutamate.

Subjects will receive a single dose of 15mg STX209, then (a week later) 30mg STX209 then (a week later) placebo . Subjects will receive STX209 via oral disintegrating tablets, administered in individual 15mg tablets.

A randomized acute dose-response design will be employed with a total study duration of 3 weeks. At each visit, baseline MEG will be obtained followed by acute single-dose drug/placebo administration, followed 60 minutes later by repeat MEG. Over the course of the three weeks, each participant will receive a single dose of placebo and a single dose of STX209 from smallest to largest (15mg, and 30mg). On each occasion, separated by 1 week, participants will receive either placebo or 15mg STX209 or 30mg STX209. MRI and MRS will be performed immediately following MEG to provide an anatomic basis for source localization as well as to assess acute effects of STX209 administration on MRS estimates of GABA and glutamate.

Period Title: Neuropsych Screening
Started 10 7 8
Completed 9 6 6
Not Completed 1 1 2
Reason Not Completed
found ineligible on neuropsych             1             0             0
Withdrawal by Subject             0             1             0
incomplete consent             0             0             2
Period Title: First Intervention (1 Day)
Started 9 6 6
Completed 9 6 6
Not Completed 0 0 0
Period Title: Washout (1 Week)
Started 9 6 6
Completed 9 6 6
Not Completed 0 0 0
Period Title: Second Intervention (1 Day)
Started 9 6 6
Completed 9 6 6
Not Completed 0 0 0
Period Title: Second Washout (1 Week)
Started 9 6 6
Completed 9 6 6
Not Completed 0 0 0
Period Title: Third Intervention (1 Day)
Started 9 6 6
Completed 9 6 6
Not Completed 0 0 0
Period Title: Third Washout (1 Week)
Started 9 6 6
Completed 9 6 6
Not Completed 0 0 0
Arm/Group Title All Participants
Hide Arm/Group Description

Subjects receive a single dose of STX209 or placebo starting at 15 mg, increasing to 30 mg on each of three separate occasions (different dose on each occasion, according to their randomization scheme Arm. Outcome measures are reported by dose administered (or placebo) and not by randomization schedule, since the same outcome measures are acquired after each dose administration. Subjects will receive STX209 via oral disintegrating tablets, administered in individual 15mg tablets.

A randomized acute dose-response design is employed with a total study duration of 3 weeks. At each visit, baseline MEG will be obtained followed by acute single-dose drug/placebo administration, followed 60 minutes later by repeat MEG. MRI and MRS will be performed immediately following MEG to provide an anatomic basis for source localization as well as to assess acute effects of STX209 administration on MRS estimates of GABA and glutamate.

Overall Number of Baseline Participants 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 21 participants
15.8  (.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
Female
0
   0.0%
Male
21
 100.0%
Race and Ethnicity Not Collected   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants
[1]
Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 21 participants
21
M50 Latency (Left Hemisphere)   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Ms
Pre-placebo Number Analyzed 19 participants
89.9  (19.2)
Pre-15mg dose Number Analyzed 20 participants
91.9  (15.0)
Pre-30mg dose Number Analyzed 20 participants
90.8  (13.9)
[1]
Measure Description: Latency of M50 evoked response component arising from left cerebral hemisphere
[2]
Measure Analysis Population Description: in 2 cases pre-placebo and in 1 case each pre-15mg and pre-30mg, evoked response data was unevaluable due to artifact
M50 Latency (Right Hemisphere)   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Ms
Pre-placebo Number Analyzed 19 participants
95.6  (24.0)
Pre-15mg dose Number Analyzed 20 participants
96.9  (27.6)
Pre-30mg dose Number Analyzed 19 participants
93.6  (21.6)
[1]
Measure Description: Latency of M50 evoked response component arising from right cerebral hemisphere
[2]
Measure Analysis Population Description: in 2 cases pre-placebo,1 case pre-15mg and and 2 cases pre-30mg, evoked response data was unevaluable due to artifact
Steady State Inter-Trial Coherence (Left Hemisphere)   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Unitless
Pre-placebo Number Analyzed 21 participants
.214  (.122)
Pre-15mg dose Number Analyzed 20 participants
.237  (.106)
Pre-30mg dose Number Analyzed 17 participants
.239  (.129)
[1]
Measure Description: inter-trial coherence (ITC) of left hemisphere responses to steady state auditory stimuli
[2]
Measure Analysis Population Description: in 1 participant prior to 15mg and in 4 participants prior to 30mg dose, data was unevaluable.
Steady State Inter-Trial Coherence (Right Hemisphere)   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Unitless
Pre-placebo Number Analyzed 21 participants
.273  (.168)
Pre-15mg dose Number Analyzed 20 participants
.316  (.181)
Pre-30mg dose Number Analyzed 17 participants
.321  (.173)
[1]
Measure Description: Inter-trial coherence (ITC) to auditory steady state stimuli arising from right cerebral hemisphere
[2]
Measure Analysis Population Description: in 1 participant prior to 15mg and in 4 participants prior to 30mg dose, data was unevaluable.
GABA (Left Hemisphere)   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Ratio
Pre-placebo Number Analyzed 14 participants
.149  (.075)
Pre-15mg dose Number Analyzed 16 participants
.154  (.078)
Pre-30mg dose Number Analyzed 16 participants
.148  (.061)
[1]
Measure Description: GABA/Cr ratio for voxel in left superior temporal gyrus
[2]
Measure Analysis Population Description: In 7 cases pre-placebo and in 5 cases each pre-15mg or 30mg, spectroscopy data was unevaluable
1.Primary Outcome
Title M50 Latency (Left Hemisphere)
Hide Description The latency of the M50 auditory evoked response component arising from the left cerebral hemisphere
Time Frame 1 hour per intervention followed by a 1 week washout for a total of three weeks
Hide Outcome Measure Data
Hide Analysis Population Description
For placebo and 15mg dose, 1 case each was unavailable due to artifact. At 30mg dose, 2 cases were unevaluable due to artifact
Arm/Group Title All Participants
Hide Arm/Group Description:
Results post placebo and each dose (15 and 30mg)
Overall Number of Participants Analyzed 21
Mean (Standard Deviation)
Unit of Measure: ms
Post placebo Number Analyzed 20 participants
90.9  (13.8)
Post-15mg dose Number Analyzed 20 participants
87.4  (16.9)
Post-30mg dose Number Analyzed 19 participants
89.3  (13.5)
2.Primary Outcome
Title M50 Latency (Right Hemisphere)
Hide Description The latency of the M50 auditory evoked response component arising from the right cerebral hemisphere
Time Frame 1 hour per intervention followed by a 1 week washout for a total of three weeks
Hide Outcome Measure Data
Hide Analysis Population Description
For placebo and 15mg dose, 1 case each was unavailable due to artifact. At 30mg dose, 4 cases were unevaluable due to artifact
Arm/Group Title All Participants
Hide Arm/Group Description:
Results post placebo and each dose (15 and 30mg)
Overall Number of Participants Analyzed 21
Mean (Standard Deviation)
Unit of Measure: ms
Post-placebo Number Analyzed 20 participants
95.1  (21.7)
Post-15mg dose Number Analyzed 20 participants
87.8  (8.6)
Post-30mg dose Number Analyzed 17 participants
93.8  (23.6)
3.Primary Outcome
Title Steady State Inter Trial Coherence (Left Hemisphere)
Hide Description The inter trial coherence (ITC) of auditory steady state response arising from the left cerebral hemisphere
Time Frame 1 hour per intervention followed by a 1 week washout for a total of three weeks
Hide Outcome Measure Data
Hide Analysis Population Description
For placebo, 3 cases and for 15mg and 30mg doses, 1 case each was unevaluable due to artifact.
Arm/Group Title All Participants
Hide Arm/Group Description:
Results post placebo and each dose (15 and 30mg)
Overall Number of Participants Analyzed 21
Mean (Standard Deviation)
Unit of Measure: unitless
Post-placebo Number Analyzed 18 participants
.253  (.134)
Post-15mg dose Number Analyzed 20 participants
.239  (.111)
Post-30mg dose Number Analyzed 20 participants
.244  (.116)
4.Primary Outcome
Title Steady State Inter Trial Coherence (Right Hemisphere)
Hide Description The inter trial coherence (ITC) of auditory steady state response arising from the right cerebral hemisphere
Time Frame 1 hour per intervention followed by a 1 week washout for a total of three weeks
Hide Outcome Measure Data
Hide Analysis Population Description
For placebo, 3 cases and for 15mg dose and 30mg doses, 1 case each was unevaluable due to artifact.
Arm/Group Title All Participants
Hide Arm/Group Description:
Results post placebo and each dose (15 and 30mg)
Overall Number of Participants Analyzed 21
Mean (Standard Deviation)
Unit of Measure: unitless
Post-placebo Number Analyzed 18 participants
.324  (.178)
Post-15mg dose Number Analyzed 20 participants
.325  (.166)
Post-30mg dose Number Analyzed 20 participants
.306  (.153)
5.Primary Outcome
Title GABA (Left Hemisphere)
Hide Description GABA/Cr ratio arising from a voxel in the left superior temporal gyrus
Time Frame 1 hour per intervention followed by a 1 week washout for a total of three weeks
Hide Outcome Measure Data
Hide Analysis Population Description
For placebo, 5 cases were unevaluable. For 15mg, 7 cases were unevaluable. For 30mg, 4 cases were unevaluable
Arm/Group Title All Participants
Hide Arm/Group Description:
Results post placebo and each dose (15 and 30mg)
Overall Number of Participants Analyzed 21
Mean (Standard Deviation)
Unit of Measure: ratio
Post-placebo Number Analyzed 16 participants
.155  (.045)
Post-15mg dose Number Analyzed 14 participants
.154  (.056)
Post-30mg dose Number Analyzed 17 participants
.164  (.062)
Time Frame 3 weeks
Adverse Event Reporting Description Although 25 participants are in the participant flow, two participants did not proceed past teh neuropsychological evaluation and onto any of the interventions and therefore did not receive any drug dose or placebo (having withdrawn or been excluded after screening). Therefore, we consider only 23 participants to have been placed at risk by the study.
 
Arm/Group Title Placebo STX209 (15mg) STX209 (30mg)
Hide Arm/Group Description Subjects will receive a single dose of placebo via oral disintegrating tablets, administered as two individual tablets. Subjects will receive a single dose of 15mg STX209 via oral disintegrating tablets, administered as two individual tablets (one being 15mg drug, one being placebo) Subjects will receive a single dose of 15mg STX209 via oral disintegrating tablets, administered as two individual tablets (each being 15mg drug)
All-Cause Mortality
Placebo STX209 (15mg) STX209 (30mg)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/23 (0.00%)      0/23 (0.00%)      0/23 (0.00%)    
Hide Serious Adverse Events
Placebo STX209 (15mg) STX209 (30mg)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/23 (0.00%)      0/23 (0.00%)      0/23 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo STX209 (15mg) STX209 (30mg)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/23 (30.43%)      11/23 (47.83%)      4/23 (17.39%)    
Gastrointestinal disorders       
vomiting   1/23 (4.35%)  1 0/23 (0.00%)  0 2/23 (8.70%)  2
General disorders       
fatigue   3/23 (13.04%)  3 4/23 (17.39%)  4 1/23 (4.35%)  1
increased appetite  [1]  1/23 (4.35%)  1 2/23 (8.70%)  2 0/23 (0.00%)  0
overly energetic   2/23 (8.70%)  2 2/23 (8.70%)  2 1/23 (4.35%)  1
Respiratory, thoracic and mediastinal disorders       
cold symptoms / upper respiratory  [2]  0/23 (0.00%)  0 4/23 (17.39%)  4 1/23 (4.35%)  1
Indicates events were collected by systematic assessment
[1]
hungry / thirsty
[2]
stuffy nose, sore throat, coughing, sneezing
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: Timothy Roberts, Professor
Organization: Children's Hospital of Philadelphia
Phone: 267 426 0307
EMail: robertstim@email.chop.edu
Layout table for additonal information
Responsible Party: Timothy Roberts, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT02278328    
Other Study ID Numbers: 14-010857
First Submitted: October 17, 2014
First Posted: October 30, 2014
Results First Submitted: June 19, 2019
Results First Posted: October 21, 2019
Last Update Posted: October 21, 2019