Scleroderma Clinical Trials: Current Studies, Open Enrollments

Author: Shelley Ensz. Scleroderma is highly variable. See Types of Scleroderma. Read Disclaimer
Symptom Treatments
Actemra (Tocilizumab)
ADRC Injections/STAR Trial
Allogenic Stem Cell Transplant
AM152 (Lysophosphatidic Acid 1)
Ambrisentan for Scleroderma
Bosentan, Tracleer
Cyclophosphamide, Cytoxan
Cyclophosphamide and rATG With Hematopoietic Stem Cell Support
Fludarabine/Donor Peripheral SCT
Macitentan (Opsumit)
Nilotnib (Tasigna)
Rituximab for PAH
SAR156597 for IPF (Sanofi)
Scleroderma Lung Study II
SPIN: Patient-centered Network
Stem Cell Transplants for Scleroderma
Twins and Siblings Study


Two cups with bubblesExcitement 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 they it is well worth the extra travel and expense to get appropriate patient care.

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.

Treating skin and lung fibrosis in systemic sclerosis: a future filled with promise? The most promising targets include inhibitors of B-cells, tyrosine kinases, 5-hydroxytryptamin receptors, interleukin-6 and Wnt signalling. PubMed, Curr Opin Pharmacol, 2013 Jun;13(3):455-62. (Also see Skin Fibrosis)

Systemic Sclerosis (Scleroderma) Symptom Treatments

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)

Autoimmune Diseases
Heart (Cardiac)
Kidney (Renal)
Lung (Pulmonary)
Photos of Symptoms
Similar Skin Diseases
Sjogren's Syndrome
Skeletal (Bones, Muscles)
Skin and Hair

Actemra (Tocilizumab) by Hoffmann-La Roche

June 2015: Phase II Study is not yet open for participant recruitment.

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.

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.

Roche Gains U.S. Breakthrough Therapy Designation for Actemra Drug. The U.S. Food and Drug Administration research showed early treatment with Actemra could prevent irreversible damage to joints in patients with systemic sclerosis and held stave off long-term disability. Dow Jones Business News, 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.

June 2015: This study is ongoing, but not recruiting participants.

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.

AIMSPRO for Systemic Sclerosis

Daval AIMSPRO as monotherapy in scleroderma yields positive results. The double-blind placebo-controlled phase II clinical study to assess the safety and tolerability of AIMSPRO as a monotherapy in Established Diffuse Cutaneous Systemic Sclerosis (Scleroderma) yielded positive results. PBR Clinical Trials. 10/11/2011.

ADRC Injections (Adipose-derived Stem and Regenerative Cells) by Cytori Therapeutics

U.S. STAR Clinical Trial enrollment begins 2015 in U.S.

First Patient Treated in the U.S. STAR Trial. Cytori has successfully begun its first ever U.S. approval trial and consistent with their experience in treating scleroderma in Europe, the patient tolerated the procedure well. Cytori, 08/25/2015.

Scleroderma Treatment With Celution Processed Adipose Derived Regenerative Cells (STAR). The primary objective of this study is to assess the safety and efficacy of the Celution Device in the processing of an autologous graft consisting of adipose derived regenerative cells (ADRCs) in the treatment of hand dysfunction due to scleroderma., 08/24/2015.

FDA Approves Cytori U.S. Pivotal Scleroderma Trial. The STAR trial will begin enrolling in 2015 and will include 80 patients, in up to 12 centers in the United States. The trial is a randomized, double-blind, placebo-controlled study of the safety and efficacy of a single administration of Cytori's lead cellular therapeutic, ECCS-50, in scleroderma patients with hand dysfunction. MarketWatch, 01/12/2015.

Completed Study. Positive Results. France Only

Cytori Therapeutics Receives Approval for Scleroderma Study in France. Per the study protocol, 13 patients will receive an injection of their own adipose-derived stem and regenerative cells (ADRCs) into the affected areas of their fingers. StreetInsider. 10/23/12.

Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Systemic Sclerosis

Open Enrollments
Stem Cell Transplantation Centers
Research Studies
Transplant Funding
Related Articles
Patient Stories

AM152, Lysophosphatidic acid 1 (LPA1) by Bristol-Myers Squibb

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. 09/12/2011.

Orphan Drug Status For LPA1 Antagonist AM152 For Treatment Of Idiopathic Pulmonary Fibrosis. Amira Pharmaceuticals, Inc. announced today that AM152, the company's lead LPA1 antagonist, has been granted an orphan drug designation by the U.S. Food and Drug Administration for the treatment of idiopathic pulmonary fibrosis. e!sciencenews. 04/22/2011.

Ambrisentan for Scleroderma

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. Identifier: NCT01093885.

Bosentan, Tracleer

Bosentan (Tracleer) 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 Heart Involvement in SSc
Tracleer for Pulmonary Fibrosis
Tracleer for Pulmonary Hypertension
Tracleer for Raynaud's
Tracleer for Scleroderma Renal Crisis
Tracleer for Skin Fibrosis
Also See

Cyclophosphamide (Cytoxan ®) and Revimmune

Current Studies, Initial Results Positive

Cyclophosphamide (Cytoxan) 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 Research
Also see

Cyclophosphamide and rATG with Hematopoietic Stem Cell Support

This study is ongoing, but not recruiting participants. Updated 10/15/12.

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.


This study is ongoing, but not recruiting participants. Updated 10/15/12.

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. (Also see Digital Ulcers)

Fludarabine and Donor Peripheral SCT

Currently recruiting. Updated 10/15/12.

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

IVIg (Intravenous Immunoglobulin)

Study dates April 2013 to August 2015.

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.

JBT101 by JB Therapeutics

Recruitment begins August 2013 in UK and US

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.

Macitentan (Opsumit)

Macitentan is also known as Opsumit by Actelion. It is a dual endothelin receptor antagonist that was submitted for FDA approval for use in pulmonary hypertension in December 2012.

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). 12/18/12. (Also see Digital Ulcer Treatments and Digital Ulcers Research)


In 2014, MEDI-551 passed Phase I for safety and tolerability in systemic scleroderma, and showed rapid sustained B-cell depletion.

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.

Nilotnib (Tasigna)

This study is ongoing, but not recruiting participants. 10/15/12.

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. (Also see Scleroderma Clinical Trials: Current Studies)

Pomalidomide for Pulmonary Fibrosis in Diffuse Scleroderma

Open Enrollment for Pomalidomide (by Celgene), 2012.

Recruiting in Australia, France, Germany, Italy, Poland, Russia, Spain, Switzerland, United Kingdom, and United States.

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. 10/15/12. (Also see Diffuse Scleroderma and Pulmonary Fibrosis Research)


Phase 2 clinical trials starting 2015.

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 for PAH

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)

B-cell depletion therapy in patients with diffuse systemic sclerosis associates with a significant decrease in PDGFR expression and activation in spindle-like cells in the skin. Rituximab (RTX) may favorably affect skin fibrosis through attenuation of PDGFR expression and activation, a finding that supports a disease-modifying role of RTX in Systemic Sclerosis (SSc). Arthritis Res Ther, 2012 Jun 14;14(3):R145. (Also see Causes of Scleroderma: B and T Cells)

Recruiting until December 2013:

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.

SAR156597 for Idiopathic Pulmonary Fibrosis (Sanofi)

Study dates April 2010 to January 2014

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.

Scleroderma Lung Study II

Still recruiting, as of 10/15/12.

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.

SPIN: Scleroderma Patient-centered Intervention Network

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, 2013;3:e003563.

Stem Cell Transplants for Scleroderma

Continuing Studies, Open Enrollment

Stem Cell Transplants (Bone Marrow Transplants) for Early Diffuse Scleroderma. ISN.

Open Enrollments
Stem Cell Transplantation Centers
Research Studies
Transplant Funding
Related Articles
Patient Stories

Twins and Siblings With and Without Autoimmune Diseases

Still enrolling as of 10/15/12.

NIH Autoimmune Twins and Siblings StudyFamilies 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)


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.

Also See

Sjögren's Treatments & Clinical Trials
Scleroderma Medical News
Scleroderma Symptoms (treatments are listed for each symptom)

Go to Stem Cell (Bone Marrow) Transfusion

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