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.
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say. The ongoing COVID-19 pandemic has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. Scleroderma News, 05/28/2020.
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
The Impact of Tocilizumab (TCZ) on Anxiety and Depression in Patients With Rheumatoid Arthritis (RA). This study has demonstrated a favourable impact of TCZ therapy on parameters reflecting depression and anxiety severity in patients with RA. PubMed, Eur J Clin Invest, 2020 Jun 1;e13268. (Also see Depression and Symptoms and Complications of Rheumatoid Arthritis)
Implication of Baseline Levels and Early Changes of C-reactive Protein (CRP) for Subsequent Clinical Outcomes of Patients With Rheumatoid Arthritis (RA) Treated With Tocilizumab. CRP and its early course may inform, to some extent, the estimation of potential therapeutic success in patients with RA. PubMed, Ann Rheum Dis, 05/05/2020. (Also see Treatments for Rheumatoid Arthritis)
Case Report: COVID-19 in a patient with systemic sclerosis (SSc) treated with tocilizumab for Ssc-interstitial lung disease (ILD.) Our case indicates that IL-6-blocking treatment given for chronic autoimmune diseases may even prevent the development of severe COVID-19. BMJ Journals, Ann Rheum Dis.
Tocilizumab and the Risk of Cardiovascular Disease (CVD): Direct Comparison Among Biologic Disease-Modifying Antirheumatic Drugs for Rheumatoid Arthritis (RA) Patients. Tocilizumab was associated with a CVD risk comparable to that for etanercept as well as a number of other biologics used for the treatment of RA. PubMed, Arthritis Care Res (Hoboken), 2019 Aug;71(8):1004-1018. (Also see Treatments for Rheumatoid Arthritis)
Therapeutic interleukin-6 blockade reverses transforming growth factor-beta pathway activation in dermal fibroblasts: insights from the faSScinate clinical trial in systemic sclerosis (SSc). The profound impact of IL-6R blockade on the activated fibroblast phenotype highlights the potential of IL-6 as a therapeutic target in SSc and other fibrotic diseases. PubMed, Ann Rheum Dis, 05/31/2018. (Also see Interleukins)
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)
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.
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.
|Overview of Stem Cells and Stem Cell Transplants
Advancements in Understanding the Role of Lysophospholipids and Their Receptors in Lung Disorders Including Bronchopulmonary Dysplasia. This article is intended to promote discussion and generate hypotheses. PubMed, Biochim Biophys Acta Mol Cell Biol Lipids, 2020 Jul;1865(7):158685.
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.
Early treatment with ambrisentan of mildly elevated mean pulmonary arterial pressure (mPAP) associated with systemic sclerosis. This is the first randomized, double-blind, placebo-controlled study testing the effect of ambrisentan in patients with mildly elevated mPAP and/or exercise pulmonary hypertension. PubMed, Arthritis Res Ther, 2019 Oct 26;21(1):217. (Also see Letairis (Ambrisentan) and Endothelin Receptor Antagonists)
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.
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 alters transitional B-cell subset proportions in patients with stable systemic lupus erythematosus. We show that Belimumab-mediated B-cell activating factor depletion reduces the Transitional 2 subset in patients. PubMed, Lupus, 2019 Oct;28(11):1337-1343. (Also see Treatments for Lupus)
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.
Phase 2 Study of Bermekimab in Treating Scleroderma Enrolls 1st Patient. The first person has been enrolled in a clinical trial evaluating the investigational therapy bermekimab in adults with scleroderma, XBiotech, the developer of bermekimab, announced. Scleroderma News, 10/07/2019.
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
Evaluation and Management of Esophageal Manifestations in Systemic Sclerosis (SSc). This review summarizes our knowledge concerning the evaluation and management of esophageal manifestations in SSc patients, including emerging therapeutic modalities. PubMed, Ann Gastroenterol, Mar-Apr 2018;31(2):165-170. (Also see Dysmotility Syndrome in Systemic Scleroderma)
Corbus Pharmaceuticals Expands Target Indications by Adding More Than 600 Compounds Focused on the Endocannabinoid System from Jenrin Discovery. Corbus Pharmaceuticals Holdings, Inc. announced today that it has licensed the exclusive worldwide rights to develop, manufacture and market drug candidates, which includes CRB-4001, a peripherally–restricted, CB1 inverse agonist targeting liver, lung, heart and kidney fibrotic diseases. Corbus Pharmaceuticals, 09/20/2018. (Also see Endocannabinoid System)
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.
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)
iBio Selects Lead Candidate for its Fibrosis Therapeutics Program. iBio, Inc. announced today that it had selected an E4-Fc fusion protein as its lead candidate for further development of a drug against fibrotic diseases, including systemic sclerosis and idiopathic pulmonary fibrosis. Globe Newswire, 03/14/2018.
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.
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.
FDA Grants Fast Track Status to Fibrocell's Gene Therapy FCX-013 for Localized Scleroderma. Fibrocell Science's gene therapy candidate FCX-013 received fast track designation by the U.S. Food and Drug Administration as a treatment for moderate––to–severe localized scleroderma. Scleroderma News, 09/06/2018.
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)
Phase 2a Trial Will Evaluate GLPG1690 in Diffuse Cutaneous Scleroderma. A Phase 2a clinical trial will test the investigational therapy GLPG1690 in patients with diffuse cutaneous scleroderma. Scleroderma News, 01/10/2019.
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.
Selonsertib for patients with bridging fibrosis or compensated cirrhosis due to NASH: Results from randomized phase III STELLAR trials. Forty-eight weeks of selonsertib monotherapy had no antifibrotic effect in patients with bridging fibrosis or compensated cirrhosis due to NASH. PubMed, J Hepatol, 2020 Jul;73(1):26-39. (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.
Hyaluronic acid and platelet-rich plasma, a new therapeutic alternative for scleroderma patients: a prospective open-label study. Our study demonstrates that filler injections of hyaluronic acid and platelet-rich plasma represent an efficient local therapeutic alternative for patients affected by scleroderma. PubMed, Arthritis Res Ther, 2019 Dec 13;21(1):286.
Hydrogen Sulfide: A Therapeutic Option in Systemic Sclerosis (SSc). This paper will review the role of hydrogen sulphide in vascular (patho-) physiology and potential disturbances in SSc. PubMed, Int J Mol Sci, 2018 Dec 19;19(12):4121.
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.
Lenabasum Fails to Show Efficacy in Diffuse Cutaneous Scleroderma (dcSSc), Phase 3 Trial Shows. Treatment with lenabasum failed to outperform a placebo when given as an add-on to standard immunosuppressants in adults with dcSSc, top-line data from a Phase 3 trial show. Scleroderma News, 09/09/2020.
Corbus Pharmaceuticals to Commence Phase 3 Study of Lenabasum for the Treatment of Rare Autoimmune Disease Dermatomyositis. Corbus Pharmaceuticals announced today that the Company will proceed with a Phase 3 trial evaluating the efficacy and safety of lenabasum for the treatment of dermatomyositis. Globe Newswire, 07/25/2018. (Also see Treatments for Dermatomyositis and Endocannabinoid System)
Lenabasum Safe and Effective in Diffuse Scleroderma Patients, One–year Trial Data Show. Lenabasum, by Corbus Pharmaceuticals, was shown to be safe, well-tolerated and of benefit to patients treated for one year in a long-term extension of a Phase 2 trial. Scleroderma News, 05/17/2018. (Also see Endocannabinoid System)
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.
Baseline Plasma Cell Gene Signature Predicts Improvement in Systemic Sclerosis (SSc) Skin Scores Following Treatment With Inebilizumab (MEDI-551) and Correlates With Disease Activity in Systemic Lupus Erythematosus and Chronic Obstructive Pulmonary Disease. An elevated pretreatment PC signature was associated with increased benefit from MEDI-551 in SSc. PubMed, Arthritis Rheumatol, 2018 Dec;70(12):2087-2095.
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.
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 in Progressive Fibrosing Interstitial Lung Diseases. In patients with progressive fibrosing interstitial lung diseases, the annual rate of decline in the forced vital capacity was significantly lower among patients who received nintedanib than among those who received placebo. PubMed, N Engl J Med, 2019 Oct 31;381(18):1718-1727. (Also see Treatments for Pulmonary Fibrosis)
FDA approves first treatment for patients with rare type of lung disease. The U.S. Food and Drug Administration today approved Ofev (nintedanib) capsules to slow the rate of decline in pulmonary function in adults with interstitial lung disease associated with systemic sclerosis or scleroderma, called SSc-ILD. FDA News Release, 09/06/2019. (Also see Treatments for Pulmonary Fibrosis)
Phase III study showed nintedanib slows the loss of pulmonary function in people living with systemic sclerosis associated interstitial lung disease (ILD). Results show that nintedanib slows the loss of pulmonary function in patients with SSc-ILD compared to placebo. Business Wire, 05/20/2019. (Also see Treatments for Pulmonary Fibrosis)
Exploring Novel Treatment for Scleroderma–Associated Interstitial Lung Disease. This study is one of three major multicenter trials in which Cleveland Clinic’s Rheumatic Lung Disease Program is participating. Consult QD, 12/13/2018. (Also see Treatments for Pulmonary Fibrosis)
Nintedanib Gets Fast Track Designation for Treatment of Systemic Sclerosis. Boeringer Ingelheim announced this morning that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to nintedanib for the treatment of systemic sclerosis with associated interstitial lung disease (SSc-ILD). Rare Disease Report, 03/19/2018.
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. (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. (Also see Diffuse Scleroderma and Pulmonary Fibrosis Research)
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.
Effects of treatment with rituximab on microcirculation in patients with long–term systemic sclerosis. Rituximab improves microcirculation in long–term SSc and stabilization and reduced progression of microcirculation abnormalities were achieved. PubMed, BMC Res Notes, 2018 Dec 10;11(1):874. (Also see Biologic Agents)
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)
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.
Assessing the response of morphea and limited scleroderma to tranilast: a small prospective study comparing topical corticosteroids to a combination of topical corticosteroids and tranilast. This was a small single center study and the role of tranilast in the management in scleroderma warrants further investigation in larger trials. Dove Press, 07/04/2018. (Also see Morphea Treatments)
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). (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.
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.
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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