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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00858689
• Date of registration: 09/03/2009
• Prospective Registration: No
• Primary sponsor: FRAXA Research Foundation
• Public title: Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome
• Scientific title: Add-on Pilot Trial of Minocycline in Fragile X Syndrome
• Date of first enrolment: October 2007
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00858689
• Study type: Interventional
• Phase: N/A

Countries of recruitment

Canada

Contacts

• Name: Carlo Paribello, M.D.
• Affiliation: Fragile X Research Foundation of Canada

Key inclusion & exclusion 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.



Age minimum: 13 Years
Age maximum: 35 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fragile X Syndrome

Intervention(s)

Drug: Minocycline

Primary Outcome(s)

ABC Irritability Subtest Score [Time Frame: 8 weeks]

ABC Irritability Subtest Score [Time Frame: 1 year]

Change From Baseline of ABC Irritability Subtest Score at 8 Weeks [Time Frame: Baseline and 8 weeks]

Secondary Outcome(s)

Non-Verbal Associative Learning Task (NVALT) [Time Frame: 1 year]

Parent Defined Target Symptoms Scale-Visual [Time Frame: 8 weeks]

Parent Defined Target Symptoms Scale-Visual [Time Frame: Baseline]

The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) [Time Frame: 8 weeks]

The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) [Time Frame: Baseline]

Vineland Adaptive Behaviour Scales (VABS) [Time Frame: 1 year]

Stanford Binet 5 (SB5) [Time Frame: 1 year]

The Peabody Picture Vocabulary Test Third Edition (PPVT-III) [Time Frame: 8 weeks]

Non-Verbal Associative Learning Task (NVALT) [Time Frame: 8 weeks]

Non-Verbal Associative Learning Task (NVALT) [Time Frame: Baseline]

Parent Defined Target Symptoms Scale-Visual [Time Frame: 1 year]

The Peabody Picture Vocabulary Test Third Edition (PPVT-III) [Time Frame: 1 year]

Vineland Adaptive Behaviour Scales (VABS) [Time Frame: 8 weeks]

Vineland Adaptive Behaviour Scales (VABS) [Time Frame: Baseline]

Clinical Global Impression Scale [Time Frame: 1 year]

Stanford Binet 5 (SB5) [Time Frame: Baseline]

The Peabody Picture Vocabulary Test Third Edition (PPVT-III) [Time Frame: Baseline]

Clinical Global Impression Scale [Time Frame: 8 weeks]

Clinical Global Impression Scale [Time Frame: Baseline]

The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) [Time Frame: 1 year]

Secondary ID(s)

010308

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Fragile X Research Foundation of Canada

Results

• Results available: Yes
• Date Posted: 18/02/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00858689