Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02642653
• Date of registration: 21/12/2015
• Prospective Registration: Yes
• Primary sponsor: University of California, Davis
• Public title: Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
• Scientific title: Combining Lovastatin and a Parent-Implemented Language Intervention in a Multimodal Treatment for Fragile X Syndrome
• Date of first enrolment: January 2016
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02642653
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 4

Countries of recruitment

United States

Contacts

• Name: Randi J Hagerman, MD
• Affiliation: UC Davis

Key inclusion & exclusion 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 ß-human
chorionic growth hormone (ß-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.



Age minimum: 10 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Genetic Diseases

Fragile X Syndrome

Intervention(s)

Drug: Lovastatin

Other: Placebo

Primary Outcome(s)

Expressive Language Sample Composite Score in the Home [Time Frame: 20 weeks]

Expressive Language Sample Composite Score in the Home [Time Frame: Baseline]

Secondary Outcome(s)

Visual Analog Scale (VAS) - Spoken Language Impairment [Time Frame: 20 weeks]

Clinical Global Impression-Improvement (CGI-I) Scale [Time Frame: 20 weeks]

FXS- Normed Aberrant Behavior Checklist (ABC) Social Avoidance Subscale [Time Frame: 10 weeks]

Clinical Global Impression- Severity (CGI-S) [Time Frame: Baseline]

FXS- Normed Aberrant Behavior Checklist (ABC) Social Avoidance Subscale [Time Frame: 20 weeks]

Visual Analog Scale (VAS) - Social Impairment [Time Frame: Baseline]

Visual Analog Scale (VAS) - Social Impairment [Time Frame: 20-weeks]

Visual Analog Scale (VAS) - Spoken Language Impairment [Time Frame: 10 weeks]

Clinical Global Impression-Improvement (CGI-I) Scale [Time Frame: 10 weeks]

Visual Analog Scale (VAS) - Social Impairment [Time Frame: 10-weeks]

Visual Analog Scale (VAS) - Spoken Language Impairment [Time Frame: Baseline]

FXS- Normed Aberrant Behavior Checklist (ABC) Social Avoidance Subscale [Time Frame: Baseline]

Secondary ID(s)

520144

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 11/09/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02642653