Autologous Hematopoietic Stem Cell Transplantation for Crohn's Disease Treatment

NCT01932658 | Withdrawn Early Phase 1

• 2 other identifiers: UCLA-001836IRB#12-001836
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety and potential clinical benefit of lymphoablation followed by autologous HSCT rescue in therapy refractory Crohn's disease

Conditions

Crohn's Disease

Keywords

autologous; Lymphoablation; hematopoietic; immune system

Outcome Measures

Primary Outcomes (1)

• The primary outcome to be measured is safety and clinical benefit of lymphoablation followed by autologous HSCT rescue in therapy refractory Crohn's disease.

  Safety will be evaluated by the amount of related adverse events. All adverse events will be recorded in a standardized way and their relationship to the study protocol will be assessed at various short and long term time points.

  Month 1- 24 months post transplant

Secondary Outcomes (1)

• Disease remission

  6 and 12 months post transplant

Other Outcomes (1)

• Change in Crohn's disease endoscopic index (SES, Appendix 2) after 6 and 12 months.

  6 and 12 months post transplant

Study Arms (1)

Hematopoietic stem cell transplantation

EXPERIMENTAL

Lymphoablation followed by autologous hematopoietic stem cell transplantation rescue.

Biological: Hematopoietic stem cell transplantation

Interventions

Hematopoietic stem cell transplantation BIOLOGICAL

Lymphoablation followed by hematopoietic stem cell transplantation to rescue the immune system.

Also known as: HSCT

Hematopoietic stem cell transplantation

Eligibility Criteria

• Age: 7 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age between 7 and 50 years (patients aged 50 - 70 can participate up to the principal investigators discretion).
• Confirmed diagnosis of active Crohn's disease:
• Diagnosis of Crohn's disease based on typical radiological appearances and or typical histology at least 6 months prior to screening.
• Active disease at the time of registration to the trial, defined as
• i)Crohn's Disease Activity Index (CDAI) \> 250, and ii)Two of the following:
• elevated C-Reactive Protein (CRP)
• endoscopic evidence of active disease confirmed by histology
• clear evidence of active small bowel Crohn's disease on Computed tomography (CT) or Magnetic Resonance (MR) enterography.
• Unsatisfactory course despite 3 immunosuppressive agents (usually azathioprine, methotrexate and infliximab, adalimumab and/or certolizumab) in addition to corticosteroids. Patients should have relapsing disease (i.e. 1 exacerbation/year) despite thiopurines, methotrexate and/or infliximab/adalimumab/certolizumab maintenance therapy or clear demonstration of intolerance / toxicity to these drugs.
• Current problems unsuitable for surgery or patient at risk for developing short bowel syndrome.
• Informed consent:
• Prepared to undergo additional study procedures as per trial schedule
• Patient has undergone intensive counseling about risks

You may not qualify if:

• Pregnancy or unwillingness to use adequate contraception during the study, in women of childbearing age. Unwillingness of using appropriate contraceptive measures in males.
• Concomitant severe disease
• renal: creatinine clearance \< 30 mL/min (measured or estimated)
• cardiac: clinical evidence of refractory congestive heart failure; left ventricular ejection fraction \< 40% by multigated radionuclide angiography (MUGA) or cardiac echo; chronic atrial fibrillation necessitating oral anticoagulation; uncontrolled ventricular arrhythmia; pericardial effusion with hemodynamic consequences as evaluated by an experienced echo cardiographer.
• pulmonary: diffusion capacity \<40%
• psychiatric disorders including active drug or alcohol abuse
• concurrent or recent history of malignant disease (excluding non-melanoma skin cancer)
• uncontrolled hypertension, defined as resting systolic blood pressure ≥ 140 and/or resting diastolic pressure ≥ 90 despite at least 2 anti-hypertensive agents.
• uncontrolled acute or chronic infection with HIV, Human T-Lymphotropic virus (HTLV-1 or 2), hepatitis viruses or any other infection the investigators consider a contraindication to participation.
• other chronic disease causing significant organ failure.
• Infection or risk thereof:
• Current clinical relevant abscess or significant active infection.
• History of tuberculosis or currently at risk for tuberculosis
• Quantiferon Gold test result or other investigations that the investigators regard as evidence of active tuberculosis.
• Abnormal chest X-ray (CXR) consistent with active infection or neoplasm.

Sponsors & Collaborators

• University of California, Los Angeles: lead

Study Sites (1)

UCLA Center for Inflammatory Bowel Diseases

Los Angeles, California, 90095, United States

Location

MeSH Terms

Conditions

Crohn Disease

Interventions

Hematopoietic Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Inflammatory Bowel Diseases; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Intervention Hierarchy (Ancestors)

Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Daniel W Hommes, MD,PhD

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 28, 2013
• First Posted: August 30, 2013
• Study Start: February 1, 2014
• Primary Completion: March 1, 2016
• Study Completion: March 1, 2017
• Last Updated: April 15, 2016

Record last verified: 2016-04

Locations

US (1)