Phase 4
N=30
Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
Fragile X Syndrome · Genetic Diseases
Bottom Line
View on ClinicalTrials.gov: NCT02642653 ↗Enrolled (actual)
30
Serious AEs
0.0%
Results posted
Sep 2019
Primary outcome: Primary: Expressive Language Sample Composite Score in the Home — 39.63; 42.47 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Lovastatin (Drug); Placebo (Other)
- Age
- Pediatric · 10+ yrs
- Sex
- All
- Sponsor
- University of California, Davis
- Primary completion
- Jul 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Expressive Language Sample Composite Score in the Home |
59.32; 54.81 | — |
| PRIMARY Expressive Language Sample Composite Score in the Home |
59.32; 54.81 | — |
| SECONDARY FXS- Normed Aberrant Behavior Checklist (ABC) Social Avoidance Subscale |
2.42; 3.38 | — |
| SECONDARY FXS- Normed Aberrant Behavior Checklist (ABC) Social Avoidance Subscale |
2.42; 3.38 | — |
| SECONDARY FXS- Normed Aberrant Behavior Checklist (ABC) Social Avoidance Subscale |
2.42; 3.38 | — |
| SECONDARY Clinical Global Impression- Severity (CGI-S) |
4.58; 4.50 | — |
| SECONDARY Clinical Global Impression-Improvement (CGI-I) Scale |
2.42; 2.38 | — |
| SECONDARY Clinical Global Impression-Improvement (CGI-I) Scale |
2.42; 2.38 | — |
| SECONDARY Visual Analog Scale (VAS) - Spoken Language Impairment |
5.66; 4.18 | — |
| SECONDARY Visual Analog Scale (VAS) - Spoken Language Impairment |
5.66; 4.18 | — |
| SECONDARY Visual Analog Scale (VAS) - Spoken Language Impairment |
5.66; 4.18 | — |
| SECONDARY Visual Analog Scale (VAS) - Social Impairment |
5.18; 3.99 | — |
| SECONDARY Visual Analog Scale (VAS) - Social Impairment |
5.18; 3.99 | — |
| SECONDARY Visual Analog Scale (VAS) - Social Impairment |
5.18; 3.99 | — |
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.
Data sourced from ClinicalTrials.gov (NCT02642653). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.