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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00853580




Registration number
NCT00853580
Ethics application status
Date submitted
23/02/2009
Date registered
2/03/2009
Date last updated
12/03/2018

Titles & IDs
Public title
A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
Scientific title
A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
Secondary ID [1] 0 0
DOD: W81XWH-05-1 0615
Secondary ID [2] 0 0
X080929007
Universal Trial Number (UTN)
Trial acronym
STARS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neurofibromatosis Type 1 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Skin 0 0 0 0
Dermatological conditions
Cancer 0 0 0 0
Other cancer types

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Lovastatin ™
Treatment: Devices - placebo

Placebo comparator: 2 - This is a prospective multi-centre randomized, placebo-controlled Phase II study to determine the efficacy of Lovastatin ™ on visual spatial learning and/or attention abilities of children with NF1 aged between 8 and less than 16 years. In addition, the effect of Lovastatin ™ on secondary measures of executive function, visual spatial skills, behavior and quality of life will be assessed. Participants will be randomized to 16-weeks of treatment with Lovastatin ™ or a matched placebo.

Experimental: 1 - This is a prospective multi-centre randomized, placebo-controlled Phase II study to determine the efficacy of Lovastatin ™ on visual spatial learning and/or attention abilities of children with NF1 aged between 8 and less than 16 years. In addition, the effect of Lovastatin ™ on secondary measures of executive function, visual spatial skills, behavior and quality of life will be assessed. Participants will be randomized to 16-weeks of treatment with Lovastatin ™ or a matched placebo.


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

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

Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Treatment: Devices
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Paired Associate Learning (Cambridge Neuropsychological Test Automated Battery).
Timepoint [1] 0 0
Baseline and Post-treatment (16 weeks)
Primary outcome [2] 0 0
Score! (Test of Everyday Attention for Children)
Timepoint [2] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [1] 0 0
Spatial Working Memory (Cambridge Neuropsychological Test Automated Battery)
Timepoint [1] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [2] 0 0
Stockings of Cambridge (Cambridge Neuropsychological Test Automated Battery) Automated Battery).
Timepoint [2] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [3] 0 0
Stop Signal Task (Cambridge Neuropsychological Test Automated Battery)
Timepoint [3] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [4] 0 0
Sky Search (Test of Everyday Attention for Children)
Timepoint [4] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [5] 0 0
Sky Search DT (Test of Everyday Attention for Children)
Timepoint [5] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [6] 0 0
Creature Counting (Test of Everyday Attention for Children)
Timepoint [6] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [7] 0 0
Commission Errors (Conners Continuous Performance Test, Second Edition; CPT-II)
Timepoint [7] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [8] 0 0
Omission Errors (Conners Continuous Performance Test, Second Edition; CPT-II)
Timepoint [8] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [9] 0 0
ADHD Inattentive Scale, Conners ADHD Scales
Timepoint [9] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [10] 0 0
ADHD Hyperactive/Impulsive Scale, Conners ADHD Scales
Timepoint [10] 0 0
Baseline and Post-treatment (16 weeks)
Secondary outcome [11] 0 0
Controlled Oral Word Association Test
Timepoint [11] 0 0
Baseline and Post-treatment (week 16)
Secondary outcome [12] 0 0
Judgement of Line Orientation Test
Timepoint [12] 0 0
Baseline and Post-treatment (week 16)
Secondary outcome [13] 0 0
Behavior Rating Inventory of Executive Function Global Executive Composite
Timepoint [13] 0 0
Baseline and Post-treatment (week 16)
Secondary outcome [14] 0 0
Object Assembly (WISC-III)
Timepoint [14] 0 0
Baseline and Post-treatment (week 16)
Secondary outcome [15] 0 0
Internalizing Behaviors, Behavior Assessment System for Children Second Edition
Timepoint [15] 0 0
Baseline and Post-treatment (week 16)
Secondary outcome [16] 0 0
Internalizing Behaviors, Behavior Assessment System for Children Second Edition
Timepoint [16] 0 0
Baseline and Post-treatment (week 16)
Secondary outcome [17] 0 0
Quality of Life Pediatric Quality of Life Inventory (PedsQL)
Timepoint [17] 0 0
Baseline and Post-treatment (week 16)
Secondary outcome [18] 0 0
Psychosocial Quality of Life PedsQL
Timepoint [18] 0 0
Baseline and Post-treatment (week 16)

Eligibility
Key inclusion criteria
* Males or females aged between 8 years and 15 years 11 months at time of enrollment who meet NIH diagnostic criteria for NF1 (Appendix 1)
* Participants must have a full-scale IQ of 70 or above. In cases where there is a statistically significant difference between verbal IQ and performance IQ (.05 level as determined by Table B3 in the WASI manual), participants will be eligible if at least one of these quotients is 70 or above
* Participants must have a cognitive impairment defined as having a score of at least one standard deviation or more below the population mean on one or more of the primary objective outcome measures (i.e., impaired on a measure of visual spatial learning and/or sustained attention)
* Participants must be medically stable
* Participants who are on a stable dose of methylphenidate and/or dextroamphetamines for at least one month prior to screening and who will remain on the same dose for the duration of the study.
* Hepatic function: Participants must have a bilirubin within normal limits and AST and ALT ± 2 times the upper limit of normal as determined by the standards at their institution
* Renal function: Participants must have an age-adjusted normal serum creatinine or a creatinine clearance of greater than 70 ml/m/1.73m2
* Hematologic function: Participants must have an absolute neutrophil count of greater than 1,500, a hemoglobin of greater than 9 gms/dl, and a platelet count of greater than 100,000 on study entry
* Participants must sign all required documents, including informed assent and HIPAA documents
* Female participants of childbearing age should not be pregnant, must have a negative pregnancy test before initiation of treatment, and take appropriate birth control precautions to participate in this study.
Minimum age
8 Years
Maximum age
15 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
* Full-scale IQ less than 70; In cases where this is a statistically significant difference between performance IQ and verbal IQ (.05 level), patients will be excluded if both quotients fall below 70
* Individuals that are not cognitively impaired on at least one of the primary objective outcome measures
* Individuals with insufficient English to complete the assessments
* Participants taking psychotropic medication other than methylphenidate and/or dextroamphetamines. These patients are eligible if, as clinically indicated, they cease medication for at least 30 days prior to screening and remain off these medication for the duration of the study
* Participants with intracranial pathology such as epilepsy, diagnosed head injury, hydrocephalus or progressive intracranial tumors (children with asymptomatic or static lesions will be eligible)
* Participants who are pregnant or breastfeeding; Participants who have received any investigational drug, other than sirolimus, within 30 days of initiation of study
* Participants who have recently taken Lovastatin. These participants will be eligible after a washout period of at least three months.
* Participants with significant hepatic, renal or hematologic function as previously defined
* Participants with a history of neuromuscular disease, excluding hypotonias thought to be associated with NF1
* Participants with a clinically significant unrelated illness, which in the judgment of the principal 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
* Low cholesterol (lower limit of a total cholesterol of 90mg/dl)
* Participants who have recently taken sirolimus within three months of enrollment. These participants will be eligible after a washout period of at least three months.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
United States of America
State/province [11] 0 0
Utah

Funding & Sponsors
Primary sponsor type
Other
Name
University of Alabama at Birmingham
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Boston Children's Hospital
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Children's Hospital of Philadelphia
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Children's National Research Institute
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
Children's Hospital Medical Center, Cincinnati
Address [4] 0 0
Country [4] 0 0
Other collaborator category [5] 0 0
Government body
Name [5] 0 0
National Cancer Institute (NCI)
Address [5] 0 0
Country [5] 0 0
Other collaborator category [6] 0 0
Other
Name [6] 0 0
University of Chicago
Address [6] 0 0
Country [6] 0 0
Other collaborator category [7] 0 0
Other
Name [7] 0 0
University of Utah
Address [7] 0 0
Country [7] 0 0
Other collaborator category [8] 0 0
Other
Name [8] 0 0
Washington University School of Medicine
Address [8] 0 0
Country [8] 0 0
Other collaborator category [9] 0 0
Other
Name [9] 0 0
Sydney Children's Hospitals Network
Address [9] 0 0
Country [9] 0 0
Other collaborator category [10] 0 0
Other
Name [10] 0 0
University of Texas Southwestern Medical Center
Address [10] 0 0
Country [10] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Kathryn North, MD
Address 0 0
University of Sydney - Westmead
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.