Crohn Disease, Fistula in Ano
Conditions
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.
Interventions
DRUGMSC-AFP
Patients will have a mesenchymal stromal cell coated anal fistula plug placed in their Crohn's perianal fistula tract.
Sponsors
Mayo Clinic
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 17 Years
Healthy volunteers
No
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)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with treatment-related adverse events (safety and toxicity) | 2 months to approximately at 24 months | Participants will have a health assessment and blood work done at each study visit to monitor for adverse events. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with response to the treatment regarding cessation of drainage from the treated fistula. | 2 months to approximately at 24 months | 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. | Week 2, Week 8, and Week 24 | Participants will have an assessment of healing radiographically by magnetic resonance imaging (MRI) at Week 2 visit, Week 8, and Week 24. |
Countries
United States
Outcome results
None listed