SCOT Scleroderma Treatment Alternative Registry (STAR Registry)

This study is currently recruiting participants.
Verified March 2009 by National Institute of Allergy and Infectious Diseases (NIAID)
Sponsor:
Information provided by:
National Institute of Allergy and Infectious Diseases (NIAID)
ClinicalTrials.gov Identifier:
NCT00860548
First received: March 11, 2009
Last updated: March 26, 2009
Last verified: March 2009
  Purpose

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.


Condition Intervention
Scleroderma, Systemic
Sclerosis
Behavioral: Telephone Call

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: An Observational, Long-Term Follow-up Study of Eligible Individuals Declining To Participate in the Scleroderma Cyclophosphamide or Transplantation (SCOT) Study

Resource links provided by NLM:


Further study details as provided by National Institute of Allergy and Infectious Diseases (NIAID):

Primary Outcome Measures:
  • Death, renal failure requiring dialysis, and pulmonary compromise requiring oxygen, pulmonary hypertension requiring treatment. [ Time Frame: Throughout study ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Medical therapies and procedures (including hospitalizations), scleroderma renal crisis, and functional status as determined by the modified Scleroderma Health Assessment Questionnaire [ Time Frame: Throughout study ] [ Designated as safety issue: No ]

Estimated Enrollment: 45
Study Start Date: June 2005
Estimated Study Completion Date: September 2014
Estimated Primary Completion Date: September 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
1
Participants with severe systemic sclerosis who are eligible for the SCOT study, but decline to participate, will be invited to participate in this sub-study
Behavioral: Telephone Call
Participants will receive telephone calls every 3 months for approximately 44 months for the purpose of outcome surveys

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.

The primary outcomes of interest include: death, renal failure requiring dialysis, and pulmonary compromise requiring oxygen, pulmonary hypertension requiring treatment. In addition the following will be recorded: medical therapies and procedures (including hospitalizations), scleroderma renal crisis, and functional status as determined by the modified Scleroderma Health Assessment Questionnaire.

The primary endpoint for this study, which is designed to be similar to the endpoint for the SCOT study, is event-free survival (EFS) at 44 months after subject enrollment. The events will be defined as any one of the following:

  1. Death.
  2. Respiratory failure defined as the need for supplementary oxygen; or
  3. Renal failure, as defined by chronic dialysis > 6 months or renal transplantation.

The secondary endpoints include:

  1. Mortality
  2. Medical therapy
  3. Occurrence of scleroderma renal crisis with outcome (dialysis requiring or not)
  4. Diagnosis and treatment for pulmonary hypertension;
  5. Need for hyperalimentation;
  6. Amputation whether surgical or auto-amputation
  7. Modified Scleroderma Health Assessment Questionnaire (m-HAQ/S-HAQ);
  8. Hospitalization or surgery.
  Eligibility

Ages Eligible for Study:   18 Years to 69 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

Criteria

Inclusion Criteria:

  • No additional inclusion criteria

Exclusion Criteria:

  • No additional exclusion criteria
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00860548

Contacts
Contact: Jason Anderson 713-500-7126

Locations
United States, Texas
University of Texas, Houston Medical School Recruiting
Houston, Texas, United States
Sponsors and Collaborators
Investigators
Study Chair: 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 Research Center
Study Chair: Keith Sullivan, MD Division of Cellular Therapy, Duke University
  More Information

Publications:
Responsible Party: Associate Director, Clinical Research Program, DAIT/NIAID
ClinicalTrials.gov Identifier: NCT00860548     History of Changes
Other Study ID Numbers: DAIT SCSSc-02, SCOT
Study First Received: March 11, 2009
Last Updated: March 26, 2009
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Scleroderma, Systemic
Scleroderma, Diffuse
Scleroderma, Localized
Sclerosis
Connective Tissue Diseases
Skin Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on April 16, 2014