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

NCT ID: NCT03449069

Last Updated: 2024-03-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 1 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-07-23
• Study Completion Date: 2023-12-31

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.

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.

Conditions

Fistula in Ano; Crohn Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

MSC-AFP

This is a single treatment group study. All patients will receive treatment of a stem cell coated fistula plug.

Group Type: EXPERIMENTAL

MSC-AFP

Intervention Type: DRUG

Patients will have a mesenchymal stromal cell coated anal fistula plug placed in their Crohn's perianal fistula tract.

Interventions

MSC-AFP

Patients will have a mesenchymal stromal cell coated anal fistula plug placed in their Crohn's perianal fistula tract.

Intervention Type: DRUG

Other Intervention Names

mesenchymal stromal cell coated anal fistula plug

Eligibility 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)

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Mayo Clinic

OTHER

Sponsor Role: lead

Responsible Party

Michael C. Stephens

Sponsor-Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Michael C Stephens

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

Mayo Clinic in Rochester

Rochester, Minnesota, United States

Countries

United States

Related Links

https://www.mayo.edu/research/clinical-trials

Mayo Clinic Clinical Trials

Other Identifiers

17-010539

Identifier Type: -

Identifier Source: org_study_id