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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT00853580 |
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Date of registration:
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23/02/2009 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
STARS |
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Scientific title:
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A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1 |
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Date of first enrolment:
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July 2009 |
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Target sample size:
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146 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00853580 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 2
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Countries of recruitment
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Australia
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United States
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Contacts
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Name:
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Maria Acosta, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Children's National Research Institute |
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Name:
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Kathryn North, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Sydney - Westmead |
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Name:
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Jonathan Payne, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Sydney - Westmead |
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Key inclusion & exclusion criteria
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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.
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.
Age minimum:
8 Years
Age maximum:
15 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Neurofibromatosis Type 1
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Intervention(s)
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Device: placebo
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Drug: Lovastatin ™
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Primary Outcome(s)
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Score! (Test of Everyday Attention for Children)
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Paired Associate Learning (Cambridge Neuropsychological Test Automated Battery).
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Secondary Outcome(s)
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Stockings of Cambridge (Cambridge Neuropsychological Test Automated Battery) Automated Battery).
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Stop Signal Task (Cambridge Neuropsychological Test Automated Battery)
[Time Frame: Baseline and Post-treatment (16 weeks)]
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ADHD Inattentive Scale, Conners ADHD Scales
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Object Assembly (WISC-III)
[Time Frame: Baseline and Post-treatment (week 16)]
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Behavior Rating Inventory of Executive Function Global Executive Composite
[Time Frame: Baseline and Post-treatment (week 16)]
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Judgement of Line Orientation Test
[Time Frame: Baseline and Post-treatment (week 16)]
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Commission Errors (Conners Continuous Performance Test, Second Edition; CPT-II)
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Omission Errors (Conners Continuous Performance Test, Second Edition; CPT-II)
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Controlled Oral Word Association Test
[Time Frame: Baseline and Post-treatment (week 16)]
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Internalizing Behaviors, Behavior Assessment System for Children Second Edition
[Time Frame: Baseline and Post-treatment (week 16)]
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Sky Search DT (Test of Everyday Attention for Children)
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Spatial Working Memory (Cambridge Neuropsychological Test Automated Battery)
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Creature Counting (Test of Everyday Attention for Children)
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Psychosocial Quality of Life PedsQL
[Time Frame: Baseline and Post-treatment (week 16)]
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ADHD Hyperactive/Impulsive Scale, Conners ADHD Scales
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Sky Search (Test of Everyday Attention for Children)
[Time Frame: Baseline and Post-treatment (16 weeks)]
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Quality of Life Pediatric Quality of Life Inventory (PedsQL)
[Time Frame: Baseline and Post-treatment (week 16)]
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Secondary ID(s)
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DOD: W81XWH-05-1 0615
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X080929007
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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