Ignite Creation Date:
2024-05-06 @ 7:25 AM
Last Modification Date:
2024-10-26 @ 11:48 AM
Study NCT ID:
NCT02530996
Status:
COMPLETED
Last Update Posted:
2021-04-05
First Post:
2015-07-29
Brief Title:
Systemic Sclerosis SSc Vasculopathy Improved Clinical Monitoring and Treatment
Sponsor:
VA Office of Research and Development
Organization:
VA Office of Research and Development
Study Overview
Official Title:
Systemic Sclerosis SSc Vasculopathy Improved Clinical Monitoring and Treatment
Status:
COMPLETED
Status Verified Date:
2021-04
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:
Scleroderma
Brief Summary:
Systemic sclerosis SSc scleroderma is a multi-organ systemic disease characterized by activation of immune cells which results in vascular dysfunction vasculopathy and subsequent scarring fibrosis SSc has a higher than expect prevalence in the US military On a national level there are 5766 SSc patients ICD-9 7101 presently cared for in the Veterans Health Administration VHA While there is no cure for SSc studies of therapeutics that can help slow disease progression are valuable to our Veterans This proposal addresses the solicitation for projects with attention to SSc requested by President Obama after reviewing potential contamination of water at Camp Lejeune This proposal is a patient-centered outreach for our Veterans with SSc to inform and prevent catastrophic endstage vascular abnormalities including digital ulcers pulmonary arterial hypertension PAH and scleroderma renal crisis in SSc The study proposes a novel application of a therapeutic for this disease A better understanding of the initiating insult and natural progression of SSc vasculopathy is needed in order to develop therapeutics with a goal of curingtreating the underlying disease This project has the potential to impact not only Veterans with SSc but also those with vascular abnormalities including digital ulcers PAH and renal crisis This proposal represents a potential major therapeutic advance for our Veterans with SSc
Detailed Description:
Although SSc is heterogeneous in the extent of organ involvement and prognosis it is accepted that all SSc cases have a progressive and usually devastating course Since vasculopathy precedes fibrosis in this disease a focus on understanding its natural history and preventative measures for vascular dysfunction has profound implications This pilot work suggests that measurement of endothelial dysfunction with flow mediated dilatation FMD holds promise as novel method to assess disease progression as well as the therapeutic efficacy of the pharmacologic compound tetrahydrobiopterin BH4 in SSc The investigators believe that BH4 which targets the endothelium has great promise to reduce SSc-related tissue hypoxia end organ damage and potentially may impact underlying disease progression The first aim will adopt an integrative approach and validate a novel non-invasive technique FMD to define vasculopathy in SSc The second aim and third aim which is reported in this PRS report will examine if BH4 is effective in ameliorating vascular dysfunction in patients with SSc and determine the role of oxidative stress in BH4-mediated improvements in vascular function in patients with SSc The overarching goal of these aims is to improve vasculopathy detection and management in Veterans with SSc
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None