Stem Cell Transplantation in Idiopathic Inflammatory Myopathy Diseases
Myositis is a disease, believed to be due to immune cells, cells which normally protect the body, but are now attacking the muscles and other organ systems within body. As a result, the affected muscles and organs fail to work properly causing weakness, difficulty swallowing, skin rash, respiratory problems, heart problems, joint stiffness, soft tissue calcification and vasculitis (blood circulation problems). The likelihood of progression of this 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 this disease), followed by return of previously collected blood stem cells will stop the progression of myositis.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||High Dose Cyclophosphamide & ATG With Hematopoietic Stem Cell Transplantation in Patients With Refractory Idiopathic Inflammatory Myopathy Diseases: A Phase I Trial|
- Toxicity;Survival;Disease improvement defined by muscle strength (one or more in MRC scale) and improvement of muscle derived enzymes (normalization) or improvement of pulmonary function tests [ Time Frame: 5 years after transplant ] [ Designated as safety issue: Yes ]
|Study Start Date:||September 2005|
|Estimated Study Completion Date:||September 2017|
|Estimated Primary Completion Date:||December 2016 (Final data collection date for primary outcome measure)|
Experimental: Hematopoietic stem cell transplantation
Autologous hematopoietic stem cells will be injected
Biological: hematopoietic stem cell transplantation
Autologous hematopoietic stem cell transplantation
Conditioning Regimen (In order to assure sterility testing, a minimum of 14 days will be required between stem cell collection and starting the conditioning regimen).
The conditioning regimen is outlined in below:
Cyclophosphamide 50 mg/kg/day will be given IV over 1 hour in 250 cc of normal saline on day -5, -4, -3, and -2. If actual weight is < ideal weight, cyclophosphamide will be given based on actual weight. If actual weight is > ideal weight, cyclophosphamide will be given as adjusted ideal weight. Adjusted ideal weight = ideal weight + 40% (actual weight minus ideal weight).
Mesna 50mg/kg/day will be given IV over 24 hours in 250 cc of normal saline or D5W starting at 10AM each dose. Weight base is calculated same as cyclophosphamide as above.
1ATG (rabbit) 0.5 mg/kg on day -6 and 1mg/kg on day -5, -4, -3, -2 and -1 (total 5.5mg/kg, no dose adjustment) will be given IV over 10 hours in 250 cc of normal saline beginning at least 1 hour after infusion of cyclophosphamide. Premedicate with acetaminophen 650 mg po and diphenhydramine 25 mg po/IV 30 minutes before the infusion.
Methylprednisolone A suggested dose of 250mg IV should be administered 30 minutes before each ATG infusion.
Hydration A suggested rate of 125 cc/hr NS should be given starting 6 hours before the first cyclophosphamide dose and continued until 24 hours after the last cyclophosphamide dose. The rate of hydration will be aggressively adjusted. BID weights will be obtained. Amount of fluid can be modified based on patient's fluid status. Minimum target urine output is 2 liters/m2/day
G-CSF 5 mcg/kg/day will be given subcutaneously and continued until the absolute neutrophil counts reaches at least 500/µl.
Rituxan 500 mg will be given IV on the day before the first dose of ATG and the day after stem cell infusion.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00278564
|Contact: Dzemila Spahovic, MDfirstname.lastname@example.org|
|United States, Illinois|
|Northwestern University, Feinberg School of Medicine||Recruiting|
|Chicago, Illinois, United States, 60611|
|Principal Investigator: Richard Burt, MD|
|Sub-Investigator: Robert Suffit, MD|
|Sub-Investigator: Kathleen Quigley, R.N.|
|Sub-Investigator: Kimberly Yaung, RN|
|Principal Investigator:||Richard Burt, MD||Northwestern University|