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A Randomized, Open-Label, Phase II Multicenter Study of High-Dose Immunosuppressive Therapy Using Total Body Irradiation, Cyclophosphamide, ATGAM, and Autologous Transplantation With Auto-CD34+HPC Versus Intravenous Pulse Cyclophosphamide for the Treatment of Severe Systemic Sclerosis (SCSSc-01)


Phase 2/Phase 3
18 Years
69 Years
Open (Enrolling)
Both
Scleroderma, Systemic, Sclerosis, Autoimmune Disease

Thank you

Trial Information

A Randomized, Open-Label, Phase II Multicenter Study of High-Dose Immunosuppressive Therapy Using Total Body Irradiation, Cyclophosphamide, ATGAM, and Autologous Transplantation With Auto-CD34+HPC Versus Intravenous Pulse Cyclophosphamide for the Treatment of Severe Systemic Sclerosis (SCSSc-01)


Inclusion Criteria:



- Severe systemic sclerosis (SSc) as defined by the American College of Rheumatology
(ACR)

- SSc, including extensive skin and internal organ involvement involving either the
lungs or the kidneys, that threatens participant's life

- Willingness to use accepted methods of contraception for at least 15 months after
starting study treatment

Exclusion Criteria:

- Lung, heart, liver, or kidney impairment that would interfere with the study or
compromise participant's survival

- Active blood vessel dilation in the stomach (Active Gastric Antral Vascular
Ectasia/GAVE, also known as "watermelon stomach"). Patients found to have this
disorder at study screening can receive treatment outside the study and then be
re-screened. For more information about this study criterion, refer to the study
protocol.

- Previous treatment with cyclophosphamide, as defined by: a) prior IV cyclophosphamide
administration for more than 6 months OR a total cumulative IV dose greater than 3
g/m2; b) prior oral cyclophosphamide administration for more than 4 months,
regardless of dose; or c) combination of prior oral and IV cyclophosphamide
administration for more than 6 months, independent of dose.

- Steroid therapy at doses of greater than 10 mg/day, or more than 2 pulses for
concurrent illnesses within prior 12 months

- Unwillingness or inability to discontinue certain disease-modifying antirheumatic
drugs (DMARDs) for the treatment of SSc

- Presence of clinically significant rheumatic diseases other than scleroderma
requiring significant immunosuppression

- Any active uncontrolled infection that would interfere with high-dose therapy or
pulse cyclophosphamide regimens

- Hepatitis B virus infected

- Hepatitis C virus infected

- HIV infected

- Blood abnormalities

- Diagnosis of cancer within 2 years prior to study entry. Participants with adequately
treated squamous cell skin cancer, basal cell carcinoma, and carcinoma in situ are
not excluded.

- Other comorbid illnesses with an estimated life expectancy of less than 5 years

- Defective formation of bone marrow cells (myelodysplasia)

- Uncontrolled hypertension

- History of hypersensitivity to murine or E. coli proteins

- History of noncompliance with prior medical care

- History of substance abuse within 5 years prior to study entry

- Pregnancy

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Global rank composite score (GRCS) at 54 months post- randomization.

Outcome Description:

The GRCS reflects each participant's "order" relative to every other participant based on the following hierarchy of component outcomes: death, event-free survival (EFS), forced vital capacity (FVC), Modified Scleroderma Health Questionnaire (SHAQ), and modified Rodnan Skin Score (mRSS).The analysis for the global rank composite score is based on an extension of the Wilcoxon signed-rank test.

Outcome Time Frame:

At 54 Months Post-Randomization

Safety Issue:

Yes

Principal Investigator

Keith Sullivan, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Division of Cellular Therapy, Duke University

Authority:

United States: Food and Drug Administration

Study ID:

DAIT SCSSc-01

NCT ID:

NCT00114530

Start Date:

June 2005

Completion Date:

June 2016

Related Keywords:

  • Scleroderma, Systemic
  • Sclerosis
  • Autoimmune Disease
  • autoimmune disease
  • Autoimmune Diseases
  • Scleroderma, Systemic
  • Scleroderma, Diffuse
  • Scleroderma, Localized
  • Sclerosis

Name

Location

University of MichiganAnn Arbor, Michigan  48109-0624
MD Anderson Cancer CenterHouston, Texas  77030-4096
Washington University School of MedicineSaint Louis, Missouri  63110
Medical University of South CarolinaCharleston, South Carolina  29425-0721
Medical College of WisconsinMilwaukee, Wisconsin  53226
Boston University School of MedicineBoston, Massachusetts  02118
City of Hope National Medical CenterLos Angeles, California  91010
Massachusetts General HospitalBoston, Massachusetts  02114-2617
University of PittsburghPittsburgh, Pennsylvania  15261
Duke UniversityDurham, North Carolina  27710
University of KentuckyLexington, Kentucky  40536-0098
UCLA Medical SchoolLos Angeles, California  90095-1670
University of Toledo Health Science CampusToledo, Ohio  43699
University of Texas-Houston Medical SchoolHouston, Texas  77030
Fred Hutchinson Cancer Research Center (FHCRC)Seattle, Washington  98109