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

Primary Purpose

Perianal Fistula

Status

Withdrawn

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Only 1 arm: treatment with MSC-AFP

About this trial

This is an interventional treatment trial for Perianal Fistula

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-ASA drugs, thiopurines, MTX, antibiotics, and anti-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 Rochester

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Only 1 arm: treatment with MSC-AFP

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of Subjects with any Adverse Events that are related to study drug

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

Number of Subjects with healing in response to the study drug treatment

The Outcome Measure will be the presence or absence of fistula drainage (Yes vs. No)

Full Information

NCT ID

NCT03014219

First Posted

January 4, 2017

Last Updated

July 3, 2017

Sponsor

William A. Faubion, M.D.

1. Study Identification

Unique Protocol Identification Number

NCT03014219

Brief Title

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

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

July 2017

Overall Recruitment Status

Withdrawn

Why Stopped

FDA approval was not obtained until adult trial completes accrual

Study Start Date

July 2017 (Anticipated)

Primary Completion Date

July 2019 (Anticipated)

Study Completion Date

July 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

William A. Faubion, M.D.

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 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.

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.

6. Conditions and Keywords

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

Perianal Fistula

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Only 1 arm: treatment with MSC-AFP

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Only 1 arm: treatment with MSC-AFP

Other Intervention Name(s)

mesenchymal stromal cells

Intervention Description

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.

Primary Outcome Measure Information:

Title

Number of Subjects with any Adverse Events that are related to study drug

Description

Time Frame

[Time Frame: 2-24 months

Secondary Outcome Measure Information:

Title

Number of Subjects with healing in response to the study drug treatment

Description

The Outcome Measure will be the presence or absence of fistula drainage (Yes vs. No)

Time Frame

Time Frame: 2-24 months

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-ASA drugs, thiopurines, MTX, antibiotics, and anti-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)

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Michael C Stephens

Organizational Affiliation

Mayo Clinic

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

William Faubion

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

12. IPD Sharing Statement

Learn more about this trial

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

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