Crohn's DiseaseEarly Phase 1HSCTNCT01932658
Autologous Hematopoietic Stem Cell Transplantation for Crohn's Disease Treatment
A Early Phase 1 study testing Hematopoietic stem cell transplantation in people with Crohn's Disease, run by University of California, Los Angeles at 1 site in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 7 to 70 years
- Conditions the study is for
- Crohn's Disease
- What is being tested?
- Hematopoietic stem cell transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2014to Mar 2017
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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)
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- 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
Who cannot join
- 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
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- 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.
- 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.
- 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.
- Significant malnutrition: Body Mass Index (BMI) ≤ 18, serum albumin < 20g/l.
- Previous poor compliance.
- Concurrent enrollment in any other protocol using an investigational drug or hematopoietic growth factor up to four weeks before study entry.
- -
Trial locations and contacts
1 site in 1 country.
United States1 site
UCLA Center for Inflammatory Bowel Diseases
Los Angeles, California, 90095
About this study
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
Read the detailed description
The treatment of Crohn's disease has proven to be quite efficacious in the majority of patients with the timely use of combination therapies for remission induction (corticosteroids and/or biologics) and maintenance of disease control (immunosuppressives and/or biologics). However, a proportion of patients fail to achieve complete and long term disease control and often require multiple intestinal surgeries with a risk of developing short bowel syndrome. Lymphoablation followed by hematopoietic stem cell transplantation to rescue the immune system has been proposed as an alternative strategy to induce long term disease control in this high risk population. It has been demonstrated that despite the potential toxicity and morbidity associated with the procedure, the benefit-risk ratio is favorable. Hence, we propose to offer HSCT to selected CD patients, and to study mechanisms of reducing T cell autoreactivity which will hopefully lead to more focused therapeutic approaches in the future.
- Conditions
- Crohn's Disease
- Being tested
- Hematopoietic stem cell transplantation
- Keywords
- autologous, Lymphoablation, hematopoietic, immune system
- Medical subject headings
- Crohn Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hematopoietic stem cell transplantationBiological
Also called HSCT
Lymphoablation followed by hematopoietic stem cell transplantation to rescue the immune system.
| Study group | Role | What they receive |
|---|---|---|
| Hematopoietic stem cell transplantation | Experimental | Lymphoablation followed by autologous hematopoietic stem cell transplantation rescue. Receives: Biological: Hematopoietic stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Month 1- 24 months post transplant
Secondary outcomes (1)
Disease remission
Second, to determine clinical benefit, the percentage of patients in sustained disease remission at 0, 2, 4, 6, 12 and 24 months post HSCT will be determined. Sustained disease remission is defined as a CDAI < 150 (Appendix 1) without the use of corticosteroids. In addition, mucosal healing will be assessed during ileocolonoscopy at 6 and 12 months following HSCT using the SES endoscopic index (see under secondary endpoints
Measured: 6 and 12 months post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2013 (estimated)
- Study startFeb 2014
- Primary completionMar 2016 (estimated)
- Study completionMar 2017 (estimated)
Why it stopped: patients insurance would not cover the cost of the study procedures
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Crohn's DiseaseStudies of the same condition, open and under way.
- More trials run by University of California, Los AngelesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Los Angeles
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Daniel Hommes, MD, University of California, Los Angeles
- Investigators
- Daniel W Hommes, MD,PhD, Principal investigator, University of California, Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01932658
- Sponsor's protocol ID
- UCLA-001836
- Other IDs
- IRB#12-001836
- Acronym
- HSCT
- Status last verified
- Apr 2016
- First submitted
- 28 Jun 2013
- Last updated
- 15 Apr 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Hematopoietic Stem Cell Transplantation for Crohn's Disease Treatment
- ClinicalTrials.gov ID
- NCT01932658
- Acronym / sponsor ID
- HSCT / UCLA-001836
- Conditions
- Crohn's Disease
- Keywords
- autologous, Lymphoablation, hematopoietic, immune system
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: patients insurance would not cover the cost of the study procedures
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of California, Los AngelesUniversity, hospital or other
- Study start
- Feb 2014
- Primary completion
- Mar 2016 (estimated)
- Study completion
- Mar 2017 (estimated)
- First posted
- 30 Aug 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
The treatment of Crohn's disease has proven to be quite efficacious in the majority of patients with the timely use of combination therapies for remission induction (corticosteroids and/or biologics) and maintenance of disease control (immunosuppressives and/or biologics). However, a proportion of patients fail to achieve complete and long term disease control and often require multiple intestinal surgeries with a risk of developing short bowel syndrome. Lymphoablation followed by hematopoietic stem cell transplantation to rescue the immune system has been proposed as an alternative strategy to induce long term disease control in this high risk population. It has been demonstrated that despite the potential toxicity and morbidity associated with the procedure, the benefit-risk ratio is favorable. Hence, we propose to offer HSCT to selected CD patients, and to study mechanisms of reducing T cell autoreactivity which will hopefully lead to more focused therapeutic approaches in the future.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hematopoietic stem cell transplantation | Experimental | Biological: Hematopoietic stem cell transplantationLymphoablation followed by autologous hematopoietic stem cell transplantation rescue. |
Who may qualify
- Ages
- 7 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- 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:
- 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:
Key exclusion criteria (20 listed)
- 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
- Infection or risk thereof:
- Significant malnutrition: Body Mass Index (BMI) ≤ 18, serum albumin < 20g/l.
- Previous poor compliance.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Hematopoietic stem cell transplantationAlso: HSCTLymphoablation followed by hematopoietic stem cell transplantation to rescue the immune system. | Biological | Hematopoietic stem cell transplantation |
Primary outcome
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
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.
1 secondary outcome
Disease remission
Time frame: 6 and 12 months post transplant
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Daniel W Hommes, MD,PhD principal investigator
University of California, Los Angeles
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Hematopoietic stem cell transplantation in people with Crohn's Disease, run by University of California, Los Angeles at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 15 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2016.