Clinical Trial: Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Non-myeloablative Autologous Hematopoietic Stem Cell Transplantation in Patients With Chronic Inflammatory Demyelinating Polyneuropathy: A Phase II Trial

Brief Summary: Chronic inflammatory demyelinating polyneuropathy is disease believed to be due to immune cells, cells which normally protect the body, but are now attacking the nerves in the body. As a result, the affected nerves fail to respond, or respond only weakly, to stimuli causing numbing, tingling, pain, and progressive muscle weakness.The likelihood of progression of the disease is high. This study is designed to examine whether treating patients with high dose cyclophosphamide (a drug which reduces the function of the immune system) and ATG (a protein that kills the immune cells that are thought to be causing disease), followed by return of the previously collected blood stem cells will stop the progression of CIDP. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the high dose cyclophosphamide and ATG is to destroy the cells in the immune system. The purpose of the stem cell infusion is to evaluate whether this treatment will produce a normal immune system that will no longer attack the body.

Detailed Summary:

Selection of the Regimen for Immunosuppressive Therapy

Cyclophosphamide with ATG is a common conditioning regimen with two decades of experience in the treatment of aplastic anemia, and has been used safely without reported mortality in the treatment of autoimmune diseases such as systemic lupus erythematosus and rheumatoid arthritis.Cy / ATG is not associated with late malignancies or cataracts. Both cyclophosphamide and anti-thymocyte globulin (horse or rabbit ATG) are potent immunosuppressive agents. ATG contributes additional immunosuppression without additional cytotoxicity. ATG given shortly pre-transplant will contribute to the elimination of host T lymphocytes that survive cyclophosphamide SLE, an autoimmune disease responsive to cyclophosphamide, responds well to a CY / ATG conditioning regimen, but we have recently found that patients with either SLE or neuromyelitis optica respond faster and may have more durable remissions to a regimen of "rituxan sandwich" in which rituxan is infused before and after standard cytoxan rATG. For these reasons, "rituxan sandwich" will be the conditioning regimen utilized in this study.

5.2 Method of Harvesting Stem Cells

Based on the experience of the pilot studies, the current protocol will mobilize stem cells with granulocyte-colony stimulating factor (G-CSF) and collect stem cells by apheresis, with subsequent bone marrow harvest performed only if needed to supplement the peripheral blood stem cells (PBSC). Based on experience of autoimmune flares in patients receiving G-CSF alone for mobilization, patients will be mobilized with cyclophosphamide 2.0 g/m2 and G-CSF 5-10 mcg/kg.

5.3 Cyclophosphamide

Cyclophosph
Sponsor: Northwestern University

Current Primary Outcome: Survival;Disease improvement; [ Time Frame: 6 months, 1, 2, 3, 4 and 5 years post transplant ]

Original Primary Outcome:

  • 1) Survival
  • 2) Disease improvement
  • 3) Time to disease progression


Current Secondary Outcome:

Original Secondary Outcome:

Information By: Northwestern University

Dates:
Date Received: January 16, 2006
Date Started: March 2005
Date Completion: December 2018
Last Updated: March 9, 2017
Last Verified: March 2017