Trial of Minocycline to Treat Children With Fragile X Syndrome

This study has been completed.
Sponsor:
Collaborator:
The National Fragile X Foundation
Information provided by (Responsible Party):
Randi J. Hagerman, MD, University of California, Davis
ClinicalTrials.gov Identifier:
NCT01053156
First received: January 19, 2010
Last updated: June 30, 2014
Last verified: June 2014
Results First Received: February 8, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator);   Primary Purpose: Treatment
Condition: Fragile X Syndrome
Interventions: Drug: minocycline hydrochloride
Drug: Placebo

  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
Recruitment occurred from January 2010 to June 2011, with the last participants completing the study in December 2011. The study site was a single site, the MIND Institute at the University of California Davis Medical Center.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Inclusion criteria: FXS confirmed by FMR1 DNA, age 3.5-16 years and stable pharmacological regimen > 4 weeks prior to study. Exclusion criteria:previous minocycline treatment, plans to change pharmacological intervention or allergy to tetracyclines. 66 patients enrolled; 55 patients with any data on outcome measures analyzed (11 withdrawn).

Reporting Groups
  Description
Minocycline First, Then Placebo Second Minocycline hydrochloride dosed orally once a day for 3 months, the switched to placebo dosed orally once a day for 3 months.
Placebo First, Minocycline Second Placebo will be given once daily for 3 months, then minocycline dosed once daily for 3 months

Participant Flow for 2 periods

Period 1:   First Intervention (Week 1-12)
    Minocycline First, Then Placebo Second     Placebo First, Minocycline Second  
STARTED     33     33  
COMPLETED     28     27  
NOT COMPLETED     5     6  

Period 2:   Second Intervention (Week 13-24)
    Minocycline First, Then Placebo Second     Placebo First, Minocycline Second  
STARTED     28     27  
COMPLETED     26     22  
NOT COMPLETED     2     5  



  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.
The baseline participants are different in number from those started in the participant flow module because in our intent to treat analysis, only those who completed one or more treatment periods were included in the analysis.

Reporting Groups
  Description
Minocycline First, Placebo Second Minocycline hydrochloride dosed orally once a day for 3 months, the switched to placebo dosed orally once a day for 3 months.
Placebo First, Minocycline Second Placebo will be given once daily for 3 months, then minocycline dosed once daily for 3 months
Total Total of all reporting groups

Baseline Measures
    Minocycline First, Placebo Second     Placebo First, Minocycline Second     Total  
Number of Participants  
[units: participants]
  28     27     55  
Age  
[units: participants]
     
<=18 years     28     27     55  
Between 18 and 65 years     0     0     0  
>=65 years     0     0     0  
Age  
[units: years]
Mean ± Standard Deviation
  9.01  ± 3.76     9.4  ± 3.39     9.21  ± 3.57  
Gender  
[units: participants]
     
Female     5     3     8  
Male     23     24     47  
Region of Enrollment  
[units: participants]
     
United States     28     27     55  



  Outcome Measures
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1.  Primary:   Clinical Global Impression Scale (CGI)   [ Time Frame: 3 months (post first treatment) and 6 months (post second treatment) ]

2.  Primary:   Visual Analogue Scale- Behavior 1   [ Time Frame: Baseline, 3 months, 6 months ]

3.  Secondary:   Visual Analogue Scale- Behaviors 2   [ Time Frame: Baseline, 3 months, 6 months ]

4.  Secondary:   Expressive Vocabulary Test-2   [ Time Frame: Baseline, 3 months and 6 months ]

5.  Secondary:   Vineland Adaptive Behavior Scale-II (VABS-II)Adaptive Behavior Composite Score   [ Time Frame: Baseline, 3 months, and 6 months ]

6.  Secondary:   Aberrant Behavior Checklist-Community Edition (ABC-C)Composite Score   [ Time Frame: Baseline, 3 months, and 6 months ]

7.  Secondary:   Visual Analogue Scale Behavior 3- VAS3   [ Time Frame: Baseline, 3 months, 6 months ]

8.  Secondary:   VAS Categorized by Behavior: Aggression/ ADHD   [ Time Frame: Baseline, 3 months, 6 months ]

9.  Secondary:   VAS Categorized by Behavior:Anxiety/ Mood   [ Time Frame: Baseline, 3 months, 6 months ]

10.  Secondary:   VAS Categorized by Behavior:Language/ Cognition   [ Time Frame: Baseline, 3 months, 6 months ]

11.  Secondary:   VAS Categorized by Behavior: Other   [ Time Frame: Baseline, 3 months, 6 months ]


  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
Results may be potentially biased by study design weaknesses, including unblinding of subjects when they completed the study, drug-related side effects unblinding and preliminary efficacy analysis results known to investigators.


  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: Randi J. Hagerman MD, Medical Director of the MIND Institute
Organization: MIND Institute, University of California Davis Medical Center
phone: 916-703-0247
e-mail: randi.hagerman@ucdmc.ucdavis.edu


Publications:

Responsible Party: Randi J. Hagerman, MD, University of California, Davis
ClinicalTrials.gov Identifier: NCT01053156     History of Changes
Other Study ID Numbers: 200917522-1
Study First Received: January 19, 2010
Results First Received: February 8, 2013
Last Updated: June 30, 2014
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration