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Phase 1 Crohn's Pediatric Sub-study of MSC AFP

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03014219
Recruitment Status : Withdrawn (FDA approval was not obtained until adult trial completes accrual)
First Posted : January 9, 2017
Last Update Posted : July 6, 2017
Sponsor:
Information provided by (Responsible Party):
William A. Faubion, M.D., Mayo Clinic

Brief Summary:

The investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Bio-A Fistula Plug) in a Phase I study using a single dose of 20 million cells. 20 patients (age 12 to 17 years) with Crohns perianal fistulas will be enrolled.

Subjects will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore fistula plug as per current clinical practice. The subjects will be subsequently followed for fistula response and closure for 24 months. This is an autologous product derived from the patient and used only for the same patient.


Condition or disease Intervention/treatment Phase
Perianal Fistula Drug: Only 1 arm: treatment with MSC-AFP Phase 1

Detailed Description:

Visit 1: Patients will be evaluated for eligibility (inclusion/exclusion checklist) and written, informed consent will be obtained. Patients will undergo general exam with vital signs. Patients will be scheduled for a fat biopsy to collect the tissue needed to grow MSC. In the event there is no cell growth from the tissue obtained from the first biopsy, one further attempt will made from a second tissue sample from this patient. However, if the second attempt fails to grow cells, no further attempts will be made, and the subject will not continue in the study.

The subjects will be subsequently followed for fistula response and closure for 24 months. Study visits are Day 1, Week 2, Week 4, Week 8, Week 12, Week 24, Week 52, and Week 104. This is an autologous product derived from the patient and used only for the same patient.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease: Pediatric Sub-study
Estimated Study Start Date : July 2017
Estimated Primary Completion Date : July 2019
Estimated Study Completion Date : July 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Fistulas

Arm Intervention/treatment
Experimental: Only 1 arm: treatment with MSC-AFP
Single Treatment Group: Eligible patients will be treated with a Gore Bio-A Fistula Plug that has been coated with autologous mesenchymal stromal cells. This is a drug study, specifically phase 1 study of autologous mesenchymal stromal cells. Single dose of 20 million cells.
Drug: Only 1 arm: treatment with MSC-AFP
Eligible patients will be treated with a fistula plug that has been coated with autologous mesenchymal stromal cell. Drug study. Single dose of 20 million cells.
Other Name: mesenchymal stromal cells




Primary Outcome Measures :
  1. Number of Subjects with any Adverse Events that are related to study drug [ Time Frame: [Time Frame: 2-24 months ]
    The primary endpoint of this study is to determine the safety of using adipose derived, autologous mesenchymal stromal cells (MSC) bound to the Gore Bio-A Fistula Plug for treatment of refractory CD perianal fistulae.


Secondary Outcome Measures :
  1. Number of Subjects with healing in response to the study drug treatment [ Time Frame: Time Frame: 2-24 months ]
    The Outcome Measure will be the presence or absence of fistula drainage (Yes vs. No)



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  1. Males and females 12-17 years of age.
  2. Residents of the United States.
  3. Crohn's disease with single or multiple draining complex perianal fistulae (definition as below) for at least three months despite standard therapy (definition below).
  4. Concurrent therapies with corticosteroids, 5-ASA drugs, thiopurines, MTX, antibiotics, and anti-TNF therapy are permitted.
  5. All patients should have undergone a colonoscopy in last 12 months to rule out malignant or premalignant condition
  6. Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia
  7. Ability to comply with protocol
  8. Competent and able to provide written informed consent (and assent where appropriate).
  9. Must have failed standard medical therapy including anti-TNF agents

Exclusion Criteria

  1. Inability to obtain informed consent (and assent where appropriate).
  2. Clinically significant medical conditions within the six months before administration of MSCs: e.g. sepsis, pneumonia active serious infection or other conditions that would, in the opinion of the investigators, compromise the safety of the patient.
  3. Specific exclusions;

    a. Evidence of hepatitis B, C, or HIV

  4. History of cancer including melanoma (with the exception of localized skin cancers)
  5. Investigational drug within thirty (30) days of baseline
  6. A resident outside the United States
  7. Pregnant or trying to become pregnant, or breast feeding.
  8. History of clinically significant auto-immunity (other than Crohn's disease) or any previous example of fat-directed autoimmunity
  9. Previous allergic reaction to a perianal fistula plug.
  10. If adipose tissue is not technically feasible
  11. Weight less than 35 kg
  12. Allergic to local anesthetics
  13. Non-enterocutaneous tracts (i.e. recto-vaginal, entero-vesicular)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03014219


Locations
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United States, Minnesota
Mayo Clinic in Rochester
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
William A. Faubion, M.D.
Investigators
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Principal Investigator: Michael C Stephens Mayo Clinic
Principal Investigator: William Faubion Mayo Clinic
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Responsible Party: William A. Faubion, M.D., Sponsor Investigator, Mayo Clinic
ClinicalTrials.gov Identifier: NCT03014219    
Other Study ID Numbers: 15-005574
First Posted: January 9, 2017    Key Record Dates
Last Update Posted: July 6, 2017
Last Verified: July 2017

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Fistula
Pathological Conditions, Anatomical