Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
Condition: Fragile X Syndrome; Genetic Diseases
MeSH terms: Fragile X Syndrome; Genetic Diseases, Inborn; Syndrome
Intervention: Lovastatin; Placebo
Intervention description: Once per day dosing; Once per day dosing
Intervention MeSH terms: Lovastatin; L 647318; Dihydromevinolin
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 30.0
Sex: All
Minimum age: 10 Years
Maximum age: 17 Years
Healthy volunteers: No
Keywords: Fra(X) Syndrome; Trinucleotide Repeat Expansion; Fragile X Syndrome; Fragile X Mental Retardation Syndrome; Intellectual Disability; Parent Implemented Language Intervention (PILI); FXS; Mental Retardation, X-linked; Genetic Diseases, X-linked; Neurobehavioral Manifestations; Chromosome Disorders

Summary:

      The purpose of the study is to test the efficacy of a 20 week multi-modal treatment comprised
      of lovastatin or placebo, and the Parent-implemented Language Intervention (PILI) in children
      with fragile X syndrome (FXS). Children will be randomized to drug or placebo in a
      double-blind design with all participating in the PILI. The primary endpoint will be to
      measure improvements in spoken language and behavior among lovastatin-treated than placebo
      treated participants.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Documentation of a full mutation with absence or deficient FMRP levels.

          -  Males and females ages 10 through 17 years

          -  Willingness of potential study participant as well as a parent or caretaker to
             participate in the protocol.

          -  Speech is the primary means of communication with three-word or longer utterances used
             on a daily basis.

          -  Intelligence quotient (IQ) ≤70 as measured by the Leiter- R.

          -  Sexually active women of childbearing potential (WCBP) must be using a medically
             acceptable method of birth control and have a negative qualitative serum β-human
             chorionic growth hormone (β-HCG) or urine pregnancy test collected at the initial
             screening visit.

        Exclusion Criteria:

          -  Persons who do not speak English.

          -  Changes in any medications (including investigational medications) within the last
             month (4 weeks). All concomitant medications must have been on a stable course for at
             least 4 weeks prior to enrollment into the study and maintain stability throughout the
             course of the study.

          -  Changes in behavioral therapy or educational programming during the study. This does
             not include scheduled school holidays.

          -  Have any disease or condition (medical or surgical) at screening that might compromise
             the hematologic, cardiovascular, pulmonary, renal, gastrointestinal, or hepatic
             systems; or other conditions that might interfere with the absorption, distribution,
             metabolism, or excretion of the investigational product, or would place the subject at
             increased risk.

          -  Patients who, in the opinion of the investigator, are unsuitable in any other way to
             participate in this study, including being unable to comply with the requirements of
             the study or displaying clinically significant abnormalities in safety assessments at
             screening.

          -  Patients on prohibited medications

          -  History of recurrent status epilepticus.

          -  Inability to withhold grapefruit and grapefruit juice from diet during the entire
             clinical trial.

          -  Subjects unwilling to abstain from alcoholic beverages during the trial.

          -  Subjects who are actively suicidal.