Phase 1
N=1
Safety and Tolerability Of Allogeneic Mesenchymal Stromal Cells in Pediatric Inflammatory Bowel Disease
Inflammatory Bowel Diseases
Bottom Line
View on ClinicalTrials.gov: NCT02150551 ↗Enrolled (actual)
1
Serious AEs
100.0%
Results posted
Jan 2023
Primary outcome: Primary: Number of Subjects Who Experience Serious Adverse Events, Adverse Events, and/or Early Treatment Discontinuations. — 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Allogeneic bone marrow-derived mesenchymal stromal cells (Biological)
- Age
- Pediatric, Adult · 12+ yrs
- Sex
- All
- Sponsor
- Catherine Bollard
- Primary completion
- Sep 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Subjects Who Experience Serious Adverse Events, Adverse Events, and/or Early Treatment Discontinuations. |
1 | — |
| SECONDARY Proportion of Patients With Clinical Response |
— | — |
| SECONDARY Number of Subjects Demonstrating an Improvement of Laboratory Tests Reflecting Systemic Inflammation. |
— | — |
Summary
In this trial, investigators will infuse donor bone marrow mesenchymal stromal cells intravenously, as a treatment for pediatric Crohn's disease or ulcerative colitis that has not responded to conventional therapies. The goals of this study are to test the safety and tolerability of donor mesenchymal stromal cells in children with Inflammatory Bowel Disease.
Mesenchymal stromal cells support the development of blood cells within the bone marrow. When isolated from a donor and infused into an animal or human, they have been demonstrated to travel to areas of inflammation, to alter immune responses, to decrease pro-inflammatory cytokines, and to promote tissue repair. Infusion of these cells does not lead to rejection. These properties lead investigators to hypothesize that that these may be they may be beneficial in treating inflammatory bowel disease.
Eligibility Criteria
Inclusion Criteria
- For the young adult cohort, patients must be ages 17 ≤22 years
- For the pediatric cohort, patients must be ages 12 ≤16 years
- Patients must have moderate-severely active CD or UC (defined in section 2.3), and documented active disease on flexible sigmoidoscopy, colonoscopy or MR enterography within the preceding 2 months.
- Patients who have failed or are intolerant of biologic therapy. Specifically, the patient will have recurrence or persistence of active disease despite current or past treatment with a biologic. At the time of enrollment, study subjects may be currently receiving 5-aminosalicylates, corticosteroids (≤ 20 mg daily or up to 0.5 mg/kg/day if weight 22 years of age
- Pregnant or breastfeeding. Serum pregnancy test must be negative at screening for female subjects of childbearing potential. Urine pregnancy test must remain negative at each of 4 infusion visits.
- Patients with toxic mega-colon or intestinal perforation
- Evidence of autoimmune chronic active hepatitis or sclerosing cholangitis.
- Patients with fever > 39° C or clinically significant active infection within 1 week (i.e. chronic infections including Hepatitis B/C or HIV or acute infections, including urinary tract infection and respiratory tract infection)
- Received an agent not approved by the FDA for marketed use in any indication or any small molecule inhibitors (i.e. naltrexone) within 60 days of enrollment.
- Subjects who are taking greater than 20 mg (or if body weight <40 kg, 0.5 mg/kg) of prednisone daily.
- Clinically significant abnormal biochemical and hematological parameters, including:
- Neutrophil count < 1000 cells/mm3
- Hemoglobin < 8 g/dl
- Platelet count ≤ 130 cells/mm3
- Creatinine ≥ 1.2 x the upper limit of normal
- Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) ≥ 2x the upper limit of normal
- Conjugated bilirubin greater than 1.2. mg/dL
- Has active infection with enteric pathogens as evidenced by positive microbiological culture of stool or C.difficile toxin PCR.
- Had bowel surgery other than perianal procedures (fistulotomy, seton placement, abscess drainage) within 3 months of enrollment.
- Has uveitis
- Has known pulmonary disease, excluding mild intermittent asthma
Data sourced from ClinicalTrials.gov (NCT02150551). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.