Non-myeloablative Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Refractory Crohn's Disease

NCT01288053 | Terminated Phase 1 Results

• 1 other identifier: DIADCDAllo2005
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

Allogeneic transplantation has been a high-risk procedure, although non-myeloablative conditioning regimens (mini-transplantation) minimizes regimen related toxicity. The investigators, therefore, propose a phase I study of matched sibling allogeneic hematopoietic stem cell transplantation with non-myeloablative conditioning. In addition, graft versus host disease (GVHD) will be virtually eliminated by CAMPATH that removes donor T cells from the graft. The goal is to assess the toxicity/efficacy (phase I) of allogeneic non-myeloablative hematopoietic stem cell transplantation for high-risk Crohn's disease. In simplistic terms, this protocol is designed to ablate an aberrant immune system and then, similar to the use of marrow transplants for immunodeficient patients, reconstitute a new immune system with lymphocyte depleted marrow.

Conditions

Crohn's Disease

Outcome Measures

Primary Outcomes (1)

• Survival

  The number of participants who survived treatment

  Up to five years

Study Arms (1)

Allogeneic Stem Cell Therapy

EXPERIMENTAL

Allogeneic Stem Cell Therapy will be performed after conditioning

Biological: Allogeneic Stem Cell Therapy

Interventions

Allogeneic Stem Cell Therapy BIOLOGICAL

Donor stem cells will be given to subject diagnosed with Crohn's disease

Allogeneic Stem Cell Therapy

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Age \>18 years and less than age 45 years at time of pretransplant evaluation and,
• Ability to give informed consent.
• And either A or B A)An established clinical diagnosis of severe CD with disease onset before 16 years old (at least 71 genetic loci predispose to pediatric CD, Limbergen, JV. Annu. Rev. Genom. Human Genet. 2009; 10:89-116) that has failed therapy with prednisone, 5 ASA products and has failed an anti-TNF therapy. Failure is defined as a CDAI (appendix A) 250-400 or a Craig Severity Score that is \> 17 (appendix C).
• B)Relapse after an autologous HSCT with a CDAI (appendix A) 250-400 or a Craig Severity Score that is \> 17 (appendix C).

You may not qualify if:

• 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.
• FEV 1/FVC \< 50% of predicted, DLCO \< 50% of predicted.
• Resting LVEF \< 50%.
• Bilirubin \> 2.0 mg/dl, transferase (AST) \> 2x upper limit of normal, unless the abnormalities are secondary to Crohn's disease.
• 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.
• Pre-study peripheral blood counts must include a platelet count greater than 100,000/ul and an absolute neutrophil count greater than 1500/ul.
• Creatinine clearance more than 20% below lower limit of normal for age and sex.
• Short gut syndrome.

Sponsors & Collaborators

• Northwestern University: lead

Study Sites (1)

Northwestern University

Chicago, Illinois, 60611, United States

Location

MeSH Terms

Conditions

Crohn Disease

Condition Hierarchy (Ancestors)

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

Limitations and Caveats

Small numbers of subjects enrolled

Results Point of Contact

• Title: Dr Richard Burt
• Organization: Northwestern University

rburt@northwestern.edu

312-695-4960

Study Officials

• Richard Burt, MD

  Northwestern University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

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

Study Record Dates

• First Submitted: January 27, 2011
• First Posted: February 2, 2011
• Study Start: January 1, 2010
• Primary Completion: June 1, 2016
• Study Completion: June 1, 2016
• Last Updated: August 6, 2018
• Results First Posted: August 6, 2018

Record last verified: 2018-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)