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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 : Completed
First Posted : November 7, 2008
Results First Posted : February 28, 2020
Last Update Posted : February 28, 2020
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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: N/A
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
Actual Primary Completion Date : November 2018
Actual Study Completion Date : November 2018


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


Secondary Outcome Measures :
  1. Quality of Life (QOL) Short Form - 36 (SF-36) [ Time Frame: pre-transplant 12mo and 5 years ]
    SF- 36 is a self-administered quality of life exam. The evaluation of the results was done by attributing scores to each question, which were then transformed into a scale ranging from 0 to 100, where 0 corresponds to the worst quality of life and 100 to the best.

  2. Post HSCT Immune -Modulating Medication and Relapse [ Time Frame: Pre transplant and 6 months, 1 year, 2 year, 3 year, 4 year and 5 year after transplant ]
    Number of immune - modulating medication and relapse evaluated 5 year - after the transplant

  3. Number of Patients Who Require No Device Assistance for Ambulation [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]
    No Device Assistance Needed for Ambulation evaluated at 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant

  4. Disability Score: Expanded Disability Status Scale (EDSS) [ Time Frame: pretransplant 6 month, 5 year ]

    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.

    The EDSS scale ranges from 0 to 10 in 0.5 unit increments that represent higher levels of disability.


  5. NMO-IgG Aquaporin- 4 Autoantibody Titer [ Time Frame: Pretransplant and 5 year Post Transplant ]
    NMO-IgG aquaporin- 4 autoantibody titer will be tested pretransplant and post 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.


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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
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United States, Illinois
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, United States, 60611
Sponsors and Collaborators
Northwestern University
Investigators
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Principal Investigator: Richard Burt, MD Northwestern University
  Study Documents (Full-Text)

Documents provided by Richard Burt, MD, Northwestern University:
Study Protocol  [PDF] April 28, 2017
Statistical Analysis Plan  [PDF] April 28, 2017

Additional Information:
Publications:
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Responsible Party: Richard Burt, MD, MD, Northwestern University
ClinicalTrials.gov Identifier: NCT00787722    
Other Study ID Numbers: DIAD Devic's Disease Auto 2008
First Posted: November 7, 2008    Key Record Dates
Results First Posted: February 28, 2020
Last Update Posted: February 28, 2020
Last Verified: November 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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:
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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
Methylprednisolone
Methylprednisolone Acetate
Methylprednisolone Hemisuccinate
Prednisolone
Prednisolone acetate
Cyclophosphamide
Rituximab
Thymoglobulin
Antilymphocyte Serum
Prednisolone hemisuccinate
Prednisolone phosphate
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action