Crohn's DiseasePhase 2NCT00271947
Crohn's Disease Stem Cell Transplantation
A Phase 2 study testing Autologous Stem Cell Transplantation in people with Crohn's Disease, run by Northwestern University at 1 site in United States.
Trial summary
Crohn's disease (CD) is a chronic illness, immunologically mediated, probably induced by the exposure of the intestine to an antigen or antigens similar to the intestine, to which immunologic tolerance is lost or a dysregulated immunity ensues. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Crohn's Disease
- What is being tested?
- Autologous Stem Cell Transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2005to Apr 2013
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age 18 years or older and less than age 55 years at time of pretransplant evaluation.
- An established clinical diagnosis of severe CD that has failed therapy with prednisone, azathioprine, 5 ASA products and metronidazole, and has failed an anti-TNF alpha inhibitor. Failure is defined as a CDAI (appendix A) 225-400.
- Pre-study peripheral blood counts must include a platelet count greater than 100,000/ul and an absolute neutrophil count greater than 1500/ul.
- In those randomized to receive the transplant, a stem cell harvest greater than 2.0 x 106 CD34 cells/kg is required.
- Ability to give informed consent.
Who cannot join
- HIV positive.
- History of coronary artery disease, or congestive heart failure.
- Uncontrolled diabetes mellitus, or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy.
- Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as head and neck cancer, or stage I breast cancer will be considered on an individual basis.
- Positive pregnancy test, lactation, inability or unwillingness to pursue effective means of birth control, failure to accept or comprehend irreversible sterility as a side effect of therapy.
- Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.
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- FEV 1/FVC < 50% of predicted, DLCO < 50% of predicted.
- Resting LVEF < 40%.
- Bilirubin > 2.0 mg/dl, transferase (AST) > 2x upper limit of normal.
- Serum creatinine > 2.0 mg/dl.
- Platelet count less than 100,000/ul, ANC less than 1500/ul.
- Patients presenting with intestinal perforation or toxic megacolon, or a suppurative problem that will require urgent surgery. In addition, the patient may not have any active infection. The presence of intestinal stomas does not exclude the patient from study.
- Splenomegaly (palpable spleen on physical exam).
- Inability to collect > 2.0 x 106 CD34+ cells/kg.
- Positive pregnancy test.
Trial locations and contacts
1 site in 1 country.
United States1 site
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, 60611
About this study
Official title: Crohn's Disease Non-myeloablative Autologous Hematopoietic Stem Cell Transplantation (CDNST) Versus Standard Therapy
Crohn's disease (CD) is a chronic illness, immunologically mediated, probably induced by the exposure of the intestine to an antigen or antigens similar to the intestine, to which immunologic tolerance is lost or a dysregulated immunity ensues. The disease has a variable course, from a mild, intermittently active illness requiring only symptomatic therapy to a fulminant illness requiring potentially dangerous immunosuppressive therapy, surgery or both. The molecular defect causing CD has not been characterized, but probably involves aberrant T cell function. Although CD often responds to immunosuppressive medication including corticosteroids, azathioprine and 6-mercaptopurine, to anti inflammatory drugs such as 5 aminosalicylate (5 ASA), or to some antimicrobial agents, including metronidazole, no therapy has been curative. In patients with severe CD, who have been unresponsive to corticosteroids, azathioprine, 5 ASA, metronidazole, and infliximab, we propose to compare the efficacy of Crohn's disease non-myeloablative autologous hematopoietic stem cell transplantation (CDNST) to standard therapy. Subsequent disease activity will be followed by (1) Crohn's disease activity index (CDAI), (2) a more global severity index, the Crohn's Severity Index, (3) type and amount of therapy for CD, and (4) clinical, hematologic and biochemical studies.
- Conditions
- Crohn's Disease
- Being tested
- Autologous Stem Cell Transplantation
- Medical subject headings
- Crohn Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Stem Cell TransplantationBiological
Autologous Stem Cell Transplantation will be performed after conditioning regimen
| Study group | Role | What they receive |
|---|---|---|
| Autologous Stem Cell Transplantation | Experimental | Autologous Stem Cell Transplantation will be performed after the conditioning regimen Receives: Biological: Autologous Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Remission or Clinical Improvement as Assessed by Crohn's Disease Activity Index (CDAI) Scores
Clinical remission defined as a CDAI less than 150 and clinical improvement defined as decline in CDI> or = 70 one year following entry. The participant was not assessed according to the criteria of the outcome measure, because the participant was lost for follow-up.
Measured: baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jan 2006 (estimated)
- Study startApr 2005
- Primary completionApr 2012
- Study completionApr 2013
Why it stopped: The PI, who was sponsor got retired
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Crohn's DiseaseStudies of the same condition, open and under way.
- More trials run by Northwestern UniversityThe 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwestern University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Richard Burt, MD, Northwestern University
- Investigators
- Robert Craig, MD, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00271947
- Sponsor's protocol ID
- CD Randomized
- Status last verified
- Feb 2014
- First submitted
- 2 Jan 2006
- Last updated
- 27 Mar 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Crohn's Disease Non-myeloablative Autologous Hematopoietic Stem Cell Transplantation (CDNST) Versus Standard Therapy
- ClinicalTrials.gov ID
- NCT00271947
- Acronym / sponsor ID
- CD Randomized
- Conditions
- Crohn's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The PI, who was sponsor got retired
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Northwestern UniversityUniversity, hospital or other
- Study start
- Apr 2005
- Primary completion
- Apr 2012 (actual)
- Study completion
- Apr 2013 (actual)
- First posted
- 4 Jan 2006 (estimated)
- Results
- Posted 27 Mar 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Crohn's disease (CD) is a chronic illness, immunologically mediated, probably induced by the exposure of the intestine to an antigen or antigens similar to the intestine, to which immunologic tolerance is lost or a dysregulated immunity ensues. The disease has a variable course, from a mild, intermittently active illness requiring only symptomatic therapy to a fulminant illness requiring potentially dangerous immunosuppressive therapy, surgery or both. The molecular defect causing CD has not been characterized, but probably involves aberrant T cell function. Although CD often responds to immunosuppressive medication including corticosteroids, azathioprine and 6-mercaptopurine, to anti inflammatory drugs such as 5 aminosalicylate (5 ASA), or to some antimicrobial agents, including metronidazole, no therapy has been curative. In patients with severe CD, who have been unresponsive to corticosteroids, azathioprine, 5 ASA, metronidazole, and infliximab, we propose to compare the efficacy of Crohn's disease non-myeloablative autologous hematopoietic stem cell transplantation (CDNST) to standard therapy. Subsequent disease activity will be followed by (1) Crohn's disease activity index (CDAI), (2) a more global severity index, the Crohn's Severity Index, (3) type and amount of therapy for CD, and (4) clinical, hematologic and biochemical studies.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous Stem Cell Transplantation | Experimental | Biological: Autologous Stem Cell TransplantationAutologous Stem Cell Transplantation will be performed after the conditioning regimen |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Age 18 years or older and less than age 55 years at time of pretransplant evaluation.
- An established clinical diagnosis of severe CD that has failed therapy with prednisone, azathioprine, 5 ASA products and metronidazole, and has failed an anti-TNF alpha inhibitor. Failure is defined as a CDAI (appendix A) 225-400.
- Pre-study peripheral blood counts must include a platelet count greater than 100,000/ul and an absolute neutrophil count greater than 1500/ul.
- In those randomized to receive the transplant, a stem cell harvest greater than 2.0 x 106 CD34 cells/kg is required.
- Ability to give informed consent.
Key exclusion criteria (15 listed)
- HIV positive.
- History of coronary artery disease, or congestive heart failure.
- Uncontrolled diabetes mellitus, or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy.
- Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as head and neck cancer, or stage I breast cancer will be considered on an individual basis.
- Positive pregnancy test, lactation, inability or unwillingness to pursue effective means of birth control, failure to accept or comprehend irreversible sterility as a side effect of therapy.
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 |
|---|---|---|
| Autologous Stem Cell TransplantationAutologous Stem Cell Transplantation will be performed after conditioning regimen | Biological | Autologous Stem Cell Transplantation |
Primary outcome
Number of Participants With Remission or Clinical Improvement as Assessed by Crohn's Disease Activity Index (CDAI) Scores
Time frame: baseline
Clinical remission defined as a CDAI less than 150 and clinical improvement defined as decline in CDI> or = 70 one year following entry. The participant was not assessed according to the criteria of the outcome measure, because the participant was lost for follow-up.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Robert Craig, MD principal investigator
Northwestern University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Autologous Stem Cell Transplantation in people with Crohn's Disease, run by Northwestern University 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 5 inclusion and 15 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: stopped early, as of the sponsor's last update on 27 Mar 2014. 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Mar 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Remission or Clinical Improvement as Assessed by Crohn's Disease Activity Index (CDAI) Scores (time frame: baseline). 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 27 Mar 2014.