Fragile X Syndrome Clinical Trial
Official title:
Add-on Pilot Trial of Minocycline in Fragile X Syndrome
Verified date | February 2016 |
Source | FRAXA Research Foundation |
Contact | n/a |
Is FDA regulated | No |
Health authority | Canada: Health Canada |
Study type | Interventional |
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).
Status | Completed |
Enrollment | 20 |
Est. completion date | January 2009 |
Est. primary completion date | November 2008 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | Both |
Age group | 13 Years to 35 Years |
Eligibility |
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. |
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
Canada | Surrey Place Centre | Toronto | Ontario |
Lead Sponsor | Collaborator |
---|---|
FRAXA Research Foundation | Fragile X Research Foundation of Canada |
Canada,
Paribello C, Tao L, Folino A, Berry-Kravis E, Tranfaglia M, Ethell IM, Ethell DW. Open-label add-on treatment trial of minocycline in fragile X syndrome. BMC Neurol. 2010 Oct 11;10:91. doi: 10.1186/1471-2377-10-91. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change From Baseline of ABC Irritability Subtest Score at 8 Weeks | 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. | Baseline and 8 weeks | No |
Primary | ABC Irritability Subtest Score | ABC Irritability subtest score was used | 8 weeks | No |
Primary | ABC Irritability Subtest Score | ABC (Aberrant behavior checklist) Irritability subtest score was used | 1 year | No |
Secondary | Parent Defined Target Symptoms Scale-Visual | Baseline | No | |
Secondary | Clinical Global Impression Scale | Baseline | No | |
Secondary | Stanford Binet 5 (SB5) | Baseline | No | |
Secondary | The Peabody Picture Vocabulary Test Third Edition (PPVT-III) | Baseline | No | |
Secondary | The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) | Baseline | No | |
Secondary | Non-Verbal Associative Learning Task (NVALT) | Baseline | No | |
Secondary | Vineland Adaptive Behaviour Scales (VABS) | Baseline | No | |
Secondary | Parent Defined Target Symptoms Scale-Visual | 8 weeks | No | |
Secondary | Parent Defined Target Symptoms Scale-Visual | 1 year | No | |
Secondary | Clinical Global Impression Scale | 8 weeks | No | |
Secondary | Clinical Global Impression Scale | 1 year | No | |
Secondary | Stanford Binet 5 (SB5) | 1 year | No | |
Secondary | The Peabody Picture Vocabulary Test Third Edition (PPVT-III) | 8 weeks | No | |
Secondary | The Peabody Picture Vocabulary Test Third Edition (PPVT-III) | 1 year | No | |
Secondary | The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) | 8 weeks | No | |
Secondary | The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) | 1 year | No | |
Secondary | Non-Verbal Associative Learning Task (NVALT) | 8 weeks | No | |
Secondary | Non-Verbal Associative Learning Task (NVALT) | 1 year | No | |
Secondary | Vineland Adaptive Behaviour Scales (VABS) | 8 weeks | No | |
Secondary | Vineland Adaptive Behaviour Scales (VABS) | 1 year | No |
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