Trial of Minocycline to Treat Children With Fragile X Syndrome

NCT ID: NCT01053156

Last Updated: 2017-05-30

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 66 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-01-31
• Study Completion Date: 2011-12-31

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.

Conditions

Fragile X Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Placebo pill

All patients will be on placebo for 3 months in this crossover study.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo will be given daily for 3 months.

Minocycline

All patients will be on minocycline for 3 months in this crossover trial.

Group Type: EXPERIMENTAL

minocycline hydrochloride

Intervention Type: DRUG

Minocycline hydrochloride dosed orally once a day for 3 months.

Interventions

minocycline hydrochloride

Minocycline hydrochloride dosed orally once a day for 3 months.

Intervention Type: DRUG

Placebo

Placebo will be given daily for 3 months.

Intervention Type: DRUG

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

• Minimum Eligible Age: 42 Months
• Maximum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The National Fragile X Foundation

OTHER

Sponsor Role: collaborator

University of California, Davis

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Randi J Hagerman, MD

Role: PRINCIPAL_INVESTIGATOR

M.I.N.D. Institute at University of California at Davis, Sacramento CA

Locations

M.I.N.D. Institute at University of California at Davis Medical Center

Sacramento, California, United States

Countries

United States

References

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.

Reference Type: BACKGROUND

PMID: 18835858 (View on PubMed)

Other Identifiers

200917522

Identifier Type: -

Identifier Source: org_study_id