AlloGeneic Human Mesenchymal Stem Cells (hMSC) in PAtients With FistuLizing Crohn's Disease Via PErifistula iNjEctions (GALENE)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02677350
• Date of registration: 01/02/2016
• Prospective Registration: Yes
• Primary sponsor: Joshua M Hare
• Public title: AlloGeneic Human Mesenchymal Stem Cells (hMSC) in PAtients With FistuLizing Crohn's Disease Via PErifistula iNjEctions (GALENE) GALENE
• Scientific title: A Phase I, Pilot Trial to Evaluate the Safety and Efficacy of Injection of Allogeneic Mesenchymal Bone-Marrow Derived Human Stem Cells in Patients With Fistulizing Crohn's Disease.
• Date of first enrolment: December 1, 2019
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT02677350
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: David Kerman, MD
• Affiliation: ISCI / University of Miami

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Provide written informed consent.

2. Male and Female subjects = 18 years of age at the time of signing the Informed Consent
Form.

3. Subjects with Fistulizing Crohn´s disease with complex perianal fistula, multiple
perianal fistulas, or rectovaginal fistula(s). The complex perianal fistula is defined
as a trans-sphincteric, supra-sphincteric or an extra-sphincteric tract. Patients with
multiple fistulas, "horseshoe" fistula," or any fistula with fecal incontinence as a
result of the Crohn's disease itself or because of previous anal fistula surgery that
cannot have more surgery are also eligible.

4. If drainage of abscess is needed, it should be done 2 or more weeks prior to onset of
therapy.

5. Have had Crohn's Disease (CD) diagnosed at least 6 months prior to enrollment based on
clinical, endoscopic, anatomic/pathologic and/or radiologic criteria.

6. Have a CDAI score <350.

7. During the course of the subject's Crohn's disease (CD), subject must have received
anti-Tumor Necrosis Factor (TNF) agents or immunomodulators which did not heal the CD
fistulas. If anti-TNFs or immunomodulators are contraindicated or led to adverse
events, patients must have failed conservative therapy with antibiotics, or setons, or
surgical intervention.

8. Subject who are currently receiving anti-TNFs, antibiotics, 5-aminosalicylic acid,
azathioprine, 6-mercaptopurine, methotrexate, prednisone, or any similar drugs at the
time of enrollment as long as the following criteria are met:

1. The patient must have been on the anti-TNF for at least 4 months

2. The dose of 5-aminosalicylic acid (5-ASA) must have been stable for at least 4
weeks prior to enrollment.

3. The dose of steroids must have been stable for at least 2 weeks prior to
enrollment.

4. The dose of antibiotics must have been stable for at least 2 weeks prior to
enrollment.

5. The dose of immunomodulators (for example, azathioprine, 6-mercaptopurine, or
methotrexate) must have been stable for at least 8 weeks prior to enrollment and
the subject on therapy for at least three months prior to enrollment

9. Patient is, in the investigator's opinion, willing and able to comply with the
protocol requirements

Exclusion Criteria:

In order to participate in this study, a patient Must Not:

1. Have a known, serious radiographic contrast allergy (gadolinium in particular)

2. Have a hematologic abnormality as evidenced by hematocrit < 25%, white blood cell <
2,500/ul or platelet values < 100,000/ul without another explanation.

3. Have liver dysfunction, as evidenced by enzymes (AST and ALT) greater than three times
the Upper limit normal.

4. Have a coagulopathy (International Normalized ratio (INR) > 1.3) not due to a
reversible cause (i.e., Coumadin). Patients on Coumadin will be withdrawn 5 days
before the procedure and confirmed to have an INR < 1.3. Patients who cannot be
withdrawn from Coumadin will be excluded from enrollment.

5. Bone marrow dysfunction, as evidenced by a 20% or more deviation from normal
hematocrit, white blood cell count or platelet values without another explanation.

6. Be an organ transplant recipient.

7. Clinical history of malignancy within 5 years (i.e., patients with prior malignancy
must be disease free for 5 years), except curatively-treated basal cell carcinoma,
squamous cell carcinoma, or cervical carcinoma.

8. Non-cardiac condition that limits lifespan to < 1 year.

9. Patients with a highly active luminal CD, i.e., if they meet any of the following
criteria: - Presence of severe proctitis (prominent friability, spontaneous bleeding,
multiple erosions, deep ulcers) or very active luminal disease that requires immediate
treatment, revealed by colonoscopy.

10. Have anal dysplasia

11. Patients that have received radiation to the pelvic/perianal area.

12. Presence of abscess or other collections not drained (revealed by baseline radiologic
study).

13. Presence of setons unless they are removed before treatment beginning.

14. Rectal and/ or anal stenosis that cannot be adequately evaluated for dysplasia by
Examination under anesthesia or endoscopy.

15. Need surgery in the perianal region for reasons other than fistulas at inclusion or
within 16 weeks after treatment administration.

16. Had a stable dose of an anti-TNF agent within the past 8 weeks before the cell
treatment administration.

17. Taking tacrolimus or cyclosporine and not on a stable maintenance dose for 2 weeks
before the start of scheduled interventions.

18. Have a history of alcohol or other addictive substances abuse within 6 months before
inclusion.

19. Severe uncontrolled diseases (chronic renal failure, cardiovascular, pulmonary or any
systemic disease).

20. Any type of medical or psychiatric disease which are considered as exclusion criteria,
in the investigator's opinion.

21. Subjects with congenital or acquired immunodeficiency.

22. Positive serology for Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV) or
Hepatitis C Virus (HCV), Human papillomavirus (HPV) or Herpes Virus.

23. Had major surgery or serious traumatism within 6 weeks of enrollment.

24. Impossibility of doing an radiological exploration (reaction to contrast material,
pacemakers, claustrophobia, etc.)

25. Have hypersensitivity to dimethyl sulfoxide (DMSO)

26. Be currently participating (or participated within the previous 30 days) in an
investigational therapeutic or device trial.

27. Be a female who is pregnant, nursing, or of childbearing potential while not
practicing effective contraceptive methods. Female patients must undergo a blood or
urine pregnancy test at screening and within 36 hours prior to injection.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fistulizing Crohn's Disease

Stem Cells

Crohn's Disease

Intervention(s)

Drug: Allogeneic Bone Marrow derived Human Mesenchymal Stem Cells (hMSCs)

Primary Outcome(s)

Evaluation of treatment emergent adverse events from the bone marrow-derived allogeneic mesenchymal stem cells implant [Time Frame: At each intervention and 7, 10, and 16 months after last intervention]

Secondary Outcome(s)

Clinically: to assess changes in the Crohn's Disease Activity Index (CDAI), the Perianal Disease Activity Index (PDAI). [Time Frame: At each intervention and 7, 10, and 16 months after last intervention]

Evaluate symptomatic patients. [Time Frame: Month 4 and Month 16]

Evaluate the effect of local treatment with allogeneic bmMSCs using the short Inflammatory Bowel Disease Questionnaire (sIBDQ) [Time Frame: At each intervention and 7, 10, and 16 months after last intervention]

Major Adverse Events [Time Frame: At each intervention and 7, 10, and 16 months after last intervention]

C-reactive protein (CRP) [Time Frame: At each intervention and 7, 10, and 16 months after last intervention]

Evaluate the effect of local treatment with allogeneic bmMSCs using the Short Form (SF)-36 score [Time Frame: At each intervention and 7, 10, and 16 months after last intervention]

Endoscopic assessment of rectum using a limited simplified endoscopic activity score for Crohn's disease (SES-CD) to evaluate extent and severity of ulcers. [Time Frame: At each intervention and 7, 10, and 16 months after last intervention]

Radiologic assessment using MRI [Time Frame: Baseline, Month 4, and 16 months only if fistula closure has not been achieved at month 4]

Secondary ID(s)

20150965

Source(s) of Monetary Support

Please refer to primary and secondary sponsors