Trial Outcomes & Findings for Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome (NCT NCT00788073)
NCT ID: NCT00788073
Last Updated: 2013-05-06
Results Overview
The Aberrant Behavior Checklist-Community Edition (ABC-C) is a 58-item questionnaire composed of five different independent subscales. The questionnaire is completed by the parent/caregiver and lists aberrant behaviors and asks about the severity of the problem. ABC-Irritability is one of the subscales and comprises of 15 items. Minimum score is 0, maximum is 45. A decreased score indicates few aberrant behaviors and clinical improvement. The entire ABC-C assessment is administered at baseline and then at the end of each Intervention Period (4 weeks after Baseline).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
63 participants
Primary outcome timeframe
After 4 weeks of treatment
Results posted on
2013-05-06
Participant Flow
Participant milestones
| Measure |
STX209:Placebo
First Intervention=STX209 (STX209 variable dose from 1mg bid to 10mg tid, capsule, oral) Second Intervention=Placebo (Placebo variable dose (same flexible dose titration protocol) bid to tid, oral)
Study Design:
Placebo-controlled, Crossover study. First Intervention(28 Days)-\> Withdrawal(14 Days) -\> Washout(7 Days)-\> Withdrawal (14 Days) Participants received all interventions.
|
Placebo:STX209
First Intervention=Placebo (Placebo variable dose (same flexible dose titration protocol) bid to tid, oral) Second Intervention=STX209 (STX209 variable dose from 1mg bid to 10mg tid, capsule, oral)
Study Design:
Placebo-controlled, Crossover study. First Intervention(28 Days)-\> Withdrawal(14 Days) -\> Washout(7 Days)-\> Withdrawal (14 Days) Participants received all interventions.
|
|---|---|---|
|
STX209 Then Placebo
STARTED
|
30
|
33
|
|
STX209 Then Placebo
COMPLETED
|
29
|
30
|
|
STX209 Then Placebo
NOT COMPLETED
|
1
|
3
|
|
Placebo Then STX209
STARTED
|
29
|
30
|
|
Placebo Then STX209
COMPLETED
|
27
|
29
|
|
Placebo Then STX209
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
STX209:Placebo
First Intervention=STX209 (STX209 variable dose from 1mg bid to 10mg tid, capsule, oral) Second Intervention=Placebo (Placebo variable dose (same flexible dose titration protocol) bid to tid, oral)
Study Design:
Placebo-controlled, Crossover study. First Intervention(28 Days)-\> Withdrawal(14 Days) -\> Washout(7 Days)-\> Withdrawal (14 Days) Participants received all interventions.
|
Placebo:STX209
First Intervention=Placebo (Placebo variable dose (same flexible dose titration protocol) bid to tid, oral) Second Intervention=STX209 (STX209 variable dose from 1mg bid to 10mg tid, capsule, oral)
Study Design:
Placebo-controlled, Crossover study. First Intervention(28 Days)-\> Withdrawal(14 Days) -\> Washout(7 Days)-\> Withdrawal (14 Days) Participants received all interventions.
|
|---|---|---|
|
STX209 Then Placebo
Adverse Event
|
1
|
0
|
|
STX209 Then Placebo
Lost to Follow-up
|
0
|
1
|
|
STX209 Then Placebo
Withdrawal by Subject
|
0
|
1
|
|
STX209 Then Placebo
unable to return due to travel
|
0
|
1
|
|
Placebo Then STX209
Adverse Event
|
2
|
0
|
|
Placebo Then STX209
Protocol Violation
|
0
|
1
|
Baseline Characteristics
Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome
Baseline characteristics by cohort
| Measure |
STX209
n=30 Participants
STX209 Variable dose, 1mg bid to 10mg tid, oral capsules, 4weeks
|
Placebo
n=33 Participants
placebo variable dose (same flexible dose titration protocol) bid to tid, oral, 4weeks
|
Total
n=63 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
22 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
46 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age Continuous
|
14.2 years
STANDARD_DEVIATION 7.29 • n=5 Participants
|
13.9 years
STANDARD_DEVIATION 6.39 • n=7 Participants
|
14.1 years
STANDARD_DEVIATION 6.78 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
28 Participants
n=5 Participants
|
27 Participants
n=7 Participants
|
55 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
30 participants
n=5 Participants
|
33 participants
n=7 Participants
|
63 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: After 4 weeks of treatmentThe Aberrant Behavior Checklist-Community Edition (ABC-C) is a 58-item questionnaire composed of five different independent subscales. The questionnaire is completed by the parent/caregiver and lists aberrant behaviors and asks about the severity of the problem. ABC-Irritability is one of the subscales and comprises of 15 items. Minimum score is 0, maximum is 45. A decreased score indicates few aberrant behaviors and clinical improvement. The entire ABC-C assessment is administered at baseline and then at the end of each Intervention Period (4 weeks after Baseline).
Outcome measures
| Measure |
STX209:Placebo
n=63 Participants
First Intervention=STX209 (STX209 variable dose from 1mg bid to 10mg tid, capsule, oral) Second Intervention=Placebo (Placebo variable dose (same flexible dose titration protocol) bid to tid, oral)
Study Design:
Placebo-controlled, Crossover study. First Intervention(28 Days)-\> Withdrawal(14 Days) -\> Washout(7 Days)-\> Withdrawal (14 Days) Participants received all interventions.
|
Placebo:STX209
n=63 Participants
First Intervention=Placebo (Placebo variable dose (same flexible dose titration protocol) bid to tid, oral) Second Intervention=STX209 (STX209 variable dose from 1mg bid to 10mg tid, capsule, oral)
Study Design:
Placebo-controlled, Crossover study. First Intervention(28 Days)-\> Withdrawal(14 Days) -\> Washout(7 Days)-\> Withdrawal (14 Days) Participants received all interventions.
|
|---|---|---|
|
Aberrant Behavior Checklist Irritability Subscore
|
-5.1 Points on a scale
Standard Error 0.95
|
-5.3 Points on a scale
Standard Error 0.95
|
POST_HOC outcome
Timeframe: 4 week treatment periodAfter completion of the study, but during data analysis, the ABC-C assessment was independently re-validated in Fragile X Syndrome subjects. The subscales were re-factored into a Fragile-X Syndrome specific ABC-C (ABC-FX). The ABC-FX contains the same 58 questions as the original ABC-C but there are six subscales. One of the subscales is Social Avoidance, which consists of 4 items. Minimum score is 0, maximum is 12. A decreased score indicates fewer social avoidant behaviors. A post-hoc analysis was performed from the study data examining the social avoidance subscale of the ABC-FX.
Outcome measures
| Measure |
STX209:Placebo
n=63 Participants
First Intervention=STX209 (STX209 variable dose from 1mg bid to 10mg tid, capsule, oral) Second Intervention=Placebo (Placebo variable dose (same flexible dose titration protocol) bid to tid, oral)
Study Design:
Placebo-controlled, Crossover study. First Intervention(28 Days)-\> Withdrawal(14 Days) -\> Washout(7 Days)-\> Withdrawal (14 Days) Participants received all interventions.
|
Placebo:STX209
n=63 Participants
First Intervention=Placebo (Placebo variable dose (same flexible dose titration protocol) bid to tid, oral) Second Intervention=STX209 (STX209 variable dose from 1mg bid to 10mg tid, capsule, oral)
Study Design:
Placebo-controlled, Crossover study. First Intervention(28 Days)-\> Withdrawal(14 Days) -\> Washout(7 Days)-\> Withdrawal (14 Days) Participants received all interventions.
|
|---|---|---|
|
ABC-FXS Social Avoidance Subscore
|
-1.2 Points on a scale
Standard Error 0.24
|
-0.1 Points on a scale
Standard Error 0.24
|
Adverse Events
STX209
Serious events: 1 serious events
Other events: 42 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 31 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
STX209
n=63 participants at risk
STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks
|
Placebo
n=63 participants at risk
variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks
|
|---|---|---|
|
Psychiatric disorders
increased irritability
|
1.6%
1/63 • Number of events 1
|
0.00%
0/63
|
Other adverse events
| Measure |
STX209
n=63 participants at risk
STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks
|
Placebo
n=63 participants at risk
variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
upper respiratory tract infection
|
12.7%
8/63 • Number of events 8
|
9.5%
6/63 • Number of events 6
|
|
Nervous system disorders
headache
|
7.9%
5/63 • Number of events 5
|
1.6%
1/63 • Number of events 1
|
|
Nervous system disorders
sedation
|
7.9%
5/63 • Number of events 5
|
1.6%
1/63 • Number of events 1
|
|
General disorders
fatigue
|
6.3%
4/63 • Number of events 4
|
1.6%
1/63 • Number of events 1
|
|
Psychiatric disorders
irritability
|
6.3%
4/63 • Number of events 4
|
6.3%
4/63 • Number of events 4
|
|
Gastrointestinal disorders
diarrhea
|
4.8%
3/63 • Number of events 3
|
7.9%
5/63 • Number of events 5
|
|
Metabolism and nutrition disorders
increased appetite
|
6.3%
4/63 • Number of events 4
|
3.2%
2/63 • Number of events 2
|
|
Gastrointestinal disorders
vomiting
|
6.3%
4/63 • Number of events 4
|
1.6%
1/63 • Number of events 1
|
|
Psychiatric disorders
aggression
|
4.8%
3/63 • Number of events 3
|
6.3%
4/63 • Number of events 4
|
|
Infections and infestations
nasopharyngitis
|
3.2%
2/63 • Number of events 2
|
9.5%
6/63 • Number of events 6
|
Additional Information
Dr. Paul Wang, Vice President of Clinical and Medical Affairs
Seaside Therapeutics
Phone: 617-374-9009
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Seaside Therapeutics agreements with investigators vary; constant is our right to embargo communications regarding trial results prior to public release for a period ≤60 days from submittal for review. We will not prohibit investigators from publishing, but will prohibit the disclosure of previously undisclosed confidential information other than study results, and request postponement of single-center publications until after disclosure of the clinical trial's primary publication.
- Publication restrictions are in place
Restriction type: OTHER