Clinical trial NCT01413100
Conditions: Systemic Scleroderma
Sponsor: Fred Hutchinson Cancer Center
Start date: 2011-09-15; Completion date: 2024-09-11
Enrollment: 21 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
This phase II trial studies how well giving cyclophosphamide and anti-thymocyte globulin together followed by peripheral blood stem cell transplant (PBSCT) and mycophenolate mofetil works in treating patients with systemic scleroderma (SSc). Stem cells are collected from the patient's blood and stored prior to treatment. To store the stem cells patients are given colony-stimulating factors, such as filgrastim (G-CSF) or chemotherapy (cyclophosphamide) to help stem cells move from the bone marrow to the blood so they can be collected and stored. After storage, patients are then given high-dose chemotherapy, cyclophosphamide, and immunosuppression with anti-thymocyte globulin to suppress the immune system to prepare for the transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy and immunosuppression. After the stem cells have "engrafted" and have matured enough to support the immune system at approximately 2-3 months, patients are given a medication called mycophenolate mofetil (MMF) or Myfortic. This medication is given to prevent worsening or reactivation of SSc and is referred to as maintenance therapy.

Detailed description:
OUTLINE:

STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim subcutaneously (SC) on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide intravenously (IV) or \*plerixafor subcutaneously (SC) on mobilization days 1-2 and filgrastim SC on mobilization days 5-7.

HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5.

TRANSPLANTATION: Patients undergo autologous PBSCT on day 0.

MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil orally (PO) twice daily (BID) for 2 years.

NOTE: \*Plerixafor is an alternative to the cyclophosphamide based mobilization.

After completion of study treatment, patients are followed at 1 month, weeks 12 and 26, and then annually for 5 years.

Arms: arm 1: STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells \>= 2.5 x 10\^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or \*plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7.

HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5.

TRANSPLANTATION: Patients undergo autologous PBSCT on day 0.

MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years.
Interventions: intervention 1: Anti-Thymocyte Globulin intervention 2: Autologous Hematopoietic Stem Cell Transplantation intervention 3: Cyclophosphamide intervention 4: Filgrastim intervention 5: Laboratory Biomarker Analysis intervention 6: Mycophenolate Mofetil intervention 7: Peripheral Blood Stem Cell Transplantation intervention 8: Plerixafor intervention 9: Quality-of-Life Assessment intervention 10: Questionnaire Administration
Intervention descriptions: intervention 1: Given IV intervention 2: Undergo autologous PBSCT intervention 3: Given IV intervention 4: Given SC intervention 5: Correlative studies intervention 6: Given PO intervention 7: Undergo autologous PBSCT intervention 8: Given SC intervention 9: Ancillary studies intervention 10: Ancillary studies

Protocol documents:
Prot_SAP_001.pdf: 


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Prot_SAP_000.pdf: 


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