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

NCT03449069 | Completed Phase 1 FDA Drug

• 1 other identifier: 17-010539
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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.

Conditions

Fistula in Ano; Crohn Disease

Outcome Measures

Primary Outcomes (1)

• 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.

  2 months to approximately at 24 months

Secondary Outcomes (2)

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

  2 months to approximately at 24 months

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

  Week 2, Week 8, and Week 24

Study Arms (1)

MSC-AFP

EXPERIMENTAL

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

Drug: MSC-AFP

Interventions

MSC-AFP DRUG

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

Also known as: mesenchymal stromal cell coated anal fistula plug

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-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

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

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Rectal Fistula; Crohn Disease

Condition Hierarchy (Ancestors)

Intestinal Fistula; Digestive System Fistula; Digestive System Diseases; Intestinal Diseases; Gastrointestinal Diseases; Rectal Diseases; Fistula; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Inflammatory Bowel Diseases; Gastroenteritis

Study Officials

• Michael C Stephens

  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: PRINCIPAL INVESTIGATOR
• PI Title: Sponsor-Investigator

Study Record Dates

• First Submitted: February 22, 2018
• First Posted: February 28, 2018
• Study Start: July 23, 2018
• Primary Completion: December 31, 2023
• Study Completion: December 31, 2023
• Last Updated: March 13, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)