Clinical trial NCT02680379: Combined Treatment of Minocycline and Lovastatin to Treat Individuals With Fragile X Syndrome
Official title: A Pilot Study Exploring the Safety and Synergistic Effect of a Minocycline/Lovastatin Combined Treatment on the Behavior of Individuals With Fragile X Syndrome; Validation of New Biochemical and Neurophysiological Markers (LovaMiX)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fragile X Syndrome
Keywords: Fragile X Syndrome
Autism
FMR1
FRAXA
Lead sponsor: Université de Sherbrooke
Sponsor class: OTHER
Collaborators: name: FRAXA Research Foundation
First posted: 2016-02-11
Enrollment: 22
Sex: ALL
Minimum age (years): 8
Maximum age (years): 45
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine whether Lovastatin, Minocycline and the combination Lovastatin/Minocycline are effective in treating behavioral symptoms in Fragile X individuals.


Eligibility criteria: Inclusion Criteria:

* Molecular diagnosis of fragile X syndrome
* The participant must be accompanied his parent, legal tutor or legal representative.
* Identify a caregiver who spends at least six hours per day with the participant (may be the parent, legal tutor, legal representative or an other person).
* IQ \< 70
* ABC-C score \> 20
* CGI-Severity score ≥ 4

Exclusion Criteria:

* Pregnant or breastfeeding participants
* Previous intolerance/allergy to statins, minocycline or tetracyclines
* Participants who have taken lovastatin or minocycline in the last 12 weeks
* Personal history of myopathy, myalgia or high creatine kinase (CK) levels
* Renal disease / liver disease / disturbed hepatorenal tests
* Participants taking more than three psychoactive medications (except anticonvulsants)
* Untreated or uncontrolled hypothyroidism
* Any other active medical condition
* Modification of psychoactive treatment in the last 6 weeks prior to randomization
* Participants under the age of 13 years who have incomplete formation of the crown of their teeth (except possibly their 3rd molars) as shown by panorex
* Concomitant use of prohibited drugs

  * Prohibited drugs include other hypolipemic including gemfibrozil (or other fibrates) and niacin (nicotinic acid), angiotensin converting enzyme (ACE), cyclosporine, danazol, amiodarone, verapamil and inhibitors P450 (CYP3A4) (itraconazole, ketoconazole, erythromycin, clarithromycin, telithromycin, inhibitors of HIV protease and nefazodone).

Primary outcomes:
measure: Change from baseline Aberrant Behavior Checklist-Community (ABC-C) total score at 8,12 and 20 weeks
timeFrame: baseline, 8 weeks, 12 weeks, 20 weeks
Secondary outcomes:
measure: Clinical Global Impression Scale improvement (CGI-I)
timeFrame: baseline, 8 weeks, 12 weeks, 20 weeks
measure: Change from baseline Social Responsiveness Scale (SRS) at 8 and 20 weeks
timeFrame: baseline, 8 weeks, 20 weeks
measure: Anxiety, depression and mood scale (ADAMS), change from baseline to 8 and 20 weeks
timeFrame: baseline, 8 weeks, 20 weeks
measure: Behavior Rating Inventory of Executive Function (BRIEF)
timeFrame: Before treatment and at the end of treatment (weeks 20)
measure: Change from baseline Vineland II; adaptive behaviour scale at 20 weeks
timeFrame: baseline, 20 weeks
Other outcomes:
measure: (optional) Change in brain activity using Functional Magnetic Resonance Imaging (fMRI) at 8 and 20 weeks
timeFrame: baseline, 8 weeks, 20 weeks
measure: (optional) Change in neurochemistry using Transcranial Magnetic Stimulation (TMS) at 8 and 20 weeks
timeFrame: baseline, 8 weeks, 20 weeks
Locations:
city: Sherbrooke
country: Canada
facility: Centre de Recherche du CHUS
state: Quebec
Study officials:
affiliation: Fragile X Clinic, Centre de recherche du CHUS
name: François Corbin, MD/PhD
role: PRINCIPAL_INVESTIGATOR
References: