SCOT Scleroderma Treatment Alternative Registry (STAR Registry)

NCT ID: NCT00860548

Last Updated: 2017-04-05

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 19 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2005-06-30
• Study Completion Date: 2016-03-31

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.

Detailed Description

For multiple reasons, the SCOT investigators and the sponsor of the SCOT trial, the Division of Allergy, Immunology, and Transplantation (DAIT) of the National Institute of Allergy and Infectious Diseases (NIAID), determined that it is important to track the course of a 'matched' group of patients, who are not exposed to these treatments but receive currently available therapy in the community. First, such a group will provide information to determine if the SCOT entry criteria do indeed identify these high-risk individuals. More importantly, such a group of patients is likely to be treated with a variety of medical regimens, including some immunosuppressive therapy with cyclophosphamide or other immunosuppressive agents that may modify the natural history of the disease. In evaluating the relative efficacy of the two treatment regimens, it will be important to assess whether outcomes in the subjects treated under the SCOT protocol have outcome profiles that differ from those associated with the matched group of patients treated in the community. One readily available group that meets these criteria are those individuals who are otherwise eligible for the SCOT trial but fail to be randomized because they either decline to participate or are denied insurance coverage to receive the SCOT treatment regimens.

The duration of this trial is 44 months. Participants will be enrolled over the same period as the SCOT trial. Participants will follow the course of treatment prescribed by their treating physician with no interference from the registry. All participant contact, including obtainment of informed consent and telephone interview regarding outcome measurements will be performed by SCOT study personnel at the University of Texas, Houston (one of the SCOT transplant centers). Participants will be contacted by phone every 3 months to determine vital status, record medical and other therapy, and administer the modified Scleroderma Health Assessment Questionnaire (S-HAQ). Medical records will be obtained to verify self-reported medical events.

Conditions

Scleroderma, Systemic; Sclerosis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Interventions

currently available therapy in the community

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

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

Inclusion Criteria:

• No additional inclusion criteria

Exclusion Criteria:

• No additional exclusion criteria

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 69 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institute of Allergy and Infectious Diseases (NIAID)

NIH

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Maureen Mayes, MD, MPH

Role: STUDY_CHAIR

The University of Texas Health Science Center, Houston

Daniel Furst, MD

Role: STUDY_CHAIR

UCLA Medical School

Peter McSweeney, MD

Role: STUDY_CHAIR

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

Leslie J. Crofford, MD

Role: STUDY_CHAIR

University of Michigan

Richard Nash, MD

Role: STUDY_CHAIR

Fred Hutchinson Cancer Center

Keith Sullivan, MD

Role: STUDY_CHAIR

Division of Cellular Therapy, Duke University

Locations

University of Texas, Houston Medical School

Houston, Texas, United States

Countries

United States

References

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.

Reference Type: BACKGROUND

PMID: 8354987 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 8452326 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 8417518 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 1620814 (View on PubMed)

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

Reference Type: BACKGROUND

PMID: 12889219 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 10555033 (View on PubMed)

Related Links

https://www.niaid.nih.gov/

National Institute of Allergy and Infectious Diseases (NIAID)

https://www.niaid.nih.gov/about/dait

Division of Allergy, Immunology, and Transplantation (DAIT)

http://www.sclerodermatrial.org/

Scleroderma: Cyclophosphamide Or Transplantation (SCOT) Study

Other Identifiers

SCOT

Identifier Type: -

Identifier Source: secondary_id

DAIT SCSSc-02

Identifier Type: -

Identifier Source: org_study_id