Ignite Creation Date:
2024-05-05 @ 11:41 AM
Last Modification Date:
2024-10-26 @ 9:11 AM
Study NCT ID:
NCT00102453
Status:
COMPLETED
Last Update Posted:
2011-10-27
First Post:
2005-01-29
Brief Title:
Pentoxifylline in Duchenne Muscular Dystrophy
Sponsor:
Cooperative International Neuromuscular Research Group
Organization:
Cooperative International Neuromuscular Research Group
Study Overview
Official Title:
An Open-Label Pilot Study of Pentoxifylline in Steroid-naive Duchenne Muscular Dystrophy
Status:
COMPLETED
Status Verified Date:
2011-10
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:
None
Brief Summary:
In this study the primary aim will be to estimate the magnitude and variability of strength change over time that may be expected for subjects on the study treatment This estimate of effect will allow us to develop a rigorous statistical plan in the future randomized study The specific estimation technique to be applied will use a linear random effects model to estimate average strength change during the 3-month lead-in period and then during the twelve-month treatment period taking into account the quantitative muscle testing QMT measures for each subject Accounting for the correlation between repeated measures from each subject by using a random effects model will yield an unbiased estimate of variability for the population average change in strength We will use an analysis of pre- and post-treatment data to inform a best estimate of treatment effect For example the difference in QMT trends pre- and post-treatment would provide a straightforward measure of efficacy
Detailed Description:
Duchenne muscular dystrophy DMD is a progressive disease of skeletal muscle caused by the absence of dystrophin due to a genetic mutation in the x-linked dystrophin gene The absence of dystrophin results in a fragile muscle membrane that permits an abnormal permeability to electrolytes especially Ca The increase in intracellular calcium triggers a pathological cascade of events that ultimately results in muscle necrosis and fibrosis which impedes normal muscle regeneration The increased knowledge of the pathophysiology of DMD opens the opportunity for pharmacological treatment with the purpose of altering the disease process and or reverting the muscle degeneration
This research study requires having Duchenne muscular dystrophy DMD and the subject to be between 4 and 7 years old We expect 5 children to take part in this study at Childrens Hospital and 10 other children to participate at other hospitals worldwide
There will be two 2 screening visits to help decide whether you will be able to participate in the study At the second screening visit there will be a blood test about 13 tablespoons of blood and an EKG Once the study doctors decide eligibility to be in the study the subject will then come back once a month for three months to have his strength tested After three months the subject will begin to take the pentoxifylline and have an MRI you will have a test called an MRI to look inside the muscles of your legs This will continue for 12 months
This research study requires having Duchenne muscular dystrophy DMD and the subject to be between 4 and 7 years old We expect 5 children to take part in this study at Childrens Hospital and 10 other children to participate at other hospitals worldwide
There will be two 2 screening visits to help decide whether you will be able to participate in the study At the second screening visit there will be a blood test about 13 tablespoons of blood and an EKG Once the study doctors decide eligibility to be in the study the subject will then come back once a month for three months to have his strength tested After three months the subject will begin to take the pentoxifylline and have an MRI you will have a test called an MRI to look inside the muscles of your legs This will continue for 12 months
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