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Scleroderma
Scleroderma is a group of autoimmune diseases that may result in changes to the skin, blood vessels, muscles, and internal organs
Prevalence
Age of Onset
ICD-10
M34
Inheritance
This condition does not appear to have a clear pattern of inheritance.
Rare View
Scleroderma is an aspect of systemic sclerosis, a systemic connective tissue disease that involves subcutaneous tissue, muscles, and internal organs. The disease can be either localized to the skin or involve other organs, as well. Symptoms may include areas of thickened skin, stiffness, feeling tired, and poor blood flow to the fingers or toes with cold exposure. Systemic sclerosis is a complex and heterogeneous disease with clinical forms ranging from limited skin involvement (limited cutaneous systemic sclerosis) to forms with diffuse skin sclerosis and severe and often progressive internal organ involvement (diffuse cutaneous systemic sclerosis), and occasionally a fulminant course (fulminant systemic sclerosis). The exact etiology of systemic sclerosis is not known. Systemic sclerosis is not inherited, although a genetic predisposition plays an important role in its development.†
5 Facts you should know
FACT
Scleroderma is an autoimmune disorder that may involve changes in the skin, blood vessels, muscles, and internal organs
FACT
The most commonly affected organs are the lungs and the kidneys
FACT
It is characterized by increased synthesis of collagen (leading to the sclerosis), damage to small blood vessels, activation of T lymphocytes, and production of altered connective tissue
FACT
Risk factors include family history, certain genetic factors, and exposure to silica
FACT
Women are more often affected than men – about 80% of cases
Interest over time
Google searches
Common signs & symptoms
Abnormal stomach morphology
Acrocyanosis
Persistent blue color of hands, feet, or parts of face
Arthralgia
Joint pain
Arthritis
Joint inflammation
Autoimmunity
Cheilitis
Inflammation of the lips
Chest pain
Cough
Current treatments
Corticosteroids
Immune suppressing medications (methotrexate, Cytoxan)
Nonsteroidal anti-inflammatory drugs (NSAIDs)
Drugs for heartburn or swallowing problems (proton pump inhibitors)
Prokinetic agents to speeding the movement of food through the stomach and intestines
Antibiotics to address malabsorption syndrome
Blood pressure medications (particularly ACE inhibitors) for high blood pressure or kidney problems
Top Clinical Trials
Title | Description | Phases | Status | Interventions | More Information |
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Belimumab and Rituximab Combination Therapy for the Treatment of Diffuse Cutaneous Systemic Sclerosis | This is a 52 week, single center, randomized, double-blind, placebo-controlled study. After patients maintain a stable dose of Mycophenolate Mofetil (MMF) for at least 1 month, they will be randomized to treatment with either Belimumab & Rituximab or placebo.Patients in both groups will be on background MMF for the entirety of the study. Belimumab will be administered subcutaneously and Rituximab intravenously. Placebo injections and infusions will be of normal saline. Randomization will be done in a 2:1 manner to favor the treatment group. It is hypothesized that that Rituximab and Belimumab combination therapy with Mycophenolate Mofetil background therapy will improve fibrosis in SSc skin when compared to treatment with placebo and Mycophenolate Mofetil in a group of patients with early dcSSc. | Phase 2 | Recruiting | Drug: Belimumab|Drug: Rituximab|Other: Placebo Subcutaneous Injection|Other: Placebo Infusion|Drug: MMF | More Info |
Sildenafil for Early Pulmonary Vascular Disease in Scleroderma | This is a Phase II randomized, double-blind, placebo-controlled trial of sildenafil in men and women with Scleroderma with mildly elevated pulmonary pressures (SSc-MEP) to determine whether sildenafil may be an effective treatment for SSc-MEP. | Phase 2 | Recruiting | Drug: Sildenafil|Other: Placebo | More Info |
A Study to Evaluate the Safety and Tolerability of Oral Ixazomib in Scleroderma-related Lung Disease Patients | The purpose of this research study is to learn about the effects of the medication ixazomib in participants with scleroderma/systemic sclerosis including its safety and tolerability, its effects on skin, lungs and other organs, and its effects on overall health and quality of life. | Phase 2 | Recruiting | Drug: Ixazomib | More Info |
Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis | The purpose of this study is to determine whether a regimen of high-dose immunoablative therapy will demonstrate safety that is consistent or improved with other published regimens in SSc patients, while maintaining a treatment effect. | Phase 2 | Recruiting | Drug: Cyclophosphamide|Drug: Mesna|Drug: Rituximab|Drug: Alemtuzumab|Drug: Thiotepa|Drug: GM-CSF|Drug: Intravenous immunoglobulin|Radiation: Total Body Irradiation|Drug: Anti Thymocyte Globulin | More Info |
Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases | A subset of autoimmune diseases (ADs) in children and young adults are life-threatening and unresponsive to conventional treatments. In these patients, the delivery of high dose immunosuppressive therapy followed by autologous stem cell transplant (ASCT) offers a treatment strategy capable of purging the pathogenic, autoreactive immune system and an opportunity for "immune reset." This strategy has been used in adults across a myriad of indications with evidence for efficacy. This study proposes a pilot study to evaluate this therapeutic strategy in children and young adults with systemic sclerosis (SSc) and systemic lupus erythematosis (SLE), two potentially life threatening autoimmune diseases that may response to this therapeutic approach. | Phase 2 | Recruiting | Biological: Depletion of CD3/CD19 in an autologous stem cell transplant | More Info |
Efficacy and Safety of Belumosudil in Subjects With Diffuse Cutaneous Systemic Sclerosis | This is a phase 2, open-label, single-cohort, multicenter trial of belumosudil in subjects with Diffuse Cutaneous Systemic Sclerosis (dcSSc). An estimated total of 12 to 15 subjects will receive belumosudil 200 mg administered orally (PO) twice daily (BID) for 52 weeks. The primary analysis will be at 24 weeks. | Phase 2 | Recruiting | Drug: Belumosudil | More Info |
A Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients With Diffuse Cutaneous Systemic Sclerosis | This is a randomized, double-blind, placebo-controlled, repeat-dose, multicenter trial. Participants will be screened within 4 weeks prior to the Baseline (Day 1) Visit. Approximately 300 participants who meet the trial eligibility criteria will be randomized on Day 1 in a 1:1:1 ratio to receive HZN-825 300 mg QD, HZN-825 300 mg BID or placebo for 52 weeks. | Phase 2 | Recruiting | Drug: HZN-825 BID|Drug: Placebo|Drug: HZN-825 QD | More Info |
Evaluation of Safety, Tolerability and Preliminary Efficacy of EHP-101 in Diffuse Cutaneous Systemic Sclerosis | The purpose of this trial is to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of EHP-101 in adult subjects with diffuse cutaneous Systemic Sclerosis (dcSSc). | Phase 2 | Recruiting | Drug: Patients will be randomized to receive EHP-101 or Placebo | More Info |
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Diffuse Cutaneous Systemic Sclerosis | To evaluate the efficacy of MT-7117 treatment in subjects with diffuse cutaneous systemic sclerosis (dcSSc) using the American College of Rheumatology Composite Response Index in Diffuse Systemic Sclerosis (ACR CRISS) at Week 52 | Phase 2 | Recruiting | Drug: MT-7117|Drug: Placebo | More Info |
KD025 in Subjects With Diffuse Cutaneous Systemic Sclerosis | This randomized, placebo-controlled phase 2 study is seeking to evaluate the efficacy and safety of belumosudil (KD025) for the treatment of diffuse cutaneous systematic sclerosis. Upon eligibility confirmation, a total of 60 adult subjects will be enrolled and randomized into 3 groups (1:1:1) to either receive orally administered belumosudil (200 mg once daily and 200 mg twice daily) or matched placebo for 28 weeks. Study drug dosing will be for 52 weeks: double-blinded for the first 28 weeks followed by an open-label extension of 24 weeks. After unblinding, the subjects on belumosudil will continue on the same belumosudil dose whereas the subjects in the placebo group will be re-randomized to one of the belumosudil doses. | Phase 2 | Recruiting | Drug: Belumosudil (KD025)|Drug: Placebo | More Info |
Oral Ifetroban to Treat Diffuse Cutaneous Systemic Sclerosis (SSc) or SSc-associated Pulmonary Arterial Hypertension | The purpose of this phase 2 multicenter, randomized, double-blind, placebo-controlled, study is to assess the safety and efficacy of ifetroban in patients with diffuse cutaneous systemic SSc (dcSSc) or SSc-associated pulmonary arterial hypertension (SSc-PAH). | Phase 2 | Recruiting | Drug: Oral Ifetroban|Drug: Oral Placebo | More Info |
Top Treatments in Research
Agent | Class/Mechanism of Action | Development Status | Company | Clinical Studies | More Information |
---|---|---|---|---|---|
FCX-013 (Gene Therapy) | Dermal fibroblasts are collected from the patient, cultured and genetically modified to regulate collagen accumulation and then injected at the site of the localized disease. | Phase 1|Phase 2 | Castle Creek Biosciences, LLC. | More Info | More Info |
ACE-1334 | ACE-1334 is designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2. TGF-beta 1 and 3 are believed to be key signaling factors in the pathogenesis of fibrotic disease. ACE-1334 has shown robust anti-fibrotic activity in multiple preclinical models of fibrosis. | Phase 1|Phase 2 | Acceleron Pharma Inc. | More Info | More Info |
Temanogrel | Temanogrel is a peripherally acting, and selective 5-HT2A receptor inverse agonist designed to inhibit serotonin (5-HT)-mediated amplification of platelet aggregation and vasoconstriction. Temanogrel is in Phase 2 for microvascular obstruction (MVO) and Raynaud's phenomenon secondary to systemic sclerosis (SSc-RP). | Phase 2 | Arena Pharmaceuticals | More Info | More Info |
Belumosudil | Belumosudil, is an orally administered, selective inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2). Belumosudil helps to resolve immune dysregulation by down-regulating pro-inflammatory Th17 cells and increasing regulatory T (Treg) cells. | Phase 2 | Kadmon Corporation, LLC | More Info | More Info |
† Medline Plus, National Library of Medicine