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
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
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 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
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
