ClinicalTrials.gov
ClinicalTrials.gov Menu

Hematopoietic Stem Cell Transplant in Devic's Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00787722
Recruitment Status : Active, not recruiting
First Posted : November 7, 2008
Last Update Posted : July 17, 2018
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Brief Summary:
This study is designed to examine whether treating Devic's disease patients with high dose cyclophosphamide together with rabbit antithymocyte globulin (rATG)/rituximab (drugs which reduce the function of the immune system), followed by return of previously collected patient's stem cells will result in improvement in Devic's disease. 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 intense chemotherapy is to destroy the cells in patient's immune system, which may be causing his/her disease. The purpose of the stem cell infusion is to produce a normal immune system that will no longer attack patient's body. The purpose of study is to examine the safety and efficacy of this treatment. The drugs used in this study treatment are drugs for commonly used for immune suppression.

Condition or disease Intervention/treatment Phase
Devic's Disease Procedure: Hematopoietic Stem Cell Transplantation Drug: Cyclophosphamide Drug: G-CSF Drug: rATG Drug: Mesna Drug: Rituximab Drug: Methylprednisolone Phase 1 Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Trial of High Dose Immunosuppressive Therapy With Hematopoietic Stem Cell Support in Devic's Disease
Actual Study Start Date : October 10, 2009
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : January 2019


Arm Intervention/treatment
Experimental: Hematopoietic Stem Cell Transplantation
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Procedure: Hematopoietic Stem Cell Transplantation
Infusion of participant's own stem cells

Drug: Cyclophosphamide
A medication used as chemotherapy and to suppress the immune system
Other Names:
  • Cytoxan
  • Neosar

Drug: G-CSF
A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
Other Names:
  • Neupogen
  • Filgrastim
  • Granix
  • Zarxio

Drug: rATG
A rabbit polyclonal antibody to lymphocytes
Other Names:
  • Thymoglobulin
  • Anti-Thymocyte Globulin

Drug: Mesna
A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Other Name: Mesnex

Drug: Rituximab
Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Other Name: Rituxan

Drug: Methylprednisolone
A corticosteroid medication used to suppress the immune system and decrease inflammation
Other Names:
  • Solu-Medrol
  • Depo-Medrol




Primary Outcome Measures :
  1. Survival [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]
    survival rate will be evaluated at 6 months,1 year, 2 year, 3 year, 4 year, 5 year after the transplant

  2. Visual Acuity [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]
    Visual Acuity will be evaluated by neurologist

  3. Decreased weakness in limb (MRS) [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]
    Decreased weakness in limb will be assessed by using muscle strength testing scale


Secondary Outcome Measures :
  1. Functional Assessment of Cancer Therapy-Bone Marrow Transplant [ Time Frame: pre-transplant, 6mo, 12mo, yearly for 5 years ]
    The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) is a self-administered quality of life exam.

  2. 36-Item Short Form Health Survey [ Time Frame: pre-transplant, 6mo, 12mo, yearly for 5 years ]
    The 36-Item Short Form Health Survey (SF-36) is a self-administered quality of life exams.

  3. Number of acute attacks [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]
    Number of acute attacks will be evaluated at 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant

  4. Time to confinement to wheelchair [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]
    Time to confinement to wheelchair will be evaluated at 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant

  5. Time to legal blindness(visual acuity of 20/200 or less in the better eye with best correction possible [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]
    Time to legal blindness(visual acuity of 20/200 or less in the better eye with best correction possible) will be evaluated at 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant

  6. Disability scores [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]
    Disability scores (disease improvement defined by at least a 1 point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least three months apart

  7. NMO-IgG aquaporin- 4 autoantibody titer [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]
    NMO-IgG aquaporin- 4 autoantibody titer will be tested at 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age between 16-65, at the time of pretransplant evaluation
  • An established diagnosis of Devic's disease (more than one acute attack)
  • NMO- IgG aquaporin-4 autoantibody positive

Exclusion Criteria:

  • Paraplegia or quadriplegia and legal blindness (defined as visual acuity of 20/200 or less in the better eye with the best correction possible)
  • Any illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy
  • Prior history of malignancy except localized basal cell, squamous skin cancer or carcinoma in situ of the cervix. Other malignancies for which the patient is judged to be cured, such as head and neck cancer, or breast cancer will be considered on an individual basis
  • Positive pregnancy test
  • Inability or unwillingness to pursue effective means of birth control. Effective birth control is defined as 1) refraining from all acts of vaginal intercourse (ABSTINENCE); 2) consistent use of birth control pills; 3) injectable birth control methods (Depo-provera, Norplant); 4) tubal sterilization or male partner who has undergone vasectomy; 5) placement of an intrauterine device (IUD); or 6) use, with every act of intercourse, of diaphragm with contraceptive jelly and/or condoms with contraceptive foam
  • Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy
  • forced expiratory volume at one (FEV1) / forced vital capacity (FVC) < 60% of predicted after bronchodilator therapy (if necessary)
  • Diffusing capacity of lung for carbon monoxide (DLCO) < 50% of predicted
  • Resting left ventricular ejection fraction (LVEF) < 50 %
  • Serum creatinine > 2.0 mg/dl
  • Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins
  • Presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have MRI exams
  • Bilirubin > 2.0 mg/dl
  • Platelet count < 100,000/ul or absolute neutrophil count (ANC) < 1000/ul
  • Psychiatric illness, mental deficiency or cognitive dysfunction making compliance with treatment or informed consent impossible
  • Active infection except asymptomatic bacteriuria
  • Inability to give informed consent
  • HIV positive
  • Transaminases > 3x of normal limits, liver cirrhosis

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00787722


Locations
United States, Illinois
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, United States, 60611
Sponsors and Collaborators
Northwestern University
Investigators
Principal Investigator: Richard Burt, MD Northwestern University

Responsible Party: Richard Burt, MD, MD, Northwestern University
ClinicalTrials.gov Identifier: NCT00787722     History of Changes
Other Study ID Numbers: DIAD Devic's Disease Auto 2008
First Posted: November 7, 2008    Key Record Dates
Last Update Posted: July 17, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Keywords provided by Richard Burt, MD, Northwestern University:
High dose immunosuppressive therapy
Hematopoietic stem cell support

Additional relevant MeSH terms:
Neuromyelitis Optica
Myelitis, Transverse
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Optic Neuritis
Optic Nerve Diseases
Cranial Nerve Diseases
Demyelinating Diseases
Eye Diseases
Autoimmune Diseases
Immune System Diseases
Cyclophosphamide
Immunosuppressive Agents
Thymoglobulin
Antilymphocyte Serum
Rituximab
Methylprednisolone Hemisuccinate
Prednisolone
Prednisolone acetate
Methylprednisolone acetate
Methylprednisolone
Prednisolone hemisuccinate
Prednisolone phosphate
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action