Allogenic Stem Cell Transplant
AM152 (Lysophosphatidic Acid 1)
Ambrisentan for Scleroderma
Bardoxolone by Reata
BEAT Clinical Trial
Cyclophosphamide and rATG With Hematopoietic Stem Cell Support
Fludarabine/Donor Peripheral SCT
Habeo Cell Therapy (ADRC/Cytori)
Rituximab for PAH
SAR156597 for IPF (Sanofi)
Scleroderma Lung Study II
SPIN: Patient-centered Network
Stem Cell Transplants for Scleroderma
Twins and Siblings Study
Excitement is brewing in the field of scleroderma research, as every day seems to bring new advances regarding possible causes of scleroderma as well as improved treatments.
The best way to participate in clinical trials for scleroderma is to consult a listed scleroderma expert. Admittedly, they are few and far between because this is such a rare disease, but it is well worth the extra travel and expense to get appropriate patient care.
Expanded Access Programs. A Expanded Access Program (EAP) allows physicians and patients access to much needed medicines when there are no other viable options left. These may be investigational therapies as well as drugs not available in the patients' country and the ultimate intention is to treat unmet clinical need. WEP Clinical. (Also see Medication Programs)
Systemic sclerosis : What is currently available for treatment? Therapy options in systemic sclerosis have changed over the past 10 years and this trend will also continue in the future. PubMed, Internist (Berl), 2016 Dec;57(12):1155-1163.
Systemic sclerosis (SSc) trial design moving forward. This article highlights recent advances in research methodology, and broadens the potential range of design and analytic considerations when planning an SSc trial. Journal of Scleroderma and Related Disorders, JSRD 2016; 1(2): 177 - 180.
Strategy for treatment of fibrosis in systemic sclerosis: Present and future. This review describes recent advances in the strategy for treatment of fibrosis in SSc and future perspectives. Wiley Online Library, 01/04/2016.
Flipping the switch on scleroderma. This research shows promise for the development of a new drug that can reverse the fibrosis process by flipping the main switch on all of the signaling pathways. Michigan State University, 04/03/2014.
Systemic Scleroderma Symptoms. At present, there are no proven treatments or cure for any forms of scleroderma. However, there are effective therapies for many of the symptoms as well as clinical trials for specific scleroderma related symptoms. ISN. (Also see Types of Scleroderma)
Photos of Symptoms
Similar Skin Diseases
Skeletal (Bones, Muscles)
Skin and Hair
Genentech Completes Phase III Enrollment for Study of Actemra in Systemic Sclerosis. Genentech has completed enrollment for a Phase III clinical trial investigating the use of Actemra (tocilizumab) in patients with systemic sclerosis. The aim of the study is to assess the efficacy and safety of Actemra compared to placebo for the treatment of systemic sclerosis. ClinicalTrials.gov, 07/25/2017.
Short–course tocilizumab (TCZ) increases risk of hepatitis B virus (HBV) reactivation in patients with rheumatoid arthritis: a prospective clinical observation. To identify the exact risk of TCZ on HBV infection and the prognosis of TCZ–related HBV reactivation, further studies with larger sample sizes and fewer confounding factors are needed. PubMed, Int J Rheum Dis, 02/03/2017. (Also see Rheumatoid Arthritis in Overlap and Liver Involvement)
Safety and efficacy of subcutaneous tocilizumab in adults with systemic sclerosis (faSScinate): a phase 2, randomised, controlled trial. The difference was greater in the tocilizumab group than in the placebo group and we found some evidence of less decline in forced vital capacity. The efficacy and safety of tocilizumab should be investigated in a phase 3 trial before definitive conclusions can be made about its risks and benefits. PubMed, Lancet, 05/05/2016.
A Study of the Efficacy and Safety of Tocilizumab in Participants With Systemic Sclerosis (SSc) [focuSSced]. This study will assess the efficacy and safety of tocilizumab compared with placebo in participants with SSc across approximately 120 global study sites. The study will consist of a 48-week, double-blind, placebo-controlled period followed by a 48-week open-label treatment period. ClinicalTrials.gov.
Efficacy of Tocilizumab in the treatment of Eosinophilic fasciitis: Report of one case. This report describes for the first time the efficacy of an anti interleukin-6 agent in Eosinophilic fasciitis treatment. PubMed, Joint Bone Spine, 07/07/2015.
Genentech Granted FDA Breakthrough Therapy Designation for Actemra® for Systemic Sclerosis. On June 10, 2015, Genentech announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation status to Actemra® (tocilizumab) for the treatment of systemic sclerosis (also known as scleroderma), a rare and potentially life-threatening disease with no FDA-approved therapy. ISN News Release, 06/10/2015.
Actemra Looks Promising in Scleroderma. Encouraging results were seen for tocilizumab (Actemra) in the treatment of diffuse systemic sclerosis in a proof-of-concept study. Medpage Today, 04/29/2015.
A Study of RoActemra/Actemra (Tocilizumab) Versus Placebo in Patients With Systemic Sclerosis. This multicenter, randomized, double-blind, placebo-controlled, two-arm, parallel-group study will evaluate the efficacy and safety of RoActemra/Actemra (tocilizumab) in patients with systemic sclerosis. ClinicalTrials.gov.
Multiplex serum protein analysis reveals potential mechanisms and markers of response to hyperimmune caprine serum (HICS) in systemic sclerosis. Our results suggest mechanisms of action for HICS, including upregulation of α-MSH, that has been shown to be anti–fibrotic in preclinical models, and possible markers to be included in future trials targeting skin in diffuse cutaneous systemic sclerosis. PubMed, Arthritis Res Ther, 2017 Mar 7;19(1):45.
Treatment of diffuse systemic sclerosis with hyperimmune caprine serum (AIMSPRO): a phase II double-blind placebo-controlled trial. These results confirm tolerability and safety of this novel biological agent in established diffuse SSc. PubMed, Ann Rheum Dis. 2014 Jan;73(1):56-61.
|Overview of Stem Cells and Stem Cell Transplants
Lysophosphatidic Acid (LPA) Signaling through the Lysophosphatidic Acid-1 Receptor Is Required for Alveolarization. These results are consistent with the abnormal lung phenotype of LPA1 KO mice, being attributable to reduced alveolar septal formation during development, rather than to increased septal destruction as occurs in the emphysema of chronic obstructive pulmonary disease. PubMed, Am J Respir Cell Mol Biol, 2016 Jul;55(1):105-16.
Bristol-Myers Squibb acquires Amira Pharmaceuticals. AM152, an orally available lysophosphatidic acid 1 (LPA1) receptor antagonist has completed Phase I clinical studies and is poised for Phase IIa proof-of-confidence studies for the treatment of idiopathic pulmonary fibrosis (IPF) and systemic Sclerosis (SSc), or scleroderma. Marketizer.
The recruitment status of this study is unknown
because the information has not been verified recently. 10/15/12.
Study of Ambrisentan With Antifibrotic Agent Combination Therapy in Diffuse Systemic Sclerosis. The hypothesis on which we are basing this study is that an endothelin receptor antagonist and disease modifying agent with antifibrotic properties will have additive influence on fibrosis, inhibit cellular and humoral hyperactivity and interfere with smooth muscle proliferation in the vessel wall. The combination of these two agents will also be the first regimen to address the heterogeneity of scleroderma manifestations including ILD, pulmonary arterial hypertension and skin manifestations. ClinicalTrials.gov. Identifier: NCT01093885.
CATALYST – for patients with CTD-PAH. CATALYST is a Phase III clinical study evaluating the safety and efficacy of bardoxolone methyl (an oral medication) for the treatment of pulmonary arterial hypertension associated with connective tissue disease (CTD-PAH). Approximately 200 patients will be enrolled at centres in various countries around the world. Reata Pharmaceuticals, Inc., 06/29/2017.
Reata's Bardoxolone Methyl as PAH Therapy Shows Promise in Initial Phase 2 Data. In the LARIAT trial, efficacy analyses showed bardoxolone methyl increased 6MWD at doses of 2.5 to 10 mg through 16 weeks of treatment. Pulmonary Hypertension News, 10/27/2015.
Pre-Qualification Survery for BEAT Clinical Trial. The investigational oral drug in this trial is called Beraprost Sodium-314d-Modified Release (BPS-314d-MR). This study will help researchers determine if the combination of the investigational oral drug and the inhaled medicine Tyvaso® (treprostinil) is safe and effective in delaying the progression of PAH. Lung Biotechnology Inc.
Belimumab for the Treatment of Early Diffuse Systemic Sclerosis: Results of a Randomized, Double–Blind, Placebo–Controlled, Pilot Trial. Significant decrease in expression of B–cell signaling and pro–fibrotic genes and pathways was observed in patients with improved modified Rodnan skin score in the belimumab group but not in the placebo group. PubMed, Arthritis Rheumatol, 10/26/2017.
Tracleer® (Bosentan) appears to be beneficial for a variety of scleroderma symptoms, such as digital ulcers, pulmonary fibrosis, pulmonary hypertension, and skin fibrosis. ISN.
Tracleer for Digital Ulcers
Tracleer for Pulmonary Fibrosis
Tracleer for Pulmonary Hypertension
|Tracleer for Raynaud's
Tracleer for Scleroderma Renal Crisis
Tracleer for Skin Fibrosis
The 5-HT1A receptor agonist buspirone improves esophageal motor function and symptoms in systemic sclerosis: a 4-week, open-label trial. More conclusive evaluation is needed; however, buspirone could potentially be given under observation for objective improvement in all patients with SSc who report reflux symptoms despite undergoing standard treatment. BioMed Central, Arthritis Research & Therapy, 09/01/2016. (Also see Dysmotility Syndrome in Systemic Scleroderma)
Cyclophosphamide and Revimmune for Scleroderma Lung Disease. Clinical trial and research information on cyclophosphamide for pulmonary alveolitis. ISN.
|SCOT Clinical Trial
SCOT Substudy 01
Northwestern University Clinical Trials
|Scleroderma Lung Study II
Cyclophosphamide and rATG With Hematopoietic Stem Cell Support Clinical Trial. To evaluate the efficacy (phase II) of two treatment modalities: pulse cyclophosphamide versus high dose cyclophosphamide and rATG (rabbit antithymocyte globulins) rescued with autologous peripheral blood stem cell transplant (PBSCT). Northwestern University. ClinicalTrials.gov.
Oral treprostinil diethanolamine for pulmonary arterial hypertension. Emerging data suggest that in carefully selected patients oral treprostinil may be able to replace continuously infused treprostinil. PubMed, Expert Rev Clin Pharmacol, 2015 Jan;8(1):55-60.
Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine (DISTOL-1). This study will evaluate the effect of treprostinil diethanolamine (UT-15C) sustained release tablets (compared to placebo) on digital ulcers in patients with scleroderma. ClinicalTrials.gov. (Also see Digital Ulcers)
Fludarabine and Total-Body Irradiation Followed By Donor Peripheral Stem Cell Transplant in Treating Patients With Severe Systemic Sclerosis. This phase II trial is studying how well giving fludarabine together with total-body irradiation followed by a donor peripheral stem cell transplant works in treating patients with severe systemic sclerosis. Clinical Trials.gov.
Study Shows Treatment with Fresolimumab Able to Reduce Skin Scarring in Systemic Scleroderma patients. Fresolimumab, sponsored by Genzyme, a drug agent that targets a chemical mediator in the body called TGF-beta, is able to block scarring which could mean a major treatment advance for scarring-mediated organ dysfunction. Scleroderma News, 06/23/2015. (Also see Causes of Scleroderma: Molecular Defect and Skin Fibrosis)
Fresolimumab In Systemic Sclerosis. The purpose of this study is to determine if fresolimumab, which is sponsored by Genzyme, is safe in treating people with systemic sclerosis (scleroderma) and to investigate the effect of fresolimumab in the skin of these individuals. ClinicalTrials.gov. 07/15/2014.
Fibrocell’s FX-013 Therapy Wins FDA Rare Pediatric Disease Designation for Localized Scleroderma. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to FX-013, a Fibrocell gene therapy candidate to treat children with moderate to severe localized scleroderma. Scleroderma News, 06/13/2017. (Also see Juvenile Scleroderma Research)
Galectin’s Candidate Therapy GR-MD-02 Patent Extended to Cover Several Illnesses Including Systemic Sclerosis. The U.S. Patent and Trademark Office has granted Galectin Therapeutics a new patent that extends coverage of its candidate therapy GR-MD-02 to treat systemic sclerosis. Scleroderma News, 06/27/2017.
Gilead Announces Top-Line Phase 2 Results for GS-4997 (Selonsertib) in Nonalcoholic Steatohepatitis (NASH), Pulmonary Arterial Hypertension (PAH) and Diabetic Kidney Disease (DKD) GS-4997 demonstrated anti-fibrotic activity in an open-label Phase 2 clinical trial that included 72 patients with NASH and moderate to severe (F2-F3) liver fibrosis, who received treatment with GS-4997. Drugs.com, 10/26/2016. (Also see Treatments for Pulmonary Hypertension)
Habeo Cell Therapy is an experimental treatment in clinical trial for scleroderma hand involvement. It entails removing fat cells from the patient, harvesting the stem cells, and reintroducing them. The Phase III trials showed some benefit, particularly for hand involvement in diffuse cutaneous scleroderma patients who have sclerodactyly (hands hardened by scleroderma.) ISN.
The protective effect of hydrogen sulfide (H2S) on systemic sclerosis associated skin and lung fibrosis in mice model. We demonstrate the beneficial effects of H2S on SSc–associated skin and lung fibrosis. H2S may be a potential therapy against this intractable disease. PubMed, Springerplus, 2016 Jul 15;5(1):1084.
Preclinical Study Shows IVA337’s Potential in Treating SSc Skin and Cardiovascular Complications. Inventiva’s lead therapy candidate IVA337 for the treatment of systemic sclerosis (SSc) was shown to prevent the progression of lung fibrosis and the remodeling of blood vessels in preclinical animal models of lung fibrosis and systemic sclerosis. Scleroderma News, 07/28/2017.
IVIG Treatment in Systemic Sclerosis. The primary outcome measure is to study the effects of IVIG on the skin in patients with scleroderma. Study centers are Georgetown University and Johns Hopkins. NIH.
JB Therapeutics raises $662K to start scleroderma trial. The company plans to use the money to run a Phase 2 clinical trial in the orphan disease systemic sclerosis, a type of scleroderma that affects the skin and vital internal organs such as the lungs, heart, and kidneys. Boston Business Journal. 04/11/12.
Safety and Tolerability of MEDI-551 in Subjects with Systemic Sclerosis (SSC): Results from A Phase 1 Randomized, Placebo-Controlled Escalating Single-Dose Study. Rapid sustained B-cell depletion was observed following single IV infusion of MEDI-551. Clinical activity was suggested by decreased mRSS. Ann Rheum Dis, 2014;73:562.
Study Reveals Promising Data on Nilotinib as a Therapy for Diffuse Systemic Sclerosis. Nilotinib was generally well-tolerated by systemic sclerosis patients. In terms of efficacy, controlled trials with a larger number of patients should be conducted in order to draw definitive conclusions, although the pilot study showed a significant improvement in MRSS. Scleroderma News.
Study Reveals Promising Data on Nilotinib as a Therapy for Diffuse Systemic Sclerosis. Nilotinib was generally well-tolerated by systemic sclerosis patients, with tolerability being primarily limited by mild cardiovascular side effects. Scleroderma News, 08/24/2015.
Nilotinib (Tasigna) in the Treatment of Systemic Sclerosis. This Phase II Open Label study is currently recruiting participants. The purpose of this study is to learn how safe and tolerable a medication called Nilotinib (Tasigna) will be for patients diagnosed with Systemic Sclerosis. The secondary goal is to assess how effective Nilotinib is in treating patients with scleroderma. ClinicalTrials.gov. (Also see Scleroderma Clinical Trials: Current Studies)
Nintedanib receives orphan drug designation for systemic sclerosis. The FDA has granted Orphan Drug Designation to Boehringer Ingelheim's nintedanib for the treatment of (SSc), including associated interstitial lung disease (SSc–ILD). Pharmacy Times, 09/27/2016.
OFEV (nintedanib) to Be Tested in Systemic Sclerosis Patients (SSc) with Interstitial Lung Disease (ILD). The potential use of nintedanib in conditions that commonly occur with fibrotic lung conditions, such as systemic sclerosis are being investigated. Scleroderma News, 12/22/2015. (Also see Pulmonary Fibrosis Treatments)
A Trial to Compare Nintedanib With Placebo for Patients With Scleroderma Related Lung Fibrosis. It is the purpose of the trial to confirm the efficacy and safety of nintedanib 150 mg bid in treating patients with systemic sclerosis – interstitial lung disease, compared with placebo. ClinicalTrials.gov. 11/17/2015.
Opsumit® (Macitentan) Prescribing Information. This is a prescription medicine used to treat pulmonary arterial hypertension (PAH, WHO Group 1). PAH is high blood pressure in the arteries of your lungs. Actelion Pharmaceuticals US, Inc.
Clinical Trial: Macitentan for the Treatment of Digital Ulcers in Systemic Sclerosis Patients (DUAL-2). The primary objective is to demonstrate the effect of macitentan on the reduction of the number of new digital ulcers in patients with systemic sclerosis and ongoing digital ulcers (DU). ClinicalTrials.gov. 12/18/12. (Also see Digital Ulcer Treatments and Digital Ulcers Research)
Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness for Subjects With Systemic Sclerosis With Interstitial Lung Disease. The purpose of this first study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of pomalidomide (CC-4047) in the treatment of subjects with diffuse cutaneous systemic sclerosis with interstitial lung disease. Study ID NCT01559129. ClinicalTrials.gov. 10/15/12. (Also see Diffuse Scleroderma and Pulmonary Fibrosis Research)
Corbus Wins Expanded U.S. Patent Coverage of Anabasum Therapy for Systemic Sclerosis. Corbus Pharmaceuticals has extended its intellectual property portfolio with a new patent for its investigative cannabinoid–derived drug candidate anabasum to treat inflammatory diseases. Scleroderma News, 11/02/2017. (Also see Endocannabinoid System)
Corbus to Begin Phase 3 Trial of Anabasum (Resunab) for Systemic Sclerosis Treatment. Corbus Pharmaceuticals, will commence a single Phase 3 clinical trial evaluating Anabasum (formerly known as Resunab or JBT-101) to treat diffuse cutaneous systemic sclerosis (SSc). The international, randomized, double–blind, placebo–controlled trial will enroll about 270 adults with SSc. Scleroderma News, 04/06/2017.
Corbus to Present Promising Data on Resunab as Systemic Sclerosis Treatment. Corbus Pharmaceuticals will present results showing that Resunab (JBT-101) can trigger specific molecular pathways which resolve inflammation and halt fibrosis. Scleroderma News, 03/07/2017.
Resunab, Potential Scleroderma Treatment, Shows Ability to Stop Inflammation in Early Clinical Trial. Corbus Pharmaceuticals reported new findings supporting the anti–inflammatory actions of its lead compound Resunab (JBT-101), which is currently being explored in a clinical trial for scleroderma. Scleroderma News, 09/29/2016.
Corbus Pharmaceuticals Receives FDA Approval for Open–Label Extension to Its Phase 2 Trial of Resunab for Systemic Sclerosis. Corbus Pharmaceuticals announced today that the U.S. Food and Drug Administration (FDA) has granted approval for a 12–month open–label extension study of the ongoing Phase 2 clinical trial of Resunab™ for the treatment of diffuse cutaneous systemic sclerosis. Corbus Pharmaceuticals, 04/12/16.
Corbus Pharmaceuticals Announces First Patient Dosed in Phase 2 Study of Resunab for Systemic Sclerosis. Corbus Pharmaceuticals announced today that the first subject was dosed in the Phase 2 clinical study of its investigational new drug Resunab™ for the treatment of diffuse cutaneous systemic sclerosis. Corbus Pharmaceuticals, 10/07/15.
Corbus Pharmaceuticals Initiates Phase 2 Study Patient Enrollment of Resunab for Systemic Sclerosis. Corbus Pharmaceuticals recently announced the initiation of patient enrollment for its Phase 2 trial of Resunab™ for the treatment of diffuse cutaneous systemic sclerosis. Scleroderma News, 09/02/2015.
Investigational Scleroderma Drug Takes Novel Approach To Treating Inflammation. A new proposed treatment for scleroderma, Resunab, will soon be in Phase 2 clinical trials. Scleroderma News, 03/16/2015.
Rituximab in Systemic Sclerosis (RECOVER) The purpose of this study is to determine whether rituximab is effective in the treatment of articular symptoms that occur in systemic sclerosis related polyarthritis. ClinicalTrials.gov.
Reduced type I collagen gene expression by skin fibroblasts of patients with systemic sclerosis after one treatment course with rituximab. These data provide further evidence of B-cell involvement in the pathogenesis of scleroderma and targeting B cells may be a promising treatment for scleroderma patients. PubMed, Clin Exp Rheumatol, 2015 Sep-Oct;33 Suppl 91(4):160-167. (Also see B Cells and T Cells)
Rituximab in diffuse cutaneous systemic sclerosis (SSc): should we be using it today? We summarize the therapeutic use of rituximab in SSc and the basic science evidence suggesting that B cells and autoantibodies are the primary drivers of fibrosis in skin and lung tissue. PubMed, Rheumatology (Oxford), 07/01/2015. (Also see Causes of Scleroderma: B Cells and T Cells)
Rituximab for Treatment of Systemic Sclerosis-Associated Pulmonary Arterial Hypertension (SSc-PAH). This trial will determine if rituximab, an immunotherapy, has a marked beneficial effect on clinical disease progression, with minimal toxicity, in patients with SSc-PAH when compared to placebo. ID NCT01086540. ClinicalTrials.gov.
To Evaluate the Effect of Different Doses of SAR156597 Given to Patients With Idiopathic Pulmonary Fibrosis (IPF). To assess in safety and tolerability of ascending doses of SAR 156597 in adult patients with Idiopathic Pulmonary Fibrosis (IPF), administered subcutaneously once weekly over a 6-week period. Sanofi. ClinicalTrials.gov.
Efficacy of mycophenolate mofetil (MMF) and oral cyclophosphamide (CYC) on skin thickness: Post–hoc analyses from the Scleroderma Lung Study I and II. (SLS-I and II) In SLS-II, MMF and CYC resulted in improvements in the modified Rodnan skin score in diffuse cutaneous systemic sclerosis over 24 months. PubMed, Arthritis Care Res (Hoboken), 05/23/2017. (Also see Immunosuppressants)
Improved cough and cougho–specific quality of life in patients treated for sclerodermao–related interstitial lung disease (SSc–ILD): Results of Scleroderma Lung Study II. Frequent cough occurs commonly in SSc–ILD and might serve as a useful surrogate marker of treatment response in SSc–ILD trials. PubMed, Chest, 12/22/2016.
Fewer Mycophenolate Adverse Events in Scleroderma Lung Study. Mycophenolate mofetil can be substituted for cyclophosphamide for the immunosuppressive treatment of scleroderma-related interstitial lung disease and might even be safer. Medscape, 11/02/2015. (Also see Pulmonary Fibrosis Treatments and Immunosuppressants)
Comparison of Therapeutic Regimens for Scleroderma Interstitial Lung Disease (The Scleroderma Lung Study II) (SLSII). The purpose of this study is to determine whether people with symptomatic scleroderma-related interstitial lung disease experience more respiratory benefits from treatment with a 2-year course of mycophenolate mofetil or treatment with a 1-year course of oral cyclophosphamide. ClinicalTrials.gov.
The Scleroderma Patient-centered Intervention Network (SPIN) Cohort: protocol for a cohort multiple randomised controlled trial (cmRCT). The use of the cmRCT design, the development of self-guided online interventions and partnerships with patient organisations will allow SPIN to develop, rigourously test and effectively disseminate psychosocial and rehabilitation interventions for people with SSc. BMJ Open.
|Overview of Stem Cells and Stem Cell Transplants
Phase 3 Trial to Begin for Terguride in Diffuse Cutaneous Scleroderma. The German pharmaceutical company Medac, in August, will begin Phase 3 of a clinical trial to access the therapeutic potential of terguride, a disease–modifying drug for the treatment of diffuse cutaneous systemic sclerosis. Scleroderma News, 06/09/2016.
Still enrolling as of 10/15/12.
Families with Twins or Siblings where one has Systemic Rheumatic Disorders (Rheumatoid Arthritis, Juvenile Rheumatoid Arthritis, Lupus, Scleroderma, or Myositis) and one does not. The goal of study 03-E-0099 is to assess why one twin or sibling developed disease and why the other brother or sister did not.
The siblings may or may not be twins, but must be of the same gender and be within a 3-year age difference. Biological parents, or, in some cases, children, will also be included in the study.
Families may enroll at the NIH Clinical Center in Bethesda, Maryland, just 9 miles north of Washington, DC or at their local physician's office. Transportation assistance may be available and there is no charge for study-related evaluations and medical tests.
For information on the study, call the NIH patient recruiting office toll free at 1-800-411-1222 (For TTY: 1-866-411-1010). National Institutes of Health Clinical Center (NIH). 11/25/05. (Also see Scleroderma Research Registries and Causes of Scleroderma: Genetics)
Cumberland Pharmaceuticals Announces New Program To Develop Vasculan™ For Systemic Sclerosis The U.S. Food and Drug Administration (FDA) has cleared Cumberland's investigational new drug application (IND) for Phase II clinical program for Vasculan™ in patients with systemic sclerosis. Cumberland Pharmaceuticals, 04/27/2016.
Velcade® (Bortezomib) is being studied in clinical trials for scleroderma pulmonary fibrosis.
Comparing and Combining Bortezomib and Mycophenolate in SSc Pulmonary Fibrosis. This is a Phase II clinical trial at Northwestern. It is still recruiting as of March 2016. Clinicaltrials.gov.
This study is currently recruiting participants.
Zibotentan Better Renal Scleroderma Outcome Study (ZEBRA) The purpose of this study is to see how effective a new endothelin blocker called Zibotentan is in treating patients who have scleroderma and have gone on to develop reduced kidney function as a complication. ClinicalTrials.gov.
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