N/A
N=20
Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome
Fragile X Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00858689 ↗Enrolled (actual)
20
Serious AEs
0.0%
Results posted
Feb 2016
Primary outcome: Primary: Change From Baseline of ABC Irritability Subtest Score at 8 Weeks — -14.37 units on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- Minocycline (Drug)
- Age
- Pediatric, Adult · 13+ yrs
- Sex
- All
- Sponsor
- FRAXA Research Foundation
- Primary completion
- Nov 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline of ABC Irritability Subtest Score at 8 Weeks |
-14.37 | — |
| PRIMARY ABC Irritability Subtest Score |
— | — |
| PRIMARY ABC Irritability Subtest Score |
— | — |
| SECONDARY Parent Defined Target Symptoms Scale-Visual |
— | — |
| SECONDARY Clinical Global Impression Scale |
— | — |
| SECONDARY Stanford Binet 5 (SB5) |
— | — |
| SECONDARY The Peabody Picture Vocabulary Test Third Edition (PPVT-III) |
— | — |
| SECONDARY The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) |
— | — |
| SECONDARY Non-Verbal Associative Learning Task (NVALT) |
— | — |
| SECONDARY Vineland Adaptive Behaviour Scales (VABS) |
— | — |
| SECONDARY Parent Defined Target Symptoms Scale-Visual |
— | — |
| SECONDARY Parent Defined Target Symptoms Scale-Visual |
— | — |
| SECONDARY Clinical Global Impression Scale |
— | — |
| SECONDARY Clinical Global Impression Scale |
— | — |
| SECONDARY Stanford Binet 5 (SB5) |
— | — |
| SECONDARY The Peabody Picture Vocabulary Test Third Edition (PPVT-III) |
— | — |
| SECONDARY The Peabody Picture Vocabulary Test Third Edition (PPVT-III) |
— | — |
| SECONDARY The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) |
— | — |
| SECONDARY The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) |
— | — |
| SECONDARY Non-Verbal Associative Learning Task (NVALT) |
— | — |
| SECONDARY Non-Verbal Associative Learning Task (NVALT) |
— | — |
| SECONDARY Vineland Adaptive Behaviour Scales (VABS) |
— | — |
| SECONDARY Vineland Adaptive Behaviour Scales (VABS) |
— | — |
Summary
Fragile X Syndrome (FXS) is the most common known inherited form of mental impairment, developmental disability and autism. Minocycline is an antibiotic that has recently been used to treat the mouse model for Fragile X, and was found to reverse the structural abnormalities that are seen their brain cells. The purpose of this research study is to determine if minocycline is an effective treatment for patients with fragile X syndrome (FXS).
Eligibility Criteria
Inclusion Criteria
- Diagnosis of FXS by clinical evaluation and confirmed by FMR1-DNA testing with presence of full mutation or mosaicism for the full mutation. Prior DNA testing reports will be accepted, when available.
- Age between 13 to 35 years inclusive at the time of informed consent.
- Male or female
- CGI-Severity Score of 4 or greater, indicative of moderate or greater severity of behavioural problems. This is a 7-point scale of clinical global impression of severity that the clinician fills out after considering all the available information on the patient, including the parent history, the examination in clinic, reports from the school and other sources.
- Score of 9 or greater on the Aberrant Behaviour Checklist - Irritability Scale (top 50th %-tile). The ABC is a global behaviour checklist implemented for the measurement of drug and other treatment effects in mentally impaired individuals. It is made up of 5 empirically derived dimensions including irritability, lethargy/withdrawal, inappropriate speech, hyperactivity, and stereotypic behaviour based on 58 items that describe various behavioural problems.
- Availability of parent and/or caregiver for all clinic visits and assessments.
- English language fluency and reading level of 6th grade or greater in one caregiver.
Exclusion Criteria
- Allergy to minocycline.
- Kidney disease or elevated renal function tests.
- Liver disease or elevated liver function tests.
- Participants with neutropenia, anemia, or thrombocytopenia.
- History of systemic lupus erythematosus or screening anti-nuclear antibody (ANA) titre of >1:40, as minocycline may cause a lupus-like reaction.
- Individuals who do not have a mother or caregiver who is willing to participate in the clinic visits.
- Individuals who are pregnant or at risk to become pregnant, specifically sexually active females will be excluded.
- Presence of persistent psychotic symptoms
- Subjects with symptom severity likely judged to endanger personal safety or safety of others.
- History of systemic lupus erythematosus or screening anti-nuclear antibody (ANA) titre of >1:40, as minocycline may cause a lupus-like reaction.
Data sourced from ClinicalTrials.gov (NCT00858689). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.