Phase 2
N=12
Systemic Sclerosis (SSc) Vasculopathy: Improved Clinical Monitoring and Treatment
Rheumatologic Disease
Bottom Line
View on ClinicalTrials.gov: NCT02530996 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Apr 2021
Primary outcome: Primary: Flow Mediated Dilatation (FMD)-Diameter of Artery — 3.79; 3.78 mm
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- BH4 (Drug); Vasculopathy assessment (Diagnostic_test); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- VA Office of Research and Development
- Primary completion
- Jun 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Flow Mediated Dilatation (FMD)-Diameter of Artery |
3.79; 3.78 | — |
| PRIMARY Flow Mediated Dilatation-shear Rate |
95; 100 | <0.05 sig |
| PRIMARY Flow Mediated Dilatation- Blood Velocity |
4.4; 4.5 | — |
| PRIMARY Flow Mediated Dilatation-blood Flow |
32; 30 | — |
| SECONDARY Oxidative Stress Measurement-MDA: Malondialdehyde |
2.8; 2.9 | — |
| SECONDARY Oxidative Stress Measurement-catalase (CAT) |
86; 78 | — |
| SECONDARY Oxidative Stress Measurement- Protein Carbonyl |
0.17; 0.16 | — |
| SECONDARY Oxidative Stress Measurement- Ferric Reducing Ability of Plasma (FRAP) |
1.8; 1.8 | — |
| SECONDARY Oxidative Stress Measurement- Superoxide Dismutase (SOD) |
10.7; 11.1 | — |
| SECONDARY Oxidative Stress Measurement- Interleukin 6 (IL-6) |
1.3; 1.4 | — |
| SECONDARY Oxidative Stress Measurement- Tumor Necrosis Factor Alpha (TNF-α, |
1.1; 1.3 | — |
| SECONDARY Oxidative Stress Measurement- C-reactive Protein (CRP) |
3.2; 3.1 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of systemic sclerosis (SSc, scleroderma) by ACR/EULAR 2013 criteria.
Exclusion Criteria
- Age < 18
- Pregnant or breast feeding
- Unwillingness to consent
Data sourced from ClinicalTrials.gov (NCT02530996). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.