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Pediatric MSC-AFP Sub-study for Crohn's Fistula

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ClinicalTrials.gov Identifier: NCT03449069
Recruitment Status : Recruiting
First Posted : February 28, 2018
Last Update Posted : November 7, 2018
Sponsor:
Information provided by (Responsible Party):
Michael C. Stephens, Mayo Clinic

Brief Summary:
The purpose of this study is to determine the safety of using an autologous mesenchymal stromal cell (MSC) coated fistula plug in people with fistulizing Crohn's disease. Autologous means these cells to coat the plug come from the patient.

Condition or disease Intervention/treatment Phase
Fistula in Ano Crohn Disease Drug: MSC-AFP Phase 1

Detailed Description:

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. 5 patients (age 12 to 17 years) with Crohn's 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.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
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
Actual Study Start Date : July 23, 2018
Estimated Primary Completion Date : February 28, 2020
Estimated Study Completion Date : February 28, 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: MSC-AFP
This is a single treatment group study. All patients will receive treatment of a stem cell coated fistula plug.
Drug: MSC-AFP
Patients will have a mesenchymal stromal cell coated anal fistula plug placed in their Crohn's perianal fistula tract.
Other Name: mesenchymal stromal cell coated anal fistula plug




Primary Outcome Measures :
  1. Number of participants with treatment-related adverse events (safety and toxicity) [ Time Frame: 2 months to approximately at 24 months ]
    Participants will have a health assessment and blood work done at each study visit to monitor for adverse events.


Secondary Outcome Measures :
  1. Number of participants with response to the treatment regarding cessation of drainage from the treated fistula. [ Time Frame: 2 months to approximately at 24 months ]
    Participants will have a clinical assessment of fistula drainage at each study visit.

  2. Number of participants with radiographic response to the treatment regarding the treated fistula. [ Time Frame: Week 2, Week 8, and Week 24 ]
    Participants will have an assessment of healing radiographically by magnetic resonance imaging (MRI) at Week 2 visit, Week 8, and Week 24.



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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-aminosalicylate (5-ASA) drugs, thiopurines, methotrexate (MTX), antibiotics, and anti-tumor necrosis factor (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): NCT03449069


Contacts
Contact: Jessica Friton 507-284-0495 friton.jessica@mayo.edu

Locations
United States, Minnesota
Mayo Clinic in Rochester Recruiting
Rochester, Minnesota, United States, 55905
Contact: Jessica Friton    507-284-0495    friton.jessica@mayo.edu   
Principal Investigator: Michael C Stephens         
Sponsors and Collaborators
Mayo Clinic
Investigators
Principal Investigator: Michael C Stephens Mayo Clinic

Responsible Party: Michael C. Stephens, Sponsor-Investigator, Mayo Clinic
ClinicalTrials.gov Identifier: NCT03449069     History of Changes
Other Study ID Numbers: 17-010539
First Posted: February 28, 2018    Key Record Dates
Last Update Posted: November 7, 2018
Last Verified: November 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Crohn Disease
Fistula
Inflammatory Bowel Diseases
Gastroenteritis
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases
Pathological Conditions, Anatomical