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Trial record 30 of 74 for:    "fragile x syndrome"

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

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ClinicalTrials.gov Identifier: NCT00858689
Recruitment Status : Completed
First Posted : March 10, 2009
Results First Posted : February 18, 2016
Last Update Posted : February 18, 2016
Sponsor:
Collaborator:
Fragile X Research Foundation of Canada
Information provided by (Responsible Party):
FRAXA Research Foundation

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fragile X Syndrome
Intervention Drug: Minocycline
Enrollment 20
Recruitment Details 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 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.
Arm/Group Title Minocyline 50 mg or 100 mg PO BID
Hide Arm/Group Description open label, single arm study of minocyline 50 mg or 100 mg PO BID
Period Title: Overall Study
Started 20
Completed 19
Not Completed 1
Arm/Group Title Minocyline 50 mg or 100 mg PO BID
Hide Arm/Group Description open label minocyline 50 mg or 100 mg PO BID
Overall Number of Baseline Participants 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
<=18 years
11
  55.0%
Between 18 and 65 years
9
  45.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants
18  (5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Female
2
  10.0%
Male
18
  90.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Canada Number Analyzed 20 participants
20
1.Primary Outcome
Title Change From Baseline of ABC Irritability Subtest Score at 8 Weeks
Hide Description The 15-item Irritability Scale includes questions about aggression, self-injury, tantrums, agitation, and unstable mood on a scale of 0 to 45 with higher scores indicating greater severity. This scale has been successfully used in previous medication studies in children with autism and in patients with FXS and in a controlled trial of ampakine CX516 in FXS. All ABC subscales showed good reliability when used by parents and caregivers of individuals with FXS to assess behavior in the CX516 study NCT00054730, and yielded intraclass correlation coefficient (ICC) values of 0.7-0.9.
Time Frame Baseline and 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ABC-I change in subjects completing 8 weeks of minocycline treatment
Arm/Group Title Minocyline 50 mg or 100 mg PO BID
Hide Arm/Group Description:
open label minocyline 50 mg or 100 mg PO BID
Overall Number of Participants Analyzed 19
Mean (Standard Deviation)
Unit of Measure: units on a scale
-14.37  (7.84)
2.Primary Outcome
Title ABC Irritability Subtest Score
Hide Description ABC Irritability subtest score was used
Time Frame 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
completed 8 weeks of minocycline treatment
Arm/Group Title Minocyline 50 mg or 100 mg PO BID
Hide Arm/Group Description:
open label minocyline 50 mg or 100 mg PO BID
Overall Number of Participants Analyzed 19
Mean (Standard Deviation)
Unit of Measure: units on a scale
7.74  (4.82)
3.Primary Outcome
Title ABC Irritability Subtest Score
Hide Description ABC (Aberrant behavior checklist) Irritability subtest score was used
Time Frame 1 year
Outcome Measure Data Not Reported
4.Secondary Outcome
Title Parent Defined Target Symptoms Scale-Visual
Hide Description [Not Specified]
Time Frame Baseline
Outcome Measure Data Not Reported
5.Secondary Outcome
Title Clinical Global Impression Scale
Hide Description [Not Specified]
Time Frame Baseline
Outcome Measure Data Not Reported
6.Secondary Outcome
Title Stanford Binet 5 (SB5)
Hide Description [Not Specified]
Time Frame Baseline
Outcome Measure Data Not Reported
7.Secondary Outcome
Title The Peabody Picture Vocabulary Test Third Edition (PPVT-III)
Hide Description [Not Specified]
Time Frame Baseline
Outcome Measure Data Not Reported
8.Secondary Outcome
Title The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)
Hide Description [Not Specified]
Time Frame Baseline
Outcome Measure Data Not Reported
9.Secondary Outcome
Title Non-Verbal Associative Learning Task (NVALT)
Hide Description [Not Specified]
Time Frame Baseline
Outcome Measure Data Not Reported
10.Secondary Outcome
Title Vineland Adaptive Behaviour Scales (VABS)
Hide Description [Not Specified]
Time Frame Baseline
Outcome Measure Data Not Reported
11.Secondary Outcome
Title Parent Defined Target Symptoms Scale-Visual
Hide Description [Not Specified]
Time Frame 8 weeks
Outcome Measure Data Not Reported
12.Secondary Outcome
Title Parent Defined Target Symptoms Scale-Visual
Hide Description [Not Specified]
Time Frame 1 year
Outcome Measure Data Not Reported
13.Secondary Outcome
Title Clinical Global Impression Scale
Hide Description [Not Specified]
Time Frame 8 weeks
Outcome Measure Data Not Reported
14.Secondary Outcome
Title Clinical Global Impression Scale
Hide Description [Not Specified]
Time Frame 1 year
Outcome Measure Data Not Reported
15.Secondary Outcome
Title Stanford Binet 5 (SB5)
Hide Description [Not Specified]
Time Frame 1 year
Outcome Measure Data Not Reported
16.Secondary Outcome
Title The Peabody Picture Vocabulary Test Third Edition (PPVT-III)
Hide Description [Not Specified]
Time Frame 8 weeks
Outcome Measure Data Not Reported
17.Secondary Outcome
Title The Peabody Picture Vocabulary Test Third Edition (PPVT-III)
Hide Description [Not Specified]
Time Frame 1 year
Outcome Measure Data Not Reported
18.Secondary Outcome
Title The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)
Hide Description [Not Specified]
Time Frame 8 weeks
Outcome Measure Data Not Reported
19.Secondary Outcome
Title The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)
Hide Description [Not Specified]
Time Frame 1 year
Outcome Measure Data Not Reported
20.Secondary Outcome
Title Non-Verbal Associative Learning Task (NVALT)
Hide Description [Not Specified]
Time Frame 8 weeks
Outcome Measure Data Not Reported
21.Secondary Outcome
Title Non-Verbal Associative Learning Task (NVALT)
Hide Description [Not Specified]
Time Frame 1 year
Outcome Measure Data Not Reported
22.Secondary Outcome
Title Vineland Adaptive Behaviour Scales (VABS)
Hide Description [Not Specified]
Time Frame 8 weeks
Outcome Measure Data Not Reported
23.Secondary Outcome
Title Vineland Adaptive Behaviour Scales (VABS)
Hide Description [Not Specified]
Time Frame 1 year
Outcome Measure Data Not Reported
Time Frame 8 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Minocyline 50 mg or 100 mg PO BID
Hide Arm/Group Description open label minocyline 50 mg or 100 mg PO BID
All-Cause Mortality
Minocyline 50 mg or 100 mg PO BID
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Minocyline 50 mg or 100 mg PO BID
Affected / at Risk (%) # Events
Total   0/20 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Minocyline 50 mg or 100 mg PO BID
Affected / at Risk (%) # Events
Total   2/20 (10.00%)    
Immune system disorders   
Increased serum ANA titre  [1]  2/20 (10.00%)  2
Indicates events were collected by systematic assessment
[1]
two participants developed an asymptomatic seroconversion of their ANA, both exhibiting a 1/80 titre with a nucleolar pattern
This was an open-label trial. Further study in a randomized controlled trial is warranted.
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
Responsible Party: FRAXA Research Foundation
ClinicalTrials.gov Identifier: NCT00858689     History of Changes
Other Study ID Numbers: 010308
First Submitted: March 9, 2009
First Posted: March 10, 2009
Results First Submitted: September 1, 2013
Results First Posted: February 18, 2016
Last Update Posted: February 18, 2016