September 27, 2007
Institutional Review Board, Hospital for Special Surgery
The safety of our participants is our top priority. The trial/study is approved and periodically reviewed by the Institutional Review Board (IRB), which includes doctors, administrators, ethicists, and members of the general public. The safety of clinical trials is reviewed by the U.S. Food and Drug Administration.
Before enrolling in a clinical trial or research study, the investigator will explain the purpose of the trial/study, its expected benefits, any possible risks or side effects, and what your role will be. If you want to join the trial/study, you must sign informed consent documents. You can leave a trial/study at any time without penalty.
For further information, see Understanding Clinical Trials/Research Studies.
This 2-arm clinical trial will enroll approximately 226 subjects with severe SSc in the United States. Subjects will be accrued over a 3-year period and randomly assigned in a 1:1 ratio to a treatment of high-dose immunosuppressive therapy with autologous stem cell rescue (HDIT transplantation) or to treatment with high-dose monthly pulse IV cyclophosphamide for 12 months. The initial treatment period will be approximately 3 months for the HDIT transplantation arm and 12 months for the cyclophosphamide arm. Subjects will be followed up to 44 months after randomization. The study will continue for at least 44 months after the last subject is randomized.
Inclusion Criteria
1. must be between the ages of 18 and 65, inclusive.
2. must have SSc
3. high risk for a fatal outcome based on the following prognostic factors:
Subjects must have (1) both “a” and “b” below and (2) at least one of “c” or “d”.
a. Diffuse cutaneous scleroderma with mRSS score of ≥ 16 verified by the same physician on 2 separate occasions ≥ 1 day and < 28 days apart.
b. Duration of SSc ≤ 4 years from the onset of first non-Raynaud’s symptom.
c. Presence of SSc-related pulmonary disease
d. History of SSc-related renal disease that is not active at the time of screening.
Subjects who present with solely skin and renal disease in the absence of other organ involvement, except classic SSc renal crisis and including nonhypertensive renal crisis, must see a nephrologist to confirm that their renal disease is secondary to only SSc.
Note: Subjects may be re-screened if they fail to meet inclusion criteria on initial evaluation.
Exclusion Criteria
Any of the following exclusions will disqualify enrollment in the study:
1. Subjects with pulmonary, cardiac, hepatic, or renal impairment that would limit their ability to receive cytoreductive therapy and compromise their survival.
2. Active Gastric Antral Vascular Ectasia (GAVE, “watermelon stomach”)
3. Previous treatment with cyclophosphamide
4. Steroid therapy
5. A history or presence of overlap syndrome
6. Active uncontrolled infection, positive Hepatitis B, Hepatitis C and/or HIV
7. Diagnosis of any malignancy within the previous 2 years
8. Pregnancy.
9. History of substance abuse within the last 5 years.
Stacey Kloiber, RN
212.774.7194
kloibers@hss.edu