Stem Cell Transplant to Treat Patients With Systemic Sclerosis
Condition: Systemic Sclerosis
MeSH terms: Scleroderma, Systemic; Scleroderma, Diffuse; Sclerosis
Study type: Interventional
Phase: Phase 1

Summary: 
      Systemic Sclerosis is a disease that may be caused by the immune system reacting against skin
      and certain organs. It is possible, that by changing the immune system we can modify the
      progression of this disease.

      Stem cells are created in the bone marrow. They mature into different types of blood cells
      that are needed including red blood cells, white blood cells, and platelets. In this study,
      we will stimulate the bone marrow to make extra stem cells. Next we will collect the stem
      cells, select specific cells, and store them. We will then give high dose chemotherapy that
      will destroy the patients immune system. We will then give back the selected stem cells we
      collected. We believe that these selected stem cells may be able to "re-create" the immune
      system without the portion that causes Systemic Sclerosis.

      The purpose of this study is to try to discover if stem cell transplantation can help
      patients with Systemic Sclerosis. We will also try to learn what the side effects are of this
      treatment in patients with Systemic Sclerosis. We hope that this treatment will help to
      relieve the symptoms patients are experiencing, although we do not know if it will.
    

Detailed description: 
      Before the transplant the research participant will receive daily G-CSF (Neupogen) for 5-6
      days. This medication will help to stimulate the production of white blood cells (WBC) that
      will be used for the stem cell transplant. The G-CSF will be given as an injection into the
      arm.

      If G-CSF does not stimulate the stem cells sufficiently, the patient will receive a single
      dose of drug called cyclophosphamide (chemotherapy) intravenously (into a vein). This drug
      will cause the blood cell counts to fall. A drug called MESNA will also be given to help
      protect the bladder from the Cyclophosphamide. After completing chemotherapy, patients will
      be started on G-CSF again until blood cell counts reach a certain level, at which time the
      patient will undergo leukopheresis. Leukopheresis is a procedure where blood is removed from
      one arm, pumped into a machine where the white blood cells are separated from most of the
      other cells and then returned through the same needle or through a needle in the other arm.
      This procedure usually takes 3 to 4 hours a day for up to 4 days in a row, depending on how
      many cells are collected each time.

      After collection of the white blood cells, special agents (called monoclonal antibodies) will
      be used in the laboratory to select out certain types of white blood cells (CD34+ cells). The
      blood cells will be separated on a machine which picks out stem cells.

      After leukopheresis, patients will receive drugs called cyclophosphamide and Mesna. They will
      also receive a drug called Atgam and radiation treatment to the entire body. This treatment
      will kill most of the blood forming cells in the bone marrow. We will then give the CD34+
      cells that were collected during leukopheresis.

      After the transplant patients will be followed closely, the same as any patient who receives
      a stem cell transplant. This follow-up will involve blood tests to see how the body is
      recovering after the chemotherapy and radiation, and a bone marrow aspiration once a year for
      2 years.
    

Intervention name(s): Cyclophosphamide; Mesna; G-CSF; Leukopheresis; Total Body Irradiation
Intervention type(s): Drug; Drug; Drug; Procedure; Procedure
Intervention MeSH terms: Cyclophosphamide
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment