NCT00716066

Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases

High-Dose Immunosuppressive Therapy Using Carmustine, Etoposide, Cytarabine, and Melphalan (BEAM) + Thymoglobulin Followed by Syngeneic or Autologous Hematopoietic Cell Transplantation for Patients With Autoimmune Neurologic Diseases

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 53 (actual)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 71 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies the side effects and how well carmustine, etoposide, cytarabine and melphalan together with antithymocyte globulin before a stem cell transplant works in treating patients with autoimmune neurologic disease that did not respond to previous therapy. In autoimmune neurological diseases, the patient's own immune system 'attacks' the nervous system which might include the brain/spinal cord and/or the peripheral nerves. Giving high-dose chemotherapy, including carmustine, etoposide, cytarabine, melphalan, and antithymocyte globulin, before a stem cell transplant weakens the immune system and may help stop the immune system from 'attacking' a patient's nervous system. When the patient's own (autologous) stem cells are infused into the patient they help the bone marrow make red blood cells, white blood cells, and platelets so the blood counts can improve.

Detailed description

OUTLINE: Patients receive carmustine intravenously (IV) on day -6, etoposide IV and cytarabine IV twice daily (BID) on days -5 to -2, melphalan IV on day -1, and antithymocyte globulin IV on days -2 and -1. Patients then undergo autologous or syngeneic stem cell transplant on day 0. Patients also receive prednisone orally (PO) once daily (QD) on days 7-21, followed by 2 week taper. After completion of study treatment, patients are followed up at 3 months, 1 year, and then annually thereafter for up to 5 years.

Conditions

• Autoimmune Disease
• Neurologic Autoimmune Disease
• Autologous Transplant Autoimmune
• Multiple Sclerosis Transplant
• MS Stem Cell Transplant
• Multiple Sclerosis Stem Cell Transplant
• Stiff Person Syndrome
• HCT for Neurologic Autoimmune Disorders
• CIDP Transplant
• Myasthenia Gravis Transplant
• Autoimmune Nervous System Disorder
• Central Nervous System Vasculitis
• Cerebellar Degeneration
• Chronic Inflammatory Demyelinating Polyneuropathy
• Lambert Eaton Myasthenic Syndrome
• Myasthenia Gravis
• Neuromyelitis Optica
• Opsoclonus Myoclonus Syndrome
• Rasmussen Subacute Encephalitis

Interventions

Type	Name	Description
BIOLOGICAL	Anti-Thymocyte Globulin	Given IV
PROCEDURE	Autologous Hematopoietic Stem Cell Transplantation	Undergo autologous or syngeneic stem cell transplantation
DRUG	Carmustine	Given IV
DRUG	Cytarabine	Given IV
DRUG	Etoposide	Given IV
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Melphalan	Given IV
PROCEDURE	Peripheral Blood Stem Cell Transplantation	Undergo autologous or syngeneic stem cell transplantation
DRUG	Prednisone	Given PO
PROCEDURE	Syngeneic Bone Marrow Transplantation	Undergo syngeneic bone marrow transplantation

Timeline

• Start date: 2008-06-01
• Primary completion: 2025-12-30
• Completion: 2030-01-31
• First posted: 2008-07-16
• Last updated: 2026-02-27

Locations

3 sites across 1 country: United States