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Trial Outcomes & Findings for MEG Study of Acute STX209 Effects in ASD (NCT NCT02278328)

NCT ID: NCT02278328

Last Updated: 2019-10-21

Results Overview

The latency of the M50 auditory evoked response component arising from the left cerebral hemisphere

Recruitment status

COMPLETED

Study phase

EARLY_PHASE1

Target enrollment

25 participants

Primary outcome timeframe

1 hour per intervention followed by a 1 week washout for a total of three weeks

Results posted on

2019-10-21

Participant Flow

Participant milestones

Participant milestones
Measure
A. Placebo Then 15mg Then 30mg
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.
B. 15mg Then Placebo Then 30mg
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.
C. 15mg Then 30mg Then Placebo
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.
Neuropsych Screening
STARTED
10
7
8
Neuropsych Screening
COMPLETED
9
6
6
Neuropsych Screening
NOT COMPLETED
1
1
2
First Intervention (1 Day)
STARTED
9
6
6
First Intervention (1 Day)
COMPLETED
9
6
6
First Intervention (1 Day)
NOT COMPLETED
0
0
0
Washout (1 Week)
STARTED
9
6
6
Washout (1 Week)
COMPLETED
9
6
6
Washout (1 Week)
NOT COMPLETED
0
0
0
Second Intervention (1 Day)
STARTED
9
6
6
Second Intervention (1 Day)
COMPLETED
9
6
6
Second Intervention (1 Day)
NOT COMPLETED
0
0
0
Second Washout (1 Week)
STARTED
9
6
6
Second Washout (1 Week)
COMPLETED
9
6
6
Second Washout (1 Week)
NOT COMPLETED
0
0
0
Third Intervention (1 Day)
STARTED
9
6
6
Third Intervention (1 Day)
COMPLETED
9
6
6
Third Intervention (1 Day)
NOT COMPLETED
0
0
0
Third Washout (1 Week)
STARTED
9
6
6
Third Washout (1 Week)
COMPLETED
9
6
6
Third Washout (1 Week)
NOT COMPLETED
0
0
0

Reasons for withdrawal

Reasons for withdrawal
Measure
A. Placebo Then 15mg Then 30mg
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.
B. 15mg Then Placebo Then 30mg
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.
C. 15mg Then 30mg Then Placebo
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.
Neuropsych Screening
found ineligible on neuropsych
1
0
0
Neuropsych Screening
Withdrawal by Subject
0
1
0
Neuropsych Screening
incomplete consent
0
0
2

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
All Participants
n=21 Participants
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.
Age, Continuous
15.8 years
STANDARD_DEVIATION .8 • n=21 Participants
Sex: Female, Male
Female
0 Participants
n=21 Participants
Sex: Female, Male
Male
21 Participants
n=21 Participants
Region of Enrollment
United States
21 participants
n=21 Participants
M50 Latency (Left Hemisphere)
Pre-placebo
89.9 ms
STANDARD_DEVIATION 19.2 • n=19 Participants • 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 (Left Hemisphere)
Pre-15mg dose
91.9 ms
STANDARD_DEVIATION 15.0 • n=20 Participants • 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 (Left Hemisphere)
Pre-30mg dose
90.8 ms
STANDARD_DEVIATION 13.9 • n=20 Participants • 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)
Pre-placebo
95.6 ms
STANDARD_DEVIATION 24.0 • n=19 Participants • in 2 cases pre-placebo,1 case pre-15mg and and 2 cases pre-30mg, evoked response data was unevaluable due to artifact
M50 Latency (Right Hemisphere)
Pre-15mg dose
96.9 ms
STANDARD_DEVIATION 27.6 • n=20 Participants • in 2 cases pre-placebo,1 case pre-15mg and and 2 cases pre-30mg, evoked response data was unevaluable due to artifact
M50 Latency (Right Hemisphere)
Pre-30mg dose
93.6 ms
STANDARD_DEVIATION 21.6 • n=19 Participants • 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)
Pre-placebo
.214 unitless
STANDARD_DEVIATION .122 • n=21 Participants • in 1 participant prior to 15mg and in 4 participants prior to 30mg dose, data was unevaluable.
Steady State Inter-Trial Coherence (Left Hemisphere)
Pre-15mg dose
.237 unitless
STANDARD_DEVIATION .106 • n=20 Participants • in 1 participant prior to 15mg and in 4 participants prior to 30mg dose, data was unevaluable.
Steady State Inter-Trial Coherence (Left Hemisphere)
Pre-30mg dose
.239 unitless
STANDARD_DEVIATION .129 • n=17 Participants • in 1 participant prior to 15mg and in 4 participants prior to 30mg dose, data was unevaluable.
Steady State Inter-Trial Coherence (Right Hemisphere)
Pre-placebo
.273 unitless
STANDARD_DEVIATION .168 • n=21 Participants • in 1 participant prior to 15mg and in 4 participants prior to 30mg dose, data was unevaluable.
Steady State Inter-Trial Coherence (Right Hemisphere)
Pre-15mg dose
.316 unitless
STANDARD_DEVIATION .181 • n=20 Participants • in 1 participant prior to 15mg and in 4 participants prior to 30mg dose, data was unevaluable.
Steady State Inter-Trial Coherence (Right Hemisphere)
Pre-30mg dose
.321 unitless
STANDARD_DEVIATION .173 • n=17 Participants • in 1 participant prior to 15mg and in 4 participants prior to 30mg dose, data was unevaluable.
GABA (Left Hemisphere)
Pre-placebo
.149 ratio
STANDARD_DEVIATION .075 • n=14 Participants • In 7 cases pre-placebo and in 5 cases each pre-15mg or 30mg, spectroscopy data was unevaluable
GABA (Left Hemisphere)
Pre-15mg dose
.154 ratio
STANDARD_DEVIATION .078 • n=16 Participants • In 7 cases pre-placebo and in 5 cases each pre-15mg or 30mg, spectroscopy data was unevaluable
GABA (Left Hemisphere)
Pre-30mg dose
.148 ratio
STANDARD_DEVIATION .061 • n=16 Participants • In 7 cases pre-placebo and in 5 cases each pre-15mg or 30mg, spectroscopy data was unevaluable

PRIMARY outcome

Timeframe: 1 hour per intervention followed by a 1 week washout for a total of three weeks

Population: For placebo and 15mg dose, 1 case each was unavailable due to artifact. At 30mg dose, 2 cases were unevaluable due to artifact

The latency of the M50 auditory evoked response component arising from the left cerebral hemisphere

Outcome measures

Outcome measures
Measure
All Participants
n=21 Participants
Results post placebo and each dose (15 and 30mg)
M50 Latency (Left Hemisphere)
Post placebo
90.9 ms
Standard Deviation 13.8
M50 Latency (Left Hemisphere)
Post-15mg dose
87.4 ms
Standard Deviation 16.9
M50 Latency (Left Hemisphere)
Post-30mg dose
89.3 ms
Standard Deviation 13.5

PRIMARY outcome

Timeframe: 1 hour per intervention followed by a 1 week washout for a total of three weeks

Population: For placebo and 15mg dose, 1 case each was unavailable due to artifact. At 30mg dose, 4 cases were unevaluable due to artifact

The latency of the M50 auditory evoked response component arising from the right cerebral hemisphere

Outcome measures

Outcome measures
Measure
All Participants
n=21 Participants
Results post placebo and each dose (15 and 30mg)
M50 Latency (Right Hemisphere)
Post-placebo
95.1 ms
Standard Deviation 21.7
M50 Latency (Right Hemisphere)
Post-15mg dose
87.8 ms
Standard Deviation 8.6
M50 Latency (Right Hemisphere)
Post-30mg dose
93.8 ms
Standard Deviation 23.6

PRIMARY outcome

Timeframe: 1 hour per intervention followed by a 1 week washout for a total of three weeks

Population: For placebo, 3 cases and for 15mg and 30mg doses, 1 case each was unevaluable due to artifact.

The inter trial coherence (ITC) of auditory steady state response arising from the left cerebral hemisphere

Outcome measures

Outcome measures
Measure
All Participants
n=21 Participants
Results post placebo and each dose (15 and 30mg)
Steady State Inter Trial Coherence (Left Hemisphere)
Post-placebo
.253 unitless
Standard Deviation .134
Steady State Inter Trial Coherence (Left Hemisphere)
Post-15mg dose
.239 unitless
Standard Deviation .111
Steady State Inter Trial Coherence (Left Hemisphere)
Post-30mg dose
.244 unitless
Standard Deviation .116

PRIMARY outcome

Timeframe: 1 hour per intervention followed by a 1 week washout for a total of three weeks

Population: For placebo, 3 cases and for 15mg dose and 30mg doses, 1 case each was unevaluable due to artifact.

The inter trial coherence (ITC) of auditory steady state response arising from the right cerebral hemisphere

Outcome measures

Outcome measures
Measure
All Participants
n=21 Participants
Results post placebo and each dose (15 and 30mg)
Steady State Inter Trial Coherence (Right Hemisphere)
Post-placebo
.324 unitless
Standard Deviation .178
Steady State Inter Trial Coherence (Right Hemisphere)
Post-15mg dose
.325 unitless
Standard Deviation .166
Steady State Inter Trial Coherence (Right Hemisphere)
Post-30mg dose
.306 unitless
Standard Deviation .153

PRIMARY outcome

Timeframe: 1 hour per intervention followed by a 1 week washout for a total of three weeks

Population: For placebo, 5 cases were unevaluable. For 15mg, 7 cases were unevaluable. For 30mg, 4 cases were unevaluable

GABA/Cr ratio arising from a voxel in the left superior temporal gyrus

Outcome measures

Outcome measures
Measure
All Participants
n=21 Participants
Results post placebo and each dose (15 and 30mg)
GABA (Left Hemisphere)
Post-placebo
.155 ratio
Standard Deviation .045
GABA (Left Hemisphere)
Post-15mg dose
.154 ratio
Standard Deviation .056
GABA (Left Hemisphere)
Post-30mg dose
.164 ratio
Standard Deviation .062

Adverse Events

Placebo

Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths

STX209 (15mg)

Serious events: 0 serious events
Other events: 11 other events
Deaths: 0 deaths

STX209 (30mg)

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Placebo
n=23 participants at risk
Subjects will receive a single dose of placebo via oral disintegrating tablets, administered as two individual tablets.
STX209 (15mg)
n=23 participants at risk
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)
STX209 (30mg)
n=23 participants at risk
Subjects will receive a single dose of 15mg STX209 via oral disintegrating tablets, administered as two individual tablets (each being 15mg drug)
Respiratory, thoracic and mediastinal disorders
cold symptoms / upper respiratory
0.00%
0/23 • 3 weeks
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.
17.4%
4/23 • Number of events 4 • 3 weeks
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.
4.3%
1/23 • Number of events 1 • 3 weeks
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.
General disorders
fatigue
13.0%
3/23 • Number of events 3 • 3 weeks
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.
17.4%
4/23 • Number of events 4 • 3 weeks
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.
4.3%
1/23 • Number of events 1 • 3 weeks
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.
General disorders
increased appetite
4.3%
1/23 • Number of events 1 • 3 weeks
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.
8.7%
2/23 • Number of events 2 • 3 weeks
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.
0.00%
0/23 • 3 weeks
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.
General disorders
overly energetic
8.7%
2/23 • Number of events 2 • 3 weeks
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.
8.7%
2/23 • Number of events 2 • 3 weeks
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.
4.3%
1/23 • Number of events 1 • 3 weeks
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.
Gastrointestinal disorders
vomiting
4.3%
1/23 • Number of events 1 • 3 weeks
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.
0.00%
0/23 • 3 weeks
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.
8.7%
2/23 • Number of events 2 • 3 weeks
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.

Additional Information

Timothy Roberts, Professor

Children's Hospital of Philadelphia

Phone: 267 426 0307

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place