Clinical Trials Logo

Clinical Trial 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 5,766 SSc patients (ICD-9 710.1) 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 curing/treating 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.


Clinical Trial 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 Design


Related Conditions & MeSH terms


NCT number NCT02530996
Study type Interventional
Source VA Office of Research and Development
Contact
Status Completed
Phase Phase 1/Phase 2
Start date January 1, 2016
Completion date December 31, 2019

See also
  Status Clinical Trial Phase
Recruiting NCT05719389 - EFFICACY AND SAFETY OF S.C. INFLIXIMAB IN PATIENTS SWITCHED FROM I.V. FORMULATION OF INFLIXIMAB
Active, not recruiting NCT05846672 - Turkish Cultural Adaptation, Validity and Reliability of the "Gait Outcomes Assessment List"
Recruiting NCT06339957 - Rheumatology Diet Study
Not yet recruiting NCT06461000 - EULAR Impact of Rheumatic and Musculoskeletal Diseases Survey
Recruiting NCT05621369 - Psorcast Mobile Study
Recruiting NCT06227468 - A Clinical Study to Map the HLA Genomic Region in the Greek Population
Completed NCT05563194 - Proof-of-concept Study of 'JIA Toolbox' for Children and Young People (CYP) With Juvenile Idiopathic Arthritis (JIA) N/A
Completed NCT05015114 - Prevalence and Factors of Sarcopenia and in Patients With Primary Sjogren's Syndrome