Now Recruiting:

Safety and Value of Self Bone Marrow Transplants Following Chemotherapy in Scleroderma Patients
This study is for patients with severe scleroderma and tests the benefit of stem cell transplantation post chemotherapy. Special medications will be used in chemotherapy to encourage blood cell precursors to multiply and move from the bone marrow to the bloodstream. These stem cells can be transplanted back into the patient's body after chemotherapy.

Phase I, dose escalation study
Enrollment Status: Recruiting
Criteria for Inclusion: 18-70 years, male or female, ECOG performance status of 0 to 2, willing to stay in Pittsburgh for 100 days post transplantation; willing to use contraception, willing to participate in follow ups and other studies.
Study Size: 15
Location: UPMC Hillman Cancer Center, Pittsburgh
Principal Investigator: UPMC Health System
Contact: Amy Schmotzer, RN, 412 647-6205 schmotzerar@upmc.edu

Prospective Assessment of Clinical and Biological Factors Determining Outcomes in Patients with Chronic Graft vs. Host Disease (GVHD)
GVHD is a complication of stem cell transplants, where T cells attack health tissues. The objective of this study is to generate hypotheses by studying the pathogenesis and natural history of the disease. Patients will undergo a thorough multi-disciplinary evaluation by a GVHD clinical team, including assessment and treatment recommendations

Enrollment Status: Recruiting
Criteria for Inclusion: Male and female
Age 1-75 if referred by the primary transplant physician for evaluation of chronic GVHD.
Study Size: 170
Location: National Cancer Institute, Bethesda, Md.
Sponsor: NIH/NCI
Contact: Patient Recruitment and Public Liaison Office, 800 411-1222 prpl@mail.cc.nih.gov

Scleroderma Registry
This registry will conduct genetic analyses for disease-related genes in patients and their family members.  The goal is to identify genes that influence disease susceptibility and to establish a repository of DNA, plasma and serum samples.

Study Design: Retrospective/prospective, natural history
Criteria for Inclusion:  Diagnosis of systemic sclerosis or family member of patient with systemic form.
Enrollment Status: Recruiting
Study Size: 5000
PI: Maureen Mayes, MD, MPH, University of Texas
Contact: Marilyn Perry, 713-500-7162; marilyn.perry@uth.tmc.edu
Sponsor: NIAMS
Study ID: NIAMS-108

National Registry for Childhood Onset Scleroderma
This project will establish a registry of patients with childhood onset scleroderma, for the purposes of studying serum antinuclear antibody (ANA) profiles and their association with clinical and laboratory features of disease and to stimulate further research.

Study participation can be completed through the mail. One blood sample and yearly written surveys for participants and their physicians will be required.
Status: Recruiting
(ANA testing and data analysis will begin after 200 subjects have been enrolled)
Sponsor: Scleroderma Foundation
PI: Thomas A. Medsger, MD, University of Pittsburg School of Medicine, 412-383-8765
Contact: Jennifer Jablon, Registry Coordinator, 412-383-8674; email jablonj@msx.dept-med.pitt.edu


Evaluation and Treatment of Patients with Dermatologic Diseases
The study follows the natural history of connective tissue disease.  Patients will be evaluated and treated according to standard procedures.

Enrollment Status: Recruiting
Criteria for Inclusion: Any age with scleroderma and other dermatologic diseases.  Not pregnant or cognitively impaired.
Study Size: 400
Location: Bethesda
PI: National Cancer Institute
Study ID: 960102; 96-C-0102
Contact: NCI 888 624-1937, ncicssc@mail.nih.gov

Immune System Related Kidney Disease
This is an observational study of patients with known kidney disease or potential for kidney disease who also have an immune disorder such as scleroderma. The purpose is to better understand the causes, signs, symptoms and response to medication. 

Enrollment Status: Recruiting
Criteria for Inclusion: Immune mediated kidney disease or potential disease.  May not have other medical problems that will confuse the results.
Study size: 9999
Location: Bethesda
PI: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Contact: 800-411-1222 prpl@mail.cc.nih.gov

Genetic Study of the FBN1 Gene and Fibrillin-1 Abnormalities in Choctaw Native Americans and Other Patients with Systemic Sclerosis
Patients provide tissue samples via punch skin biopsy from the upper arm or back. The tissue samples will be used for studies of fibrillin-1 synthesis and cellular processing, and FBN1 mutational analysis, linkage analysis and genetic mapping.

Study Type: Observational
Study Design: Screening
Status: Recruiting
Eligibility criteria: All major ethnic groups (Caucasian, Hispanic, African American), diagnosed with systemic sclerosis in accordance with American College of Rheumatology preliminary criteria; positive for systemic scleroderma antibodies
Study Size: 80
PI: Filemon Tan, MD, 713-500-6892
Sponsor: National Center for Research Resources, University of Texas
Location and Contact: University of Texas-Houston Medical School, Houston, TX  77030
Recruiting: Filemon Tan, MD,  713-500-6892

Studies of Families with Twins or Siblings with Discordant Rheumatic Disorders
This study examines families in which one sibling or one twin has a systemic rheumatic disesase and one does not. It will evaluate the differences in blood cell metabolism, cell types in the blood and environmental exposures or genetic factors that might explain why one is diseased and one disease-free.

Enrollment Status: Recruiting
Criteria for Inclusion: Twin pair or sibling pair, one of which has scleroderma. Must have same biological parents. Cannot have active infection needing therapy .  No antibiotics, severe trauma or vaccinations within 8 weeks of start.
Study size: 1650
Location: Bethesda
PI: National Institute of Environmental Health Sciences (NIEHS)
Contact: 800 411-1222  prpl@mail.cc.nih.gov

Safety and Efficacy of Oral Bosentan on Healing/Prevention of Finger Ulcers  (RAPIDS-2)
This is a test of a marketed drug, Bosentan (Tracleer), that is approved for pulmonary arterial hypertension in a new use, digital ulcers.  An earlier trial, RAPIDS-1, showed that Bosentan significantly reduced the number of new digital ulcers versus placebo.  This trial increases the treatment period from 16 weeks to 24 weeks and continues to test safety and efficacy.

Study Design: Randomized, double-blind, placebo controlled
Phase III: Safety, efficacy
Criteria for Inclusion: Systemic sclerosis, diffuse or limited, with at least one digital ulcer. Cannot have severe pulmonary arterial hypertension.
Study size: 180
Location: 20 US centers; 5 Canadian centers
PI: Actelion Pharmaceuticals
Contact: See www.sctc-online.org/studies/rapids2.htm for center contacts
Study ID: RAPIDS-2/AC-052-331

Safety and efficacy of Pletal (cilostazol) for the treatment of juvenile primary and secondary Raynaud’s phenomenon
The study will evaluate the safety and effectiveness of the drug Pletal (cilostazol) in reducing the severity of symptoms and the number of secondary Raynaud’s episodes in juvenile patients. Children with secondary Raynaud’s phenomenon have an underlying condition such as scleroderma or systemic lupus.

Study Design: Randomized, Double-Blind, Placebo Control, Parallel
Status: Recruiting
Eligibility Criteria: 5 to 16 years old; both genders; meet diagnostic criteria for primary or secondary Raynaud’s phenomenon
Location: Multicenter
PI: Otsuka America Pharmaceutical, 2440 Research Blvd., Rockville, MD 20850, 301-417-0900

Six month study for patients with moderate to severe dry eye syndrome
The study will evaluate the effectiveness of a medication to treat dry eye syndrome in patients diagnosed with moderate to severe dry eye syndrome, an autoimmune condition, and/or females 65 years or older.

Enrollment Status: Recruiting 
Criteria for Inclusion: 18 years or older; diagnosed with moderate to severe dry eye syndrome for at least 6 months, and one or all of the following: diagnosed with an autoimmune disorder, female 65 years or older   
Study size: 270 
Location: 13 states. See info@dryeyestudy.com for listing
PI: Allergan
Contact: Rheumatology Research International 1-888-297-4247; info@dryeyestudy.com 

A Transition Study from Flolan to Remodulin (treprostinil sodium) in Patients with Pulmonary Arterial Hypertension
The trial will evaluate patients transitioned off the drug Flolan and administered the subcutaneous study drug (Remodulin or placebo). This is a three-part study consisting of screening, baseline and treatment phases.

Study Design: Treatment, Randomized, Double-Blind, Placebo Control, Parallel
Phase IV, Safety and efficacy
Status: Recruiting
Eligibility Criteria: 18-75 years of age; current confirmed diagnosis of WHO Functional Class II or III pulmonary arterial hypertension (either PPH or PAH associated with the scleroderma spectrum of diseases); clinically stable with regard to PAH for at least 30 days; baseline 6-minute walk distance of at least 250 meters; receiving Flolan treatment for at least 6 months with documented clinical benefit from Flolan treatment on an exercise assessment; other.
Location: Multicenter
Sponsor: United Therapeutics, 1110 Spring St., Silver Spring, MD 20910, 301-608-9292

Autologous Stem Cell Transplant
This study provides an infusion of stem cells to patients to mitigate disease progression. Includes chemotherapy and treatment with immunosuppressive drug. Requires hospitalization and outpatient participation. Patients must stay in Pittsburgh for the duration and will need to return 3 times after the treatment.

Study Design: Non randomized, open label, uncontrolled
Phase I
Enrollment Status: Recruiting
Criteria for Inclusion: Adequate organ function, not pregnant.  Not on immunosuppressives within the last 4 weeks except for steroids; cannot have active infections, HIV or Hepatitis C.  Age 18-70
Study size: 15
Location: Pittsburgh
PI: Andrew Yeager, MD, University of Pittsburgh Medical Center
Contact: Carol Blair, 412 383-8672; BlairC@msx.dept-med.pitt.edu
Sponsor: NIAMS, University of Pittsburgh Cancer Institute, Amgen, Sangstat Medical Corp.
Study ID: NIAMS-047/ N01 AR-9-2239

ASTIS Trial: Autologous Stem Cell Transplantation International Scleroderma Trial
The ASTIS Trial is an international clinical study for patients suffering from severe, progressive systemic sclerosis. The aim of the study is to compare two treatments with respect to their beneficial effect on the disease course as well as their safety: high dose immunoablation followed by autologous stem cell transplantation (considered the investigative treatment) versus pulse-therapy cyclophosphamide (considered the control treatment).

Study Design: Prospective, Randomized
Phase III, Safety and efficacy
Enrollment Status: Recruiting
Eligibility Criteria: Age 16-60 years; diagnosis of systemic scleroderma (not limited to face and hands); clinically important involvement of heart, lungs or kidneys as assessed by specific investigations;  disease duration 4 years or less; acceptable mental and physical condition as determined by extensive screening investigations
Location: Multicenter, Europe, U.S., see www.astistrial.com for listing
Contact: Study administration: Felix-Platter Spital, Rheumatology University  Hospital Clinic, Basel, Switzerland; email: astistrial-fps@unibas.ch
In the U.S.: Dr K.K. Fields, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL, 813-979-7227; email: garretdl@moffitt.usf.edu

Total Body Irradiation in combination with Cyclophosphamide, anti-thymocyte Globulin and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children with Refractory Autoimmune Disorders
This study’s objective is to determine the safety, effectiveness, and long-term complications of this combination therapy that includes stem cell transplants in children with scleroderma and other diseases. Involves collection of stem cells, total body irradiation, chemotherapy, and stem cell transplantation. 10-year follow-up.

Study Design: Multicenter, safety and efficacy. 
Enrollment Status: Recruiting
Criteria for Inclusion: 2-18 years, scleroderma diagnosis; failed standard therapy or standard therapy too toxic; no serious CNS damage.
Study Size:  20
Location: Seattle
PI: Ann Woolfrey, Fred Hutchinson Cancer Center
Study ID: 199/15575; FHCRC-1353.00
Contact: Ann Woolfrey 206 667-4453

Psychological treatments for scleroderma.
This trial will study the effectiveness of two different psychological treatment approaches for people who are living with scleroderma: cognitive behavior therapy and disease/health education.  It will focus on quality of life for patients and determine whether self-help interventions can be used to manage pain, depression and feelings about disfigurement.

Study Design: Randomized, open-label, controlled
Phase II: Efficacy
Criteria for Inclusion: CREST or systemic scleroderma diagnosis
Levels of pain, depression, concern with appearance determined by testing, ages 18-85
Enrollment Status: Recruiting.
Study Size: 201
Principal Investigator (PI):  Jennifer Haythornthwaite, Johns Hopkins Bayview Medical Center, Baltimore
Location: Baltimore
Contact for Recruitment: Keya Medley, 410-614-3396, Ext. 1
Sponsor: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Study ID: NIAMS-056

Efficacy and safety of oral bosentan in pulmonary fibrosis associated with scleroderma
The study will investigate the safety, effectiveness, and tolerability of oral bosentan as a possible treatment for interstitial lung disease associated with systemic sclerosis. Bosentan is currently approved for the treatment of symptoms of pulmonary arterial hypertension.

Study Design: Randomized, Double-Blind, Placebo Control, Parallel
Status: Recruiting
Eligibility Criteria: Diffuse or limited systemic sclerosis
Significant interstitial lung disease on HCRT scan
Location: Multi-center, national/international. See www.build-2.com for listing.
PI/Sponsor: Actelion Pharmaceutical
Contact: See www.build-2.com

Safety and Efficacy of Sitaxsentan Sodium (Thelin) in Patients with Pulmonary Arterial Hypertension
The study will evaluate the safety and effectiveness of Thelin (sitaxsentan sodium) compared to placebo in the treatment of patients with pulmonary arterial hypertension (PAH). A group of patients will be randomly selected to receive standard treatment with Tracleer to compare safety and effectiveness with Thelin.

Phase III, Safety and efficacy
Study Design: Randomized, Double-Blind, Placebo-Controlled with an Open-label Bosentan Arm
Status: Recruiting
Eligibility Criteria: Current diagnosis of symptomatic PAH classified by one of the following: primary pulmonary hypertension (PPH), PAH associated with connective tissue diseases; or PAH associated with the following congenital heart defects: 1. repaired ASD, VSD, or PDA greater than one year post-operative, 2. unrepaired secundum ASD (with resting oxygen saturation greater than 88% in room air measured by oximeter), other
Study size: 240
PI: Encysive Pharmaceuticals
Location and Contact Information: Encysive Pharmaceuticals, Houston, TX, 77401
Recruiting: Pablo Garces, MD, 713-578-6500
PharmaTech Solutions 1-866-332-3275

Stem Cell Transplant to Treat Patients with Systemic Sclerosis
The study will investigate whether stem cell transplantation can modify the progression of scleroderma. The protocol involves stimulating the bone marrow to make extra stem cells which will be collected and stored. The patient will receive high dose chemotherapy to destroy the immune system. Selected stem cells that were collected will be introduced back into the patient, thus recreating the immune system but without the components that cause systemic sclerosis.

Phase I, Safety, efficacy
Study Design: Non-randomized, Open Label, Active Control, Single Group
Status: Recruiting
Eligibility Criteria: Patients <60 years; must have either one major or two minor criteria for systemic sclerosis; rapidly progressing diffuse skin disease; internal organ involvement, other.
Sponsor: Baylor College of Medicine, The Methodist Hospital
Location and Contact information: The Methodist Hospital, Houston, Texas, 77030
PI: Malcolm K. Brenner, MB, PhD, 832-824-4668, mbrenner@bcm.tmc.edu