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

Primary Purpose

Fistula in Ano, Crohn Disease

Status

Recruiting

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

MSC-AFP

About this trial

This is an interventional treatment trial for Fistula in Ano

Eligibility Criteria

12 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria

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

Exclusion Criteria

Inability to obtain informed consent (and assent where appropriate).
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.
Specific exclusions;
a. Evidence of hepatitis B, C, or HIV
History of cancer including melanoma (with the exception of localized skin cancers)
Investigational drug within thirty (30) days of baseline
A resident outside the United States
Pregnant or trying to become pregnant, or breast feeding.
History of clinically significant auto-immunity (other than Crohn's disease) or any previous example of fat-directed autoimmunity
Previous allergic reaction to a perianal fistula plug.
If adipose tissue is not technically feasible
Weight less than 35 kg
Allergic to local anesthetics
Non-enterocutaneous tracts (i.e. recto-vaginal, entero-vesicular)

Sites / Locations

Mayo Clinic in RochesterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

MSC-AFP

Arm Description

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

Outcomes

Primary Outcome Measures

Number of participants with treatment-related adverse events (safety and toxicity)

Participants will have a health assessment and blood work done at each study visit to monitor for adverse events.

Secondary Outcome Measures

Number of participants with response to the treatment regarding cessation of drainage from the treated fistula.

Participants will have a clinical assessment of fistula drainage at each study visit.

Number of participants with radiographic response to the treatment regarding the treated fistula.

Participants will have an assessment of healing radiographically by magnetic resonance imaging (MRI) at Week 2 visit, Week 8, and Week 24.

Full Information

NCT ID

NCT03449069

First Posted

February 22, 2018

Last Updated

October 2, 2023

Sponsor

Mayo Clinic

1. Study Identification

Unique Protocol Identification Number

NCT03449069

Brief Title

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

Official Title

A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease: Pediatric Sub-study

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Recruiting

Study Start Date

July 23, 2018 (Actual)

Primary Completion Date

December 31, 2023 (Anticipated)

Study Completion Date

December 31, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Mayo Clinic

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Fistula in Ano, Crohn Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

5 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

MSC-AFP

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

MSC-AFP

Other Intervention Name(s)

mesenchymal stromal cell coated anal fistula plug

Intervention Description

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

Primary Outcome Measure Information:

Title

Number of participants with treatment-related adverse events (safety and toxicity)

Description

Participants will have a health assessment and blood work done at each study visit to monitor for adverse events.

Time Frame

2 months to approximately at 24 months

Secondary Outcome Measure Information:

Title

Number of participants with response to the treatment regarding cessation of drainage from the treated fistula.

Description

Participants will have a clinical assessment of fistula drainage at each study visit.

Time Frame

2 months to approximately at 24 months

Title

Number of participants with radiographic response to the treatment regarding the treated fistula.

Description

Participants will have an assessment of healing radiographically by magnetic resonance imaging (MRI) at Week 2 visit, Week 8, and Week 24.

Time Frame

Week 2, Week 8, and Week 24

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria Males and females 12-17 years of age. Residents of the United States. Crohn's disease with single or multiple draining complex perianal fistulae (definition as below) for at least three months despite standard therapy (definition below). Concurrent therapies with corticosteroids, 5-aminosalicylate (5-ASA) drugs, thiopurines, methotrexate (MTX), antibiotics, and anti-tumor necrosis factor (TNF) therapy are permitted. All patients should have undergone a colonoscopy in last 12 months to rule out malignant or premalignant condition Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia Ability to comply with protocol Competent and able to provide written informed consent (and assent where appropriate). Must have failed standard medical therapy including anti-TNF agents Exclusion Criteria Inability to obtain informed consent (and assent where appropriate). 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. Specific exclusions; a. Evidence of hepatitis B, C, or HIV History of cancer including melanoma (with the exception of localized skin cancers) Investigational drug within thirty (30) days of baseline A resident outside the United States Pregnant or trying to become pregnant, or breast feeding. History of clinically significant auto-immunity (other than Crohn's disease) or any previous example of fat-directed autoimmunity Previous allergic reaction to a perianal fistula plug. If adipose tissue is not technically feasible Weight less than 35 kg Allergic to local anesthetics Non-enterocutaneous tracts (i.e. recto-vaginal, entero-vesicular)

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Jessica Friton

Phone

507-284-0495

friton.jessica@mayo.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Michael C Stephens

Organizational Affiliation

Mayo Clinic

Official's Role

Principal Investigator

Facility Information:

Facility Name

Mayo Clinic in Rochester

City

Rochester

State/Province

Minnesota

ZIP/Postal Code

55905

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Jessica Friton

Phone

507-284-0495

friton.jessica@mayo.edu

First Name & Middle Initial & Last Name & Degree

Michael C Stephens

12. IPD Sharing Statement

Plan to Share IPD

Links:

URL

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

Description

Mayo Clinic Clinical Trials

Learn more about this trial

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

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