SCOT Scleroderma Treatment Alternative Registry (STAR Registry)
| Status: | Completed |
|---|---|
| Conditions: | Neurology, Dermatology |
| Therapuetic Areas: | Dermatology / Plastic Surgery, Neurology |
| Healthy: | No |
| Age Range: | 18 - 69 |
| Updated: | 4/13/2015 |
| Start Date: | June 2005 |
| End Date: | September 2014 |
| Contact: | Jason Anderson |
| Phone: | 713-500-7126 |
An Observational, Long-Term Follow-up Study of Eligible Individuals Declining To Participate in the Scleroderma Cyclophosphamide or Transplantation (SCOT) Study
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.
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.
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.
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.
Inclusion Criteria:
- No additional inclusion criteria
Exclusion Criteria:
- No additional exclusion criteria
We found this trial at
1
site
Click here to add this to my saved trials
