Scleroderma, SystemicNCT00860548
SCOT Scleroderma Treatment Alternative Registry (STAR Registry)
An observational study of Scleroderma, Systemic and Sclerosis, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 69 years
- Conditions the study is for
- Scleroderma, Systemic, Sclerosis
- What is being tested?
- currently available therapy in the community
- How many people
- 19 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2005to Mar 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 69 years
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- No additional inclusion criteria
Who cannot join
- No additional exclusion criteria
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas, Houston Medical School
Houston, Texas, 77030
About this study
Official title: An Observational, Long-term Follow-up Study of Eligible Individuals Declining To Participate in the Scleroderma Cyclophosphamide or Transplantation (SCOT) Study (SCSSc-02)
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.
Read the 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
- Being tested
- currently available therapy in the community
- Medical subject headings
- Scleroderma, Systemic, Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
currently available therapy in the communityDrug
Participants will receive telephone calls every 3 months for approximately 44 months for the purpose of outcome surveys
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 44 months after subject enrollment
Secondary outcomes (7)
Functional status as determined by the Modified Scleroderma Health Assessment Questionnaire (m-HAQ/S-HAQ)
Measured: 44 months after subject enrollment
Mortality due to any cause
Measured: 44 months after subject enrollment
Medical therapies and procedures (including hospitalizations)
Measured: 44 months after subject enrollment
Diagnosis and treatment for pulmonary hypertension
Measured: 44 months after subject enrollment
Need for hyperalimentation
Measured: 44 months after subject enrollment
Amputation whether surgical or auto-amputation
Measured: 44 months after subject enrollment
Hospitalization or surgery
Measured: 44 months after subject enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2009 (estimated)
- Study startJun 2005
- Primary completionMar 2016
- Study completionMar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Scleroderma, SystemicStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Maureen Mayes, MD, MPH, Study chair, The University of Texas Health Science Center, HoustonDaniel Furst, MD, Study chair, UCLA Medical SchoolPeter McSweeney, MD, Study chair, Blood and Marrow Transplant Program, Presbyterian/St. Luke's Medical Center, Rocky Mountain Cancer CenterLeslie J. Crofford, MD, Study chair, University of MichiganRichard Nash, MD, Study chair, Fred Hutchinson Cancer CenterKeith Sullivan, MD, Study chair, Division of Cellular Therapy, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00860548
- Sponsor's protocol ID
- DAIT SCSSc-02
- Other IDs
- SCOT
- Status last verified
- Apr 2017
- First submitted
- 11 Mar 2009
- Last updated
- 5 Apr 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 8354987Bulpitt 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.
PubMed 8452326Follansbee 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.
PubMed 8417518Lee 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.
PubMed 1620814Steen VD, Mayes MD, Merkel PA. Assessment of kidney involvement. Clin Exp Rheumatol. 2003;21(3 Suppl 29):S29-31.
PubMed 12889219Clements 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.
PubMed 10555033
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational, Long-term Follow-up Study of Eligible Individuals Declining To Participate in the Scleroderma Cyclophosphamide or Transplantation (SCOT) Study (SCSSc-02)
- ClinicalTrials.gov ID
- NCT00860548
- Acronym / sponsor ID
- DAIT SCSSc-02
- Conditions
- Scleroderma, Systemic, Sclerosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- Jun 2005
- Primary completion
- Mar 2016 (actual)
- Study completion
- Mar 2016 (actual)
- First posted
- 12 Mar 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 69 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (1 listed)
- No additional inclusion criteria
Key exclusion criteria (1 listed)
- No additional exclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| currently available therapy in the communityParticipants will receive telephone calls every 3 months for approximately 44 months for the purpose of outcome surveys | Drug | – |
Primary outcome
Event-free survival (EFS)
Time frame: 44 months after subject enrollment
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.
7 secondary outcomes
Functional status as determined by the Modified Scleroderma Health Assessment Questionnaire (m-HAQ/S-HAQ)
Time frame: 44 months after subject enrollment
Mortality due to any cause
Time frame: 44 months after subject enrollment
Medical therapies and procedures (including hospitalizations)
Time frame: 44 months after subject enrollment
Diagnosis and treatment for pulmonary hypertension
Time frame: 44 months after subject enrollment
Need for hyperalimentation
Time frame: 44 months after subject enrollment
Amputation whether surgical or auto-amputation
Time frame: 44 months after subject enrollment
Hospitalization or surgery
Time frame: 44 months after subject enrollment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2017.
- Background: PubMed 8354987Czirjak 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.
- Background: PubMed 8452326Bulpitt 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.
- Background: PubMed 8417518Follansbee 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.
- Background: PubMed 1620814Lee 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.
- Background: PubMed 12889219Steen VD, Mayes MD, Merkel PA. Assessment of kidney involvement. Clin Exp Rheumatol. 2003;21(3 Suppl 29):S29-31.
- Background: PubMed 10555033Clements 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.
- National Institute of Allergy and Infectious Diseases (NIAID)Link provided by the sponsor.
- Division of Allergy, Immunology, and Transplantation (DAIT)Link provided by the sponsor.
- Scleroderma: Cyclophosphamide Or Transplantation (SCOT) StudyLink provided by the sponsor.
Related trials for Scleroderma, Systemic
RecruitingPhase 1
A Trial in Healthy Adult Participants and Adults With Autoimmune Diseases to Test How HBM7020 is Tolerated and How it Behaves in the Body
Rheumatoid Arthritis, Systemic Lupus Erythematosus
Active, not recruitingPhase 2
A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With Progressive Pulmonary Fibrosis
Pulmonary Fibrosis, Progressive Pulmonary Fibrosis
RecruitingObservational study
Quantification of Calcinosis Cutis Disease Burden Using Computed Tomography Images
Systemic Sclerosis (SSc), Calcinosis Cutis
Not yet recruitingPhase 2
Hyaluronidase Injections for Sclerodactyly in Patients With Scleroderma
Scleroderma, Sclerodactyly
RecruitingPhase 1/2
Phase I/II Study of AD-PluReceptor Plus Tafasitamab-cxix and Lymphodepleting Chemotherapy in Patients With Autoimmune Disorders
Autoimmune Disorders, Systemic Sclerosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Maureen Mayes, MD, MPH study chair
The University of Texas Health Science Center, Houston
Daniel Furst, MD study chair
UCLA Medical School
Peter McSweeney, MD study chair
Blood and Marrow Transplant Program, Presbyterian/St. Luke's Medical Center, Rocky Mountain Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Scleroderma, Systemic and Sclerosis, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event-free survival (EFS) (time frame: 44 months after subject enrollment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00860548, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Apr 2017.