Scleroderma Treatments and Clinical Trials

Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Systemic Sclerosis This study is designed to examine whether treating patients with high dose Cyclophosphamide and Fludarabine (drugs which reduce the function of your immune system) and CAMPATH-1H (a protein that kills the immune cells that are thought to be causing the disease), followed by return of blood stem cells that have been previously collected from patients brother or sister will stop or reverse the disease. Northwestern University, Chicago, Illinois. ClinicalTrials.gov.

ASTIS Study

ASTIS Study: High Dose Immunoablation and Hematopoietic Stem cell Transplantation versus Monthly Intravenous Pulse Therapy Cyclophosphomide in Severe Systemic Sclerosis. ISN.

Scleroderma Care and Research Journal, Vol. 3, No. 1.

Scleroderma Care and Research Journal: Autumn 2005. This free online PDF medical journal issue includes articles on High-Dose Immunosuppressive Therapy and Autologous Hematopoietic Stem Cell Transplantation for Treatment of Severe Systemic Sclerosis and New Therapies for Pulmonary Arterial Hypertension Associated with Systemic Sclerosis. SCTC Autumn 2005. (Also see: Scleroderma Care and Research Journal, ASTIS Trial and SCOT Trial) Posted 10-17-05.

Bosentan, Tracleer

Digital Ulcers, Pulmonary Fibrosis,
Pulmonary Hypertension (PH), and Skin Fibrosis

Overview of Bosentan
Bosentan for Digital Ulcers
Bosentan for Heart Involvement in SSc
Bosentan for Pulmonary Fibrosis
Bosentan for Pulmonary Hypertension

Bosentan for Raynaud's
Bosentan for Skin Fibrosis
Media Stories about Bosentan
Also See

Bosentan (Tracleer) appears to be beneficial for a variety of scleroderma symptoms, such as digital ulcers, pulmonary fibrosis, pulmonary hypertension, and skin fibrosis. ISN.

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.

Also see: ASTIS Trial, Pulmonary Fibrosis, Stem Cell Transplants.

Imatinib Mesylate (Gleevec)

Northwestern Memorial Study Tests Cancer Drug on Scleroderma Patients. The study is the first of its kind to examine if Gleevec, a cancer drug commonly used to treat leukemia, will treat the skin thickness in scleroderma patients by blocking the pathway that causes fibrosis. The Earth Times. 03/06/08.

A Study Using The Experimental Drug Called Imatinib (Gleevec) in Subjects With Systemic Sclerosis. The purpose of this study is to assess the safety and tolerability of imatinib (gleevec) in subjects who have systemic sclerosis. (UCLA). ClinicalTrials.gov. (Also see: Scleroderma Treatments)

Imatinib in Systemic Sclerosis. This is an open label pilot study to evaluate the safety and efficacy of imatinib in patients with progressive SSc refractory to other treatment(s). Validated measures of skin thickness and disease activity will be determined over 6-months of therapy and compared with baseline measures. (Stanford University). ClinicalTrials.gov. (Also see: Scleroderma Treatments)

Gleevec in the Treatment of Systemic Sclerosis. This is a Phase IIa, single center, prospective open label clinical trial. All patients will be treated with active drug for 12 months. The primary objective of this study will be to determine the safety and tolerability of Gleevec in patients with systemic sclerosis, but important secondary outcomes of relevance will be improvement in disease status as defined by skin scores and indices of pulmonary function. (Hospital for Special Surgery, New York) ClinicalTrials.gov. (Also see: Scleroderma Treatments)

The effect of imatinib (Glivec) on scleroderma and normal dermal fibroblasts: a preclinical study. This study suggests that imatinib can serve as therapy to limit dermal fibroblast proliferation in scleroderma. (PubMed) Dermatology. 2008; 216(2):109-17. (Also see: Skin Involvement)

Pulmonary Fibrosis with Systemic Sclerosis

Open Enrollment, 2008

Recruiting is taking place for a multi-center placebo controlled research study for pulmonary fibrosis with systemic sclerosis sponsored by Novartis Pharmaceuticals. Male and female patients, 18 to 65 years of age may be eligible for participation.

Interested patients can contact the Medical College of Wisconsin (414-456-7019), University of Houston (713-500-7118), UCLA (310-825-4744), Boston University (617-638-4344) or at John Hopkins University (410-550-8582) in Baltimore, Maryland. Novartis Pharmaceuticals. Posted 01-04-08. (Also see: Pulmonary Fibrosis and What is Scleroderma?)

Quinapril for Scleroderma in Bangor, North Wales, UK

Study completed. Quinapril is not effective for scleroderma.

Quinapril Ineffective for Sclerosis Treatment in a Long-Term Study. Long-term administration of the ACE inhibitor quinapril failed to significantly improve symptoms of limited cutaneous systemic sclerosis or reduce the frequency or severity of Raynaud's episodes, compared with placebo. “Based on these findings, we would not recommend ACE inhibitors in limited cutaneous systemic sclerosis.” Expired article, Rheumatology News. Volume 6, Issue 6, Page 3 (June 2007). (Also see:Limited Scleroderma and Raynaud's)

SCOT Trial

Open Enrollment, 2005-2008

SCOT Clinical Trial, which stands for "Scleroderma Cyclophosphamide Or Transplantation", will compare two potential therapies: autologous stem cell transplantation versus high-dose monthly cyclophosphamide. Study dates 2005-2008. (Also see: Cyclophosphamide, Cytoxan and Stem Cell Transfusion)

High Dose Cyclophosphamide (CYC) Without Stem Cell Transplantation in Systemic Sclerosis. High dose CYC without stem cell transplantation leads to rapid improvement of the modified Rodnan skin, HAQ, and PGA scores in the severe early diffuse SSc. Christopher V. Tehlirian. 689 ACR 2006. (Also see: Cytoxan, ASTIS Clinical Trial, and Skin Fibrosis)

Scleroderma Care and Research Journal, Vol. 3, No. 1.

Sildenafil Citrate (Viagra) for Pulmonary Hypertension

Continuing Studies, Positive Results

Surface Logix Commences Phase 2a Clinical Trial of SLx-2101 in Raynaud's Disease. SLx-2101 is a novel, long-acting PDE-5 inhibitor being developed for the management of diseases associated with vascular dysfunction such as Raynaud's. Digital50. Surface Logix Inc. 06/28/07. (Also see: Raynaud's Treatments)

(Also see: Pulmonary Hypertension)

Stem Cell Transplants for Scleroderma

Continuing Studies, Open Entrollment

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

Open Enrollment
Stem Cell Transplantation Centers
Research Studies

Transplant Funding
Media Stories
Patient Stories

Twins and Siblings With and Without Autoimmune Diseases

Open Enrollment (A 5-year study)

NIH Autoimmune Twins and Siblings Study 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 )