Autologous Transplant Targeted Against Crohn's

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT04154735
• Date of registration: 08/03/2019
• Prospective Registration: Yes
• Primary sponsor: Northwestern University
• Public title: Autologous Transplant Targeted Against Crohn's ATTAC
• Scientific title: Autologous Hematopoietic Stem Cell Transplant for Crohn's Disease
• Date of first enrolment: November 2019
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT04154735
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Richard Burt, MD
• Affiliation: Northwestern University

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age 18 years and less than age 50 years at the time of pre-transplant evaluation

2. Ability to give informed consent

3. An established clinical diagnosis of severe Crohn's Disease* that has failed therapy
with prednisone or budesonide (Entocort) and either a or b below:

1. At least two anti-tumor necrosis factor (TNF) drugs (e.g., infliximab (Remicade),
adalimumab (Humira), or certolizumab pegol (Cimzia))

2. One anti-TNF drug as above and either vedolizumab (Entyvio) or ustekinumab
(Stelara)

- Severe Crohn's Disease is defined as a CDAI (see Appendix A) of 250 to 400
or a Craig's Crohn's Severity Index (CCSI, see Appendix B) that is > 17.

Exclusion Criteria:

1. Uncontrolled diabetes mellitus or any other illness that in the opinion of the
investigators would jeopardize the ability of the patient to tolerate aggressive
treatment

2. Prior history of malignancy (except localized basal cell or squamous cell skin cancer,
or carcinoma in situ of the cervix). 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

3. Positive pregnancy test, inability to pursue effective means of birth control, or
failure to willingly accept or comprehend irreversible sterility as a side effect of
therapy

4. HIV positive

5. Hepatitis B or C positive

6. Psychiatric illness or mental deficiency making compliance with treatment or informed
consent impossible

7. Untreated life-threatening cardiac arrhythmia on EKG or 24-hour holter or history of
coronary artery disease or congestive heart failure

8. Left ventricular ejection fraction (LVEF) <50%

9. Forced vital capacity (FVC) <60% of predicted after bronchodilator therapy (if
necessary) or diffusing capacity of the lungs for carbon monoxide (DLCO) hemoglobin
corrected <60 % predicted

10. Serum creatinine >2 mg/dl

11. 24-hour urine creatinine clearance <90

12. Liver transaminases >2x of normal limits, or bilirubin >2 mg/dl unless due to Crohn's
Disease

13. Major hematological abnormalities such as platelet count < 100,000/ul or absolute
neutrophil count (ANC) < 1500/ul

14. Failure to collect at least 2 x10^6 cluster of differentiation 34 (CD34+) cells/kg

15. Any active infection

16. Known hypersensitivity to mouse, rabbit, or E. coli derived proteins

17. Short Bowel Syndrome defined as intestinal dysfunction with the presence of
significant malabsorption of both macronutrients and micronutrients or when
gastrointestinal function is inadequate to maintain nutrient and hydration status
without intravenous or enteral supplementation.

18. History of anorexia nervosa (serum albumin = 20 g/L, body mass index = 18)

19. Patients presenting with intestinal perforation or toxic megacolon or a problem that
will require urgent surgery. The presence of intestinal stomas, strictures, or
fistulae does not exclude the patient from study.

20. Unable or unwilling to stop using and/or smoking tobacco products

21. Abnormal peripheral blood cytogenetics



Age minimum: 18 Years
Age maximum: 49 Years
Gender: All

Health Condition(s) or Problem(s) studied

Crohn's Disease

Intervention(s)

Drug: Tacrolimus

Drug: G-CSF

Drug: Mesna

Drug: Rifaximin

Drug: Alemtuzumab

Drug: Cyclophosphamide

Drug: Fludarabine

Primary Outcome(s)

Clinical remission [Time Frame: 6 months, 1 year, 2 years, 3 years]

Overall survival [Time Frame: 3 years]

Complete remission [Time Frame: 1 year, 2 years, 3 years]

Treatment-related mortality [Time Frame: 3 years]

Secondary Outcome(s)

Craig's Crohn's Severity Index [Time Frame: 6 months, 1 year, 2 years, 3 years]

Crohn's Disease Endoscopic Index of Severity (CDEIS) [Time Frame: 6 months, 1 year, 2 years, 3 years]

Endoscopic remission [Time Frame: 6 months, 1 year, 2 years, 3 years]

Endoscopic severity scales [Time Frame: 6 months, 1 year, 2 years, 3 years]

Histologic remission on colonoscopy with biopsy [Time Frame: 6 months, 1 year, 2 years, 3 years]

Drug-free clinical remission [Time Frame: 1 year, 2 years, 3 years]

Relapse-free survival [Time Frame: 6 months, 1 year, 2 years, 3 years]

Quality of life short form Survey (SF-36) [Time Frame: 6 months, 1 year, 2 years, 3 years]

Stool markers [Time Frame: 6 months, 1 year, 2 years, 3 years]

Inflammatory Bowel Disease Questionnaire [Time Frame: 6 months, 1 year, 2 years, 3 years]

Secondary ID(s)

DIAD.ATTAC.2018

Source(s) of Monetary Support

Please refer to primary and secondary sponsors