Autologous Hematopoietic Stem Cell Transplantation for Crohn's Disease Treatment

• Register: ClinicalTrials.gov
• Last refreshed on: 25 April 2016
• Main ID: NCT01932658
• Date of registration: 28/06/2013
• Prospective Registration: Yes
• Primary sponsor: University of California, Los Angeles
• Public title: Autologous Hematopoietic Stem Cell Transplantation for Crohn's Disease Treatment HSCT
• Scientific title: Autologous Hematopoietic Stem Cell Transplantation for Crohn's Disease Treatment
• Date of first enrolment: February 2014
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT01932658
• Study type: Interventional
• Study design: Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 0

Countries of recruitment

United States

Contacts

• Name: Daniel W Hommes, MD,PhD
• Affiliation: University of California, Los Angeles

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age between 7 and 50 years (patients aged 50 - 70 can participate up to the principal
investigators discretion).

2. Confirmed diagnosis of active Crohn's disease:

1. Diagnosis of Crohn's disease based on typical radiological appearances and or
typical histology at least 6 months prior to screening.

2. 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:

1. elevated C-Reactive Protein (CRP)

2. endoscopic evidence of active disease confirmed by histology

3. clear evidence of active small bowel Crohn's disease on Computed tomography (CT)
or Magnetic Resonance (MR) enterography.

3. 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.

4. Current problems unsuitable for surgery or patient at risk for developing short bowel
syndrome.

5. Informed consent:

1. Prepared to undergo additional study procedures as per trial schedule

2. Patient has undergone intensive counseling about risks

Exclusion Criteria:

1. Pregnancy or unwillingness to use adequate contraception during the study, in women
of childbearing age. Unwillingness of using appropriate contraceptive measures in
males.

2. Concomitant severe disease

1. renal: creatinine clearance < 30 mL/min (measured or estimated)

2. 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.

3. pulmonary: diffusion capacity <40%

4. psychiatric disorders including active drug or alcohol abuse

5. concurrent or recent history of malignant disease (excluding non-melanoma skin
cancer)

6. uncontrolled hypertension, defined as resting systolic blood pressure = 140
and/or resting diastolic pressure = 90 despite at least 2 anti-hypertensive
agents.

7. 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.

8. other chronic disease causing significant organ failure.

3. Infection or risk thereof:

1. Current clinical relevant abscess or significant active infection.

2. Perianal fistula without free drainage. Perianal fistulas is not an exclusion
provided there is natural free drainage or a seton suture(s)have been placed.

3. History of tuberculosis or currently at risk for tuberculosis

4. Quantiferon Gold test result or other investigations that the investigators
regard as evidence of active tuberculosis.

5. Abnormal chest X-ray (CXR) consistent with active infection or neoplasm.

4. Significant malnutrition: Body Mass Index (BMI) = 18, serum albumin < 20g/l.

5. Previous poor compliance.

6. Concurrent enrollment in any other protocol using an investigational drug or
hematopoietic growth factor up to four weeks before study entry.



Age minimum: 7 Years
Age maximum: 70 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Crohn's Disease

Intervention(s)

Biological: Hematopoietic stem cell transplantation

Primary Outcome(s)

The primary outcome to be measured is safety and clinical benefit of lymphoablation followed by autologous HSCT rescue in therapy refractory Crohn's disease. [Time Frame: Month 1- 24 months post transplant]

Secondary Outcome(s)

Disease remission [Time Frame: 6 and 12 months post transplant]

Secondary ID(s)

UCLA-001836

IRB#12-001836

Source(s) of Monetary Support

Please refer to primary and secondary sponsors