SCOT Scleroderma Treatment Alternative Registry (STAR Registry)

NCT00860548 | Completed DMC

• 2 other identifiers: DAIT SCSSc-02SCOT
• Study Type: observational
• Target: 19
• Locations: 1 country
• Sites: 1

Brief Summary

The Scleroderma Cyclophosphamide Or Transplant (SCOT) Trial is a Phase II/III interventional trial comparing two treatments for early, severe scleroderma. These two interventions are high dose immunosuppressive therapy followed by autologous stem cell transplantation and monthly high dose pulse cyclophosphamide (the later for 12 doses). While standard of care might be considered the optimal control arm for a trial such as this one, no such standard of care is available for the population of scleroderma patients defined by the eligibility criteria for this trial. The rheumatologists on the protocol team believe that the SCOT cyclophosphamide regimen represents the best control arm for this study. However, given concerns over use of a treatment arm as a control that has not been established as a standard of care, this registry was established. The registry will be a prospective, observational study of subjects with severe systemic sclerosis (SSc) who are eligible to participate in the Scleroderma Cyclophosphamide or Transplantation (SCOT) Study but are denied insurance coverage or decline to participate prior to randomization. Subjects will be accrued over the same period as the SCOT study. Subjects will follow the course of treatment prescribed by their treating physician with no interference from the registry. The primary purpose of this study is to document the disease course and outcome in a group of participants who are eligible for the SCOT study, but declined to participate, in order to determine whether their outcome is better, worse, or no different than those who participate in the treatment phase of the trial.

Conditions

Scleroderma, Systemic; Sclerosis

Outcome Measures

Primary Outcomes (1)

• Event-free survival (EFS)

  The events will be defined as any one of the following: * Death. * Respiratory failure defined as the need for supplementary oxygen; or * Renal failure, as defined by chronic dialysis \> 6 months or renal transplantation.

  44 months after subject enrollment

Secondary Outcomes (7)

• Functional status as determined by the Modified Scleroderma Health Assessment Questionnaire (m-HAQ/S-HAQ)

  44 months after subject enrollment

• Mortality due to any cause

  44 months after subject enrollment

• Medical therapies and procedures (including hospitalizations)

  44 months after subject enrollment

• Diagnosis and treatment for pulmonary hypertension

  44 months after subject enrollment

• Need for hyperalimentation

  44 months after subject enrollment

Interventions

currently available therapy in the community DRUG

Participants will receive telephone calls every 3 months for approximately 44 months for the purpose of outcome surveys

Eligibility Criteria

• Age: 18 Years - 69 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants with severe systemic sclerosis who are eligible for the SCOT study, but either decline to participate or are denied insurance coverage, prior to randomization will be invited to participate in this registry.

Sponsors & Collaborators

• National Institute of Allergy and Infectious Diseases (NIAID): lead

Study Sites (1)

University of Texas, Houston Medical School

Houston, Texas, 77030, United States

Location

Related Publications (6)

• Czirjak L, Nagy Z, Szegedi G. Survival analysis of 118 patients with systemic sclerosis. J Intern Med. 1993 Sep;234(3):335-7. doi: 10.1111/j.1365-2796.1993.tb00753.x.

  PMID: 8354987 BACKGROUND

• Bulpitt KJ, Clements PJ, Lachenbruch PA, Paulus HE, Peter JB, Agopian MS, Singer JZ, Steen VD, Clegg DO, Ziminski CM, Alarcon GS, Luggen ME, Polisson RP, Willkens RF, Reading JC, Williams HJ, Ward JR. Early undifferentiated connective tissue disease: III. Outcome and prognostic indicators in early scleroderma (systemic sclerosis). Ann Intern Med. 1993 Apr 15;118(8):602-9. doi: 10.7326/0003-4819-118-8-199304150-00005.

  PMID: 8452326 BACKGROUND

• Follansbee WP, Zerbe TR, Medsger TA Jr. Cardiac and skeletal muscle disease in systemic sclerosis (scleroderma): a high risk association. Am Heart J. 1993 Jan;125(1):194-203. doi: 10.1016/0002-8703(93)90075-k.

  PMID: 8417518 BACKGROUND

• Lee P, Langevitz P, Alderdice CA, Aubrey M, Baer PA, Baron M, Buskila D, Dutz JP, Khostanteen I, Piper S, et al. Mortality in systemic sclerosis (scleroderma). Q J Med. 1992 Feb;82(298):139-48.

  PMID: 1620814 BACKGROUND

• Steen VD, Mayes MD, Merkel PA. Assessment of kidney involvement. Clin Exp Rheumatol. 2003;21(3 Suppl 29):S29-31.

  PMID: 12889219 BACKGROUND

• Clements PJ, Wong WK, Hurwitz EL, Furst DE, Mayes M, White B, Wigley F, Weisman M, Barr W, Moreland L, Medsger TA Jr, Steen V, Martin R, Collier D, Weinstein A, Lally E, Varga J, Weiner S, Andrews B, Abeles M, Seibold J. Correlates of the disability index of the health assessment questionnaire: a measure of functional impairment in systemic sclerosis. Arthritis Rheum. 1999 Nov;42(11):2372-80. doi: 10.1002/1529-0131(199911)42:113.0.CO;2-J.

  PMID: 10555033 BACKGROUND

Related Links

• National Institute of Allergy and Infectious Diseases (NIAID)
• Division of Allergy, Immunology, and Transplantation (DAIT)
• Scleroderma: Cyclophosphamide Or Transplantation (SCOT) Study

MeSH Terms

Conditions

Scleroderma, Systemic; Sclerosis

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Maureen Mayes, MD, MPH

  The University of Texas Health Science Center, Houston

  STUDY CHAIR

• Daniel Furst, MD

  UCLA Medical School

  STUDY CHAIR

• Peter McSweeney, MD

  Blood and Marrow Transplant Program, Presbyterian/St. Luke's Medical Center, Rocky Mountain Cancer Center

  STUDY CHAIR

• Leslie J. Crofford, MD

  University of Michigan

  STUDY CHAIR

• Richard Nash, MD

  Fred Hutchinson Cancer Center

  STUDY CHAIR

• Keith Sullivan, MD

  Division of Cellular Therapy, Duke University

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 11, 2009
• First Posted: March 12, 2009
• Study Start: June 1, 2005
• Primary Completion: March 1, 2016
• Study Completion: March 1, 2016
• Last Updated: April 5, 2017

Record last verified: 2017-04

Locations

US (1)