A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1

Summary

The specific aim of this study is to determine whether Lovastatin ™ significantly improves visual spatial learning and/or sustained attention in children with NF1.

Secondary Aims:

To evaluate the effect of Lovastatin ™ on measures of executive function, behavior and quality of life in children with NF1 and cognitive deficits.

To further evaluate the toxicity and tolerability of Lovastatin ™ in children with NF1 and cognitive deficits.

Hypotheses

It is hypothesized that Lovastatin ™ will improve the visual spatial memory and/or attention deficits in children with NF1. This is based on studies demonstrating that Lovastatin ™ has significantly improved impairments in visual spatial memory and attention in the NF1 murine model.

It is further expected that Lovastatin ™ will be safe and well tolerated over a 16-week period.

Conditions

• Neurofibromatosis Type 1

Interventions

DRUG

Lovastatin ™

Lovastatin starting at 20mg for 2 weeks, increasing to 40mg for 14 weeks. Total duration of trial is 16 weeks.

DEVICE

placebo

Starting at 20mg for 2 weeks, then increasing to 40mg for 14 additional weeks for a total duration of treatment of 16 weeks.

Sponsors & Collaborators

• Boston Children's Hospital

  collaborator OTHER

• Children's Hospital of Philadelphia

  collaborator OTHER

• Children's National Research Institute

  collaborator OTHER

• Children's Hospital Medical Center, Cincinnati

  collaborator OTHER

• National Cancer Institute (NCI)

  collaborator NIH

• University of Chicago

  collaborator OTHER

• University of Utah

  collaborator OTHER

• Washington University School of Medicine

  collaborator OTHER

• Sydney Children's Hospitals Network

  collaborator OTHER

• University of Texas Southwestern Medical Center

  collaborator OTHER

• University of Alabama at Birmingham

  lead OTHER

Principal Investigators

• Kathryn North, MD · University of Sydney - Westmead

• Maria Acosta, MD · Children's National Research Institute

• Jonathan Payne, MD · University of Sydney - Westmead

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

8 Years

Max Age

15 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2009-07-31

Primary Completion

2014-05-31

Completion

2016-12-31

Countries

• United States
• Australia

Study Locations