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

NCT03014219 | Withdrawn Phase 1 FDA Drug

• 1 other identifier: 15-005574
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Perianal Fistula

Outcome Measures

Primary Outcomes (1)

• 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.

  [Time Frame: 2-24 months

Secondary Outcomes (1)

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

  Time Frame: 2-24 months

Study Arms (1)

Only 1 arm: treatment with MSC-AFP

EXPERIMENTAL

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.

Drug: Only 1 arm: treatment with MSC-AFP

Interventions

Only 1 arm: treatment with MSC-AFP DRUG

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.

Also known as: mesenchymal stromal cells

Only 1 arm: treatment with MSC-AFP

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.
• 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)

Sponsors & Collaborators

• William A. Faubion, M.D.: lead

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Study Officials

• Michael C Stephens

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

• William Faubion

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor Investigator

Study Record Dates

• First Submitted: January 4, 2017
• First Posted: January 9, 2017
• Study Start: July 1, 2017
• Primary Completion: July 1, 2019
• Study Completion: July 1, 2020
• Last Updated: July 6, 2017

Record last verified: 2017-07

Locations

US (1)