High-dose Cyclophosphamide for Severe Refractory Crohn Disease
This research is being done to see if people with Crohn's disease who receive high-dose cyclophosphamide have an improvement of their disease, how long the benefit may last, and how safe cyclophosphamide is. This study is for patient with medically refractory disease that is not easily amenable to surgery.
Cyclophosphamide is an FDA-approved chemotherapy medication that is also frequently used to treat autoimmune illness; use of cyclophosphamide for autoimmune disease is not approved by the FDA. An autoimmune illness is when the immune system mistakenly attacks self, targeting the cells, tissues, and organs of a person's own body. There are many different autoimmune diseases and they can each affect the body is different ways. Crohn's disease is an autoimmune disease that primarily affects the small and large intestines. High dose-cyclophosphamide has been successfully used to treat Crohn's, primarily as part of a conditioning regimen for autologous stem cell transplantation. However, this therapy is limited in Crohn's because of it's serious infectious risks. This current study involves using high-dose cyclophosphamide without need for stem cell transplantation. This appears to be a safer approach in other autoimmune illnesses that have been studied.
Drug: High-dose Cyclophosphamide
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||High-dose Cyclophosphamide for Severe Refractory Crohn Disease|
- Evaluation of safety of the High-Dose Cyclophosphamide (HDC) protocol [ Time Frame: 3 Years ] [ Designated as safety issue: Yes ]Tablulation of serious adverse events associate with the HDC protocol
- HDC-Induced Steroid-free remission [ Time Frame: 3 Years ] [ Designated as safety issue: No ]To determine if HDC therapy can induce and maintain a steroid-free clinical remission (defined as CDAI<150) at 12 and 52 weeks. Applies to patients without an existing ostomy.
- HDC-Induced Mucosal Healing [ Time Frame: 3 Years ] [ Designated as safety issue: No ]To determine if HDC therapy can induce sustained mucosal healing defined as absence of ulcers on colonoscopy
- Improvement in patient reported quality of life [ Time Frame: 3 years ] [ Designated as safety issue: No ]To determine if HDC can lead to improvement in inflammatory bowel disease questionnaire scores at weeks 12 and 52
- Molecular Mechanisms of High-dose Cyclophosphamide (HiCy) Therapy [ Time Frame: 3 Years ] [ Designated as safety issue: No ]To investigate the molecular mechanisms by which HiCy therapy works by analyzing the effects of HiCy on the levels of serum cytokines (using multiplex ELISA), and correlate the data with clinical activity and treatment response.
|Study Start Date:||March 2015|
|Estimated Study Completion Date:||March 2018|
|Estimated Primary Completion Date:||March 2018 (Final data collection date for primary outcome measure)|
Experimental: High-dose Cyclophosphamide
Drug: High-dose Cyclophosphamide
Please refer to this study by its ClinicalTrials.gov identifier: NCT01836289
|Contact: Mark G. lazarev, MDfirstname.lastname@example.org|
|United States, Maryland|
|Johns Hopkins Medical Institutions||Recruiting|
|Baltimore, Maryland, United States, 21205|
|Contact: Mark G. lazarev, MD 410-502-3147 email@example.com|
|Principal Investigator:||Mark G. Lazarev, MD||Johns Hopkins University|