A randomised placebo-controlled study of lovastatin in children with neurofibromatosis type 1

Phase 2

Completed

• Conditions: eurofibromatosis type 1; Neuropsychological impairment; Neurofibromatosis type 1; Human Genetics and Inherited Disorders - Other human genetics and inherited disorders

• Registration Number: ACTRN12607000560493
• Lead Sponsor: Prof Kathryn North

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2007-11-01
• Last Update Posted: 13 January 2020

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 142

Inclusion Criteria

1. Males or females aged between 8-15 years of age who meet National Institutes of Health (NIH) diagnostic criteria for neurofibromatosis type 1 (NF1). 2. Full scale intellectual quotient (IQ) of 70 or above. 3. Cognitive impairment defined as having a score of at least 1 standard deviation or more below the normative mean on one or more of the primary outcome measures.

Exclusion Criteria

1. Full scale IQ score less than 70.
2. Individuals with insufficient English to complete the assessments.
3. Participants taking stimulant medication or Straterra, as it is unclear whether lovastatin and ADHD medication utilise similar biological pathways, possibly leading to an interaction between the two medications
4. Participants on psychotropic or antiepileptic medication
5. Participants with intracranial pathology such as epilepsy, diagnosed head injury, hydrocephalus or progressive intracranial tumors (children with asymptomatic or static lesions will be eligible).
6. Participants who are pregnant or breastfeeding.
7. Participants with a clinically significant unrelated illness, which in the judgement of the principle or associate investigator, would compromise the participant's ability to tolerate the medication or potentially interfere with the participant's ability to participate in the required testing.
8. Children with very low LDL cholesterol levels pre-treatment (1 mmol/L). LDL levels within the normal range are required pre-treatment as lovastatin has been shown to lower levels of LDL cholesterol by approximately 30%.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Visuospatial learning: Mean Total Errors Adjusted score on the Paired Associate Learning subtest from the Cambridge Neuropsychological Test Automated Battery (CANTAB).[Baseline, post-treatment (16 weeks after commencement of intervention) and 8 weeks after cessation of intervention.];Sustained attention: Mean score on Score! subtest of the Test of Everyday Attention for Children (TEA-Ch).[Baseline, post-treatment (16 weeks after commencement of intervention) and 8 weeks after cessation of intervention.]