Scleroderma: Cyclophosphamide or Transplantation

• Status: Completed
• Trial end date: 2016-04-01
• Target enrollment: 0
• Participant gender: All

Summary

SCOT is a clinical research study designed for people with severe forms of scleroderma. SCOT stands for Scleroderma: Cyclophosphamide Or Transplantation. The SCOT study will compare the potential benefits of stem cell transplant and high-dose monthly cyclophosphamide (Cytoxan) in the treatment of scleroderma.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Collaborator: Rho Federal Systems Division, Inc.
• Treatments: Antilymphocyte Serum; Cyclophosphamide; Immunosuppressive Agents; Methylprednisolone; Methylprednisolone acetate; Methylprednisolone Hemisuccinate; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate

Criteria

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; and

• 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/m^2; 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 or

• 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 Escherichia coli (e.g., E. coli) proteins; History of noncompliance with prior medical care;

• History of substance abuse within 5 years prior to study entry; or

• Pregnancy.