NCT01413100

Scleroderma Treatment With Autologous Transplant (STAT) Study

A Phase II Multi-center Study of High-Dose Cyclophosphamide and Antithymocyte Globulin Followed by Autologous Hematopoietic Cell Transplantation With Post Transplant Maintenance for the Treatment of Systemic Sclerosis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Systemic Scleroderma

Interventions

Type	Name	Description
BIOLOGICAL	Anti-Thymocyte Globulin	Given IV
PROCEDURE	Autologous Hematopoietic Stem Cell Transplantation	Undergo autologous PBSCT
DRUG	Cyclophosphamide	Given IV
BIOLOGICAL	Filgrastim	Given SC
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Mycophenolate Mofetil	Given PO
PROCEDURE	Peripheral Blood Stem Cell Transplantation	Undergo autologous PBSCT
DRUG	Plerixafor	Given SC
OTHER	Quality-of-Life Assessment	Ancillary studies
OTHER	Questionnaire Administration	Ancillary studies

Timeline

• Start date: 2011-09-15
• Primary completion: 2023-09-15
• Completion: 2024-09-11
• First posted: 2011-08-10
• Last updated: 2025-01-07
• Results posted: 2025-01-07

Locations

12 sites across 1 country: United States