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Hematopoietic Stem Cell Transplant in Devic's Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by Northwestern University
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University
ClinicalTrials.gov Identifier:
NCT00787722
First received: October 31, 2008
Last updated: September 23, 2014
Last verified: September 2014
  Purpose

This study is designed to examine whether treating Devic's disease patients with high dose cyclophosphamide together with 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 Intervention Phase
Devic's Disease
Procedure: Hematopoietic stem cell transplantation
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Trial of High Dose Immunosuppressive Therapy With Hematopoietic Stem Cell Support in Devic's Disease

Resource links provided by NLM:


Further study details as provided by Northwestern University:

Primary Outcome Measures:
  • PASAT 25-foot walk 9-hole peg test [ Time Frame: pre-transplant, 6mo, 12mo, yearly for 5 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Self-administered quality of life exams (FACT-BMT and SF-36) [ Time Frame: pre-transplant, 6mo, 12mo, yearly for 5 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 10
Study Start Date: October 2008
Estimated Study Completion Date: October 2017
Estimated Primary Completion Date: October 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Hematopoietic stem cell transplantation
Hematopoietic stem cell transplantation will be performed after conditioning regimen.
Procedure: Hematopoietic stem cell transplantation

Mobilization

G-CSF- guidelines, 5-10 mcg/kg/day will be started day +5 and continued until the absolute neutrophil counts reaches at least 1,000

Cytoxan dose of 2gm/m will be infused over two hours.

Mesna, given the dose of 2.0 gm/m, infusion to be given over 24 hours.

Transplant Conditioning Regimen

Cyclophosphamide 50 mg/kg/day will be given IV over 2 hours in 500 cc of normal saline.

r ATG 0.5 mg/kg given on day -5, then 1.0 mg/kg given on day -4, then 1.5 mg/kg given on days -3 through -1.

Rituxan ( Rituximab ) - The dose of 500 mg of Rituximab will be given on days -6 and on day + 1.

G-CSF - guidelines, 5-10 mcg/kg/day will be started day + 5 and continued until the absolute neutrophil counts reaches at least 1,000/μl.


  Show Detailed Description

  Eligibility

Ages Eligible for Study:   16 Years to 65 Years
Genders Eligible for Study:   Both
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 IUD (intrauterine device); 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
  • FEV1/FVC < 60% of predicted after bronchodilator therapy (if necessary)
  • DLCO < 50% of predicted
  • Resting 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 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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00787722

Contacts
Contact: Dzemila Spahovic, MD 312-695-4960 d-spahovic@northwestern.edu

Locations
United States, Illinois
Northwestern University, Feinberg School of Medicine Recruiting
Chicago, Illinois, United States, 60611
Sub-Investigator: Roumen Balabanov, MD         
Sub-Investigator: Dusan Stefoski, MD         
Sub-Investigator: Robert Suffit, MD         
Sponsors and Collaborators
Northwestern University
Investigators
Principal Investigator: Richard Burt, MD Northwestern University
  More Information

No publications provided

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
Study First Received: October 31, 2008
Last Updated: September 23, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Northwestern University:
High dose immunosuppressive therapy
Hematopoietic stem cell support

Additional relevant MeSH terms:
Neuromyelitis Optica
Autoimmune Diseases
Autoimmune Diseases of the Nervous System
Cranial Nerve Diseases
Demyelinating Autoimmune Diseases, CNS
Demyelinating Diseases
Eye Diseases
Immune System Diseases
Multiple Sclerosis
Myelitis, Transverse
Nervous System Diseases
Optic Nerve Diseases
Optic Neuritis
Immunosuppressive Agents
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on November 27, 2014