Ignite Creation Date:
2024-05-05 @ 9:17 PM
Last Modification Date:
2024-10-26 @ 10:02 AM
Study NCT ID:
NCT00853580
Status:
COMPLETED
Last Update Posted:
2018-03-12
First Post:
2009-02-23
Brief Title:
A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
Sponsor:
University of Alabama at Birmingham
Organization:
University of Alabama at Birmingham
Study Overview
Official Title:
A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
Status:
COMPLETED
Status Verified Date:
2018-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
STARS
Brief Summary:
The specific aim of this study is to determine whether Lovastatin significantly improves visual spatial learning andor 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 andor 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
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 andor 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
Detailed Description:
Study Design
This is a prospective multi-centre randomized placebo-controlled Phase II study to determine the efficacy of Lovastatin on visual spatial learning andor 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 It is plausible and ethical to employ a placebo group as no standard therapy with established efficacy is being withheld There is no cross-over in this study due to a lack of data concerning the length of possible washout effects The Lovastatin dose will begin at 20 mg once dailycontinuous dosing and escalate over a two-week period to 40 mg once dailycontinuous dosing and continue at this dose for 14 weeks Participants will be carefully monitored for side effects The safety of Lovastatin will be evaluated using laboratory tests clinical signs and adverse effects which will be monitored at regular intervals over the 16-week period Primary and secondary outcome measures will be administered at baseline 16 weeks post-treatment and at follow-up 8 weeks after cessation of treatment to determine any carry-over effects The safety of Lovastatin will also be evaluated with regular monitoring of side-effects during the trial
Study Population
This is a Phase II study involving children with NF1 aged between 8 years to 15 years 11 months old at time of enrollment with evidence of cognitive impairment defined as having a score of at least one standard deviation or more below the population mean on a measure of visual spatial learning andor attention
A total of 142 participants with NF1 aged between 8 years and 15 years 11 months will be enrolled in the study The age limits were selected on the basis that Lovastatin has been shown to be safe in children aged between 8 and 17 years old In addition one of the primary outcome measures attention only has normative data for up to 15 years 11 months Therefore the maximum age limit for participants at time of enrolment is 15 years 11 months so that normative data can be used to determine whether participants are impaired The pediatric NF1 population is an ideal group in which to study the cognitive effects of Lovastatin because it represents an opportunity for early pharmacological intervention of cognitive deficits
This is a prospective multi-centre randomized placebo-controlled Phase II study to determine the efficacy of Lovastatin on visual spatial learning andor 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 It is plausible and ethical to employ a placebo group as no standard therapy with established efficacy is being withheld There is no cross-over in this study due to a lack of data concerning the length of possible washout effects The Lovastatin dose will begin at 20 mg once dailycontinuous dosing and escalate over a two-week period to 40 mg once dailycontinuous dosing and continue at this dose for 14 weeks Participants will be carefully monitored for side effects The safety of Lovastatin will be evaluated using laboratory tests clinical signs and adverse effects which will be monitored at regular intervals over the 16-week period Primary and secondary outcome measures will be administered at baseline 16 weeks post-treatment and at follow-up 8 weeks after cessation of treatment to determine any carry-over effects The safety of Lovastatin will also be evaluated with regular monitoring of side-effects during the trial
Study Population
This is a Phase II study involving children with NF1 aged between 8 years to 15 years 11 months old at time of enrollment with evidence of cognitive impairment defined as having a score of at least one standard deviation or more below the population mean on a measure of visual spatial learning andor attention
A total of 142 participants with NF1 aged between 8 years and 15 years 11 months will be enrolled in the study The age limits were selected on the basis that Lovastatin has been shown to be safe in children aged between 8 and 17 years old In addition one of the primary outcome measures attention only has normative data for up to 15 years 11 months Therefore the maximum age limit for participants at time of enrolment is 15 years 11 months so that normative data can be used to determine whether participants are impaired The pediatric NF1 population is an ideal group in which to study the cognitive effects of Lovastatin because it represents an opportunity for early pharmacological intervention of cognitive deficits
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| DOD W81XWH-05-1 0615 | OTHER | Department of Defense | None |