High-dose Cyclophosphamide for Severe Refractory Crohn Disease

NCT ID: NCT01836289

Last Updated: 2018-08-13

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE1/PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-03-31
• Study Completion Date: 2018-03-31

Brief Summary

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.

Conditions

Crohn's Disease; Crohn Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

High-dose Cyclophosphamide

High-dose Cyclophosphamide

Group Type: EXPERIMENTAL

High-dose Cyclophosphamide

Intervention Type: DRUG

Interventions

High-dose Cyclophosphamide

Intervention Type: DRUG

Other Intervention Names

Endoxan®; Cytoxan®; CTX; Neosar®; Procytox®; Revimmune™

Eligibility Criteria

Inclusion Criteria

• ≥ 18 years of age, males and females will be eligible
• Moderate to severe Crohn's Disease (CD) with CDAI > 220, in addition to evidence of ulceration on ileocolonoscopy or active disease on small bowel imaging (in patients with an ostomy, CDAI criteria do not apply)
• Disease progression (primary or secondary non-responder, or reaction to) to at least one anti-tumor necrosis factor (TNF) agent (infliximab, adalimumab, certolizumab pegol), and additionally had disease progression despite one of the following immunosuppressant drugs: azathioprine, 6-mercaptopurine, methotrexate, cyclosporine, natalizumab, vedolizumab
• Willingness to participate in a clinical trial
• Approval by Enrollment Panel, who will collectively decide on the appropriateness of possible study study participants

Exclusion Criteria

• Pregnant or nursing women
• Sexually active men and women who do not agree to use effective means of birth control during treatment period
• Evidence of primarily fibrostenosing disease without active inflammatory disease on disease staging
• Co-morbid conditions including cardiac disease with an ejection fraction of < 45%, chronic renal failure with serum creatinine > 2.0, liver disease with total bilirubin > 2.0, (excluding hyperbilirubinemia secondary to Gilbert's disease) or transaminitis > 3x upper limit of normal.
• History of serious allergic reaction to cyclophosphamide
• History of malignancy in the last 5 years (excluding non-melanomatous skin cancers)
• Patients who are pre-terminal
• Toxic megacolon
• Active infection
• White blood cell count < 3000 cells/ul, platelets < 100K / ul, hemoglobin < 10.0 g/dL
• Any use of thiopurines, methotrexate or anti-TNF agents in the previous four weeks prior to treatment

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 100 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Johns Hopkins University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mark G. Lazarev, MD

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins University

Other Identifiers

NA_00052668

Identifier Type: -

Identifier Source: org_study_id