Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome

This study has been completed.
Sponsor:
Collaborator:
Fragile X Research Foundation of Canada
Information provided by (Responsible Party):
FRAXA Research Foundation
ClinicalTrials.gov Identifier:
NCT00858689
First received: March 9, 2009
Last updated: February 16, 2016
Last verified: February 2016
Results First Received: September 1, 2013  
Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Fragile X Syndrome
Intervention: Drug: Minocycline

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Twenty subjects with FXS between 13 and 35 years of age were enrolled between December 2007 and June 2008, and after baseline testing they were started on an 8-week treatment course of minocycline added on to any other medications being administered at the time of enrollment.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Inclusion criteria included (1) diagnosis of FXS by clinical evaluation and confirmed by FMR1-DNA testing with presence of full mutation or mosaicism for the full mutation.

Reporting Groups
  Description
Minocyline 50 mg or 100 mg PO BID open label, single arm study of minocyline 50 mg or 100 mg PO BID

Participant Flow:   Overall Study
    Minocyline 50 mg or 100 mg PO BID  
STARTED     20  
COMPLETED     19  
NOT COMPLETED     1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Minocyline 50 mg or 100 mg PO BID open label minocyline 50 mg or 100 mg PO BID

Baseline Measures
    Minocyline 50 mg or 100 mg PO BID  
Number of Participants  
[units: participants]
  20  
Age  
[units: participants]
 
<=18 years     11  
Between 18 and 65 years     9  
>=65 years     0  
Age  
[units: years]
Mean (Standard Deviation)
  18  (5)  
Gender  
[units: participants]
 
Female     2  
Male     18  
Region of Enrollment  
[units: participants]
 
Canada     20  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Change From Baseline of ABC Irritability Subtest Score at 8 Weeks   [ Time Frame: Baseline and 8 weeks ]

2.  Primary:   ABC Irritability Subtest Score   [ Time Frame: 8 weeks ]

3.  Primary:   ABC Irritability Subtest Score   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

4.  Secondary:   Parent Defined Target Symptoms Scale-Visual   [ Time Frame: Baseline ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

5.  Secondary:   Clinical Global Impression Scale   [ Time Frame: Baseline ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

6.  Secondary:   Stanford Binet 5 (SB5)   [ Time Frame: Baseline ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

7.  Secondary:   The Peabody Picture Vocabulary Test Third Edition (PPVT-III)   [ Time Frame: Baseline ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

8.  Secondary:   The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)   [ Time Frame: Baseline ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

9.  Secondary:   Non-Verbal Associative Learning Task (NVALT)   [ Time Frame: Baseline ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

10.  Secondary:   Vineland Adaptive Behaviour Scales (VABS)   [ Time Frame: Baseline ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

11.  Secondary:   Parent Defined Target Symptoms Scale-Visual   [ Time Frame: 8 weeks ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

12.  Secondary:   Parent Defined Target Symptoms Scale-Visual   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

13.  Secondary:   Clinical Global Impression Scale   [ Time Frame: 8 weeks ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

14.  Secondary:   Clinical Global Impression Scale   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

15.  Secondary:   Stanford Binet 5 (SB5)   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

16.  Secondary:   The Peabody Picture Vocabulary Test Third Edition (PPVT-III)   [ Time Frame: 8 weeks ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

17.  Secondary:   The Peabody Picture Vocabulary Test Third Edition (PPVT-III)   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

18.  Secondary:   The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)   [ Time Frame: 8 weeks ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

19.  Secondary:   The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

20.  Secondary:   Non-Verbal Associative Learning Task (NVALT)   [ Time Frame: 8 weeks ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

21.  Secondary:   Non-Verbal Associative Learning Task (NVALT)   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

22.  Secondary:   Vineland Adaptive Behaviour Scales (VABS)   [ Time Frame: 8 weeks ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

23.  Secondary:   Vineland Adaptive Behaviour Scales (VABS)   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
This was an open-label trial. Further study in a randomized controlled trial is warranted.


  More Information
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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:  
Name/Title: Michael R. Tranfaglia MD
Organization: FRAXA Research Foundation
phone: 978-462-1990
e-mail: info@fraxa.org


Publications of Results:

Responsible Party: FRAXA Research Foundation
ClinicalTrials.gov Identifier: NCT00858689     History of Changes
Other Study ID Numbers: 010308
Study First Received: March 9, 2009
Results First Received: September 1, 2013
Last Updated: February 16, 2016
Health Authority: Canada: Health Canada