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Trial record 27 of 53 for:    Developmental Disabilities | ( Map: Indiana, United States )

Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome

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ClinicalTrials.gov Identifier: NCT00788073
Recruitment Status : Completed
First Posted : November 10, 2008
Results First Posted : May 6, 2013
Last Update Posted : May 6, 2013
Sponsor:
Information provided by (Responsible Party):
Seaside Therapeutics, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Fragile X Syndrome
Interventions Drug: STX209
Drug: Placebo
Enrollment 63
Recruitment Details  
Pre-assignment Details  
Arm/Group Title STX209:Placebo Placebo:STX209
Hide Arm/Group Description

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.

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.

Period Title: STX209 Then Placebo
Started 30 33
Completed 29 30
Not Completed 1 3
Reason Not Completed
Adverse Event             1             0
Lost to Follow-up             0             1
Withdrawal by Subject             0             1
unable to return due to travel             0             1
Period Title: Placebo Then STX209
Started 29 30
Completed 27 29
Not Completed 2 1
Reason Not Completed
Adverse Event             2             0
Protocol Violation             0             1
Arm/Group Title STX209 Placebo Total
Hide Arm/Group Description STX209 Variable dose, 1mg bid to 10mg tid, oral capsules, 4weeks placebo variable dose (same flexible dose titration protocol) bid to tid, oral, 4weeks Total of all reporting groups
Overall Number of Baseline Participants 30 33 63
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 30 participants 33 participants 63 participants
<=18 years
22
  73.3%
24
  72.7%
46
  73.0%
Between 18 and 65 years
8
  26.7%
9
  27.3%
17
  27.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 30 participants 33 participants 63 participants
14.2  (7.29) 13.9  (6.39) 14.1  (6.78)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 30 participants 33 participants 63 participants
Female
2
   6.7%
6
  18.2%
8
  12.7%
Male
28
  93.3%
27
  81.8%
55
  87.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 30 participants 33 participants 63 participants
30 33 63
1.Primary Outcome
Title Aberrant Behavior Checklist Irritability Subscore
Hide Description 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).
Time Frame After 4 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title STX209:Placebo Placebo:STX209
Hide Arm/Group Description:

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.

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.

Overall Number of Participants Analyzed 63 63
Least Squares Mean (Standard Error)
Unit of Measure: Points on a scale
-5.1  (0.95) -5.3  (0.95)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection STX209:Placebo, Placebo:STX209
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.05
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -0.2
Estimation Comments [Not Specified]
2.Post-Hoc Outcome
Title ABC-FXS Social Avoidance Subscore
Hide Description After 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.
Time Frame 4 week treatment period
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title STX209:Placebo Placebo:STX209
Hide Arm/Group Description:

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.

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.

Overall Number of Participants Analyzed 63 63
Least Squares Mean (Standard Error)
Unit of Measure: Points on a scale
-1.2  (0.24) -0.1  (0.24)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection STX209:Placebo, Placebo:STX209
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.008
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.0
Estimation Comments [Not Specified]
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title STX209 Placebo
Hide Arm/Group Description STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks
All-Cause Mortality
STX209 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
STX209 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/63 (1.59%)      0/63 (0.00%)    
Psychiatric disorders     
increased irritability *  1/63 (1.59%)  1 0/63 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
STX209 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   42/63 (66.67%)      31/63 (49.21%)    
Gastrointestinal disorders     
diarrhea *  3/63 (4.76%)  3 5/63 (7.94%)  5
vomiting *  4/63 (6.35%)  4 1/63 (1.59%)  1
General disorders     
fatigue *  4/63 (6.35%)  4 1/63 (1.59%)  1
Infections and infestations     
nasopharyngitis *  2/63 (3.17%)  2 6/63 (9.52%)  6
Metabolism and nutrition disorders     
increased appetite *  4/63 (6.35%)  4 2/63 (3.17%)  2
Nervous system disorders     
headache *  5/63 (7.94%)  5 1/63 (1.59%)  1
sedation *  5/63 (7.94%)  5 1/63 (1.59%)  1
Psychiatric disorders     
irritability *  4/63 (6.35%)  4 4/63 (6.35%)  4
aggression *  3/63 (4.76%)  3 4/63 (6.35%)  4
Respiratory, thoracic and mediastinal disorders     
upper respiratory tract infection *  8/63 (12.70%)  8 6/63 (9.52%)  6
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
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.
Results Point of Contact
Name/Title: Dr. Paul Wang, Vice President of Clinical and Medical Affairs
Organization: Seaside Therapeutics
Phone: 617-374-9009 ext 1015
Responsible Party: Seaside Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT00788073     History of Changes
Other Study ID Numbers: 22001
First Submitted: November 7, 2008
First Posted: November 10, 2008
Results First Submitted: February 7, 2013
Results First Posted: May 6, 2013
Last Update Posted: May 6, 2013