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

Overview

About this study

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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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)

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Michael Stephens, M.D.

Closed for enrollment

More information

Publications

Publications are currently not available
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