Clinical trial NCT01053156: Trial of Minocycline to Treat Children With Fragile X Syndrome
Official title: Randomized Double-Blind Controlled Cross Over Trial of Minocycline in Children With Fragile X Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Fragile X Syndrome
Keywords: Fragile X syndrome
minocycline
targeted treatment
Lead sponsor: University of California, Davis
Sponsor class: OTHER
Collaborators: name: The National Fragile X Foundation
First posted: 2010-01-21
Enrollment: 66
Sex: ALL
Minimum age (years): 42
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: This is a single center study at the UC Davis MIND Institute in patients age 3.5-16 years of age with fragile X syndrome (FXS), funded by a National Fragile X Foundation Grant. It is a controlled trial of minocycline, an antibiotic commonly used in children for infection or for treatment of neurodegenerative disorders. We are investigating its use in FXS because it lowers matrix metalloproteinase 9 (MMP9) levels, which are high in FXS, and it also strengthens brain connections in the animal models of FXS. We hypothesize that minocycline will likely be helpful for language, behavior and/or cognition in fragile X patients.

Detailed description: This is a single center study at the UC Davis MIND Institute in patients age 3.5-16 years of age with fragile X syndrome (FXS), funded by a National Fragile X Foundation Grant. It is a controlled trial of minocycline, an antibiotic commonly used in children for infection or for treatment of neurodegenerative disorders. We are investigating its use in FXS because it lowers matrix metalloproteinase 9 (MMP9) levels, which are high in FXS, and it also strengthens brain connections in the animal models of FXS. We hypothesize that minocycline will likely be helpful for language, behavior and/or cognition in fragile X patients.

The aim of this study is to carry out a double-blind placebo controlled trial of minocycline treatment in children with FXS who are 3.5 to 16 years of age. At baseline, we will assess behavior and perceptual and cognitive development. After the children have been treated for 3 months with either minocycline or placebo, they undergo the same baseline testing. They will then cross over and be treated for a second 3 months. We will carry out testing again at the end of the second 3 month period. We will also assess the side effects of minocycline treatment throughout the study.

Eligibility criteria: Inclusion Criteria:

* Must have fragile X syndrome with molecular documentation
* Current pharmacological treatment regimen has been stable for at least 4 weeks

Exclusion Criteria:

* Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study
* subjects who are unable to take oral medication
* subjects who have been on minocycline previously
* subjects who are allergic to minocycline or tetracyclines
* subjects who are pregnant
* subjects with history of lupus or hepatic dysfunction

Primary outcomes:
measure: Clinical Global Impression Scale (CGI)
timeFrame: 3 months (post first treatment) and 6 months (post second treatment)
measure: Visual Analogue Scale- Behavior 1
timeFrame: Baseline, 3 months, 6 months
Secondary outcomes:
measure: Visual Analogue Scale- Behaviors 2
timeFrame: Baseline, 3 months, 6 months
measure: Expressive Vocabulary Test-2
timeFrame: Baseline, 3 months and 6 months
measure: Vineland Adaptive Behavior Scale-II (VABS-II)Adaptive Behavior Composite Score
timeFrame: Baseline, 3 months, and 6 months
measure: Aberrant Behavior Checklist-Community Edition (ABC-C)Composite Score
timeFrame: Baseline, 3 months, and 6 months
measure: Visual Analogue Scale Behavior 3- VAS3
timeFrame: Baseline, 3 months, 6 months
measure: VAS Categorized by Behavior: Aggression/ ADHD
timeFrame: Baseline, 3 months, 6 months
measure: VAS Categorized by Behavior:Anxiety/ Mood
timeFrame: Baseline, 3 months, 6 months
measure: VAS Categorized by Behavior:Language/ Cognition
timeFrame: Baseline, 3 months, 6 months
measure: VAS Categorized by Behavior: Other
timeFrame: Baseline, 3 months, 6 months
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: M.I.N.D. Institute at University of California at Davis Medical Center
state: California
Study officials:
affiliation: M.I.N.D. Institute at University of California at Davis, Sacramento CA
name: Randi J Hagerman, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Bilousova TV, Dansie L, Ngo M, Aye J, Charles JR, Ethell DW, Ethell IM. Minocycline promotes dendritic spine maturation and improves behavioural performance in the fragile X mouse model. J Med Genet. 2009 Feb;46(2):94-102. doi: 10.1136/jmg.2008.061796. Epub 2008 Oct 3.