Phase 2
Completed N=105
A Study To Investigate The Safety And Possible Clinical Benefit Of Multistem(r) In Patients With Moderate To Severe Ulcerative Colitis
Colitis, Ulcerative
Source: ClinicalTrials.gov NCT01240915 ↗
Enrolled (actual)
105
Serious AEs
22.9%
Results posted
Feb 2016
Primary outcomePrimary: Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8 — 3.13; 3.10; 0.10; -0.30 scores on a scale — p=0.96
Summary
MultiStem(r) is a new biological product, manufactured from human stem cells obtained from adult bone marrow or other nonembryonic tissue sources. Factors expressed by MultiStem cells are believed to reduce inflammation and regulate immune system function, protect damaged or injured cells and tissue, promote formation of new blood vessels, and augment tissue repair and healing. MultiStem cell treatment resulted in significant efficacy in a mouse model of Graft versus Host Disease with almost complete reversal of gastrointestinal pathology (similar to pathology that would be expected in Ulcerative Colitis). These data, together with safety data generated in 2 other clinical trials, suggest that MultiStem has the potential to be a new treatment option for patients with ulcerative colitis. This is the first study of MultiStem in this patient population and will cautiously explore the safety/toleration and potential benefit of this new treatment in patients with moderate to severe disease.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Endoscopic Score (as Measured by Modified Baron Score) at Week 8 |
3.13; 3.10; 0.10; -0.30 | 0.96 |
| PRIMARY Change From Baseline in Rectal Bleeding Mayo Subscore at Week 4 |
1.42; 1.23; -0.44; -0.38 | 0.54 |
| PRIMARY Change From Baseline in Rectal Bleeding Mayo Subscore at Week 8 |
-0.46; -0.43 | 0.67 |
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
2; 3; 0; 4; 5; 20 | — |
| PRIMARY Number of Participants With Treatment-Emergent AEs by System Organ Class (SOC) |
0; 0; 0; 0; 0; 5 | — |
| PRIMARY Number of Treatment-Emergent AEs by Severity |
6; 16; 0; 8; 13; 61 | — |
| SECONDARY Change From Baseline in Rectal Bleeding Mayo Subscore at Week 12 and Week 16 |
NA; NA; NA; NA; NA; -0.77 | — |
| SECONDARY Number of Participants With Laboratory Test Abnormalities |
2; 2; 1; 4; 4; 14 | — |
| SECONDARY Number of Participants With Potentially Clinically Significant Vital Signs Findings |
0; 0; 0; 0; 2; 0 | — |
| SECONDARY Fold Change From Baseline in Fecal Calprotectin at Weeks 4, 8, 12, and 16 |
690.7549; 1189.8169; 739.2300; 898.2758; 874.1363; 576.5796 | 0.53 |
| SECONDARY Fold Change From Baseline in C-Reactive Protein (CRP) at Weeks 4, 8, 12, and 16 |
1.272; 1.527; 0.229; 0.578; 0.180; 0.613 | 0.79 |
| SECONDARY Percentage of Participants With Rectal Bleeding Mayo Subscore of Zero at Weeks 4, 8, 12, and 16 |
50.0; 66.7; 50.0; 33.3; 40.0; 52.2 | 0.34 |
| SECONDARY Percentage of Participants in Endoscopic Remission at Week 8 |
0; 0; 0; 0; 16.7; 4.3 | 0.57 |
| SECONDARY Percentage of Participants in Clinical Remission at Week 8 |
0; 0; 0; 0; 0; 0 | 0.85 |
| SECONDARY Percentage of Participants With Decrease From Baseline of at Least 1 Point in Rectal Bleeding Mayo Subscore at Weeks 4, 8, 12, and 16 |
100; 66.7; 0; 0; 40.0; 43.5 | 0.05 |
| SECONDARY Percentage of Participants With Endoscopic Response at Week 8 |
0; 0; 0; 0; 0; 8.7 | 0.84 |
| SECONDARY Percentage of Participants in Clinical Response at Week 8 |
50.0; 33.3; 0; 0; 0; 4.3 | 0.96 |
| SECONDARY Change From Baseline in Total Mayo Scores at Week 8 |
10.00; 7.33; 8.50; 7.75; 7.50; 8.74 | 0.87 |
| SECONDARY Change From Baseline in Partial Mayo Scores at Weeks 4, 8, 12, and 16 |
7.50; 5.33; 6.00; 5.75; 5.50; 6.22 | 0.80 |
| SECONDARY Change From Baseline in Biopsy Histology Scores at Week 8 |
9.0; 9.7; 11.0; 7.0; 10.8; 9.4 | 0.75 |
| SECONDARY Change From Baseline in Patient-Reported Rectal Bleeding up to Week 16 |
12.50; 8.00; 6.75; 11.75; 8.42; 7.50 | 0.51 |
Eligibility Criteria
Inclusion Criteria
- Subjects must have a documented diagnosis of ulcerative colitis at least 6 months prior to screening.
- Subjects must have active moderate-to-severe ulcerative colitis based on Mayo score.
- Subjects must have Modified Baron endoscopic score of at least 2 determined within 7 days of first dosing.
- Subjects must have failed or be intolerant (as determined by the investigator) of at least one of the following treatments for UC: Oral corticosteroids, azathioprine or 6-mercaptopurine (6-MP), or anti-tumor necrosis factor (TNF) therapy, eg, infliximab or adalimumab.
- Subjects must be on stable steroid doses.
Exclusion Criteria
- Subjects who have abnormal organ and marrow function.
- Subjects with a diagnosis of indeterminate colitis, or clinical findings suggestive of Crohn's disease.
- Subjects who meet Truelove-Witts criteria for severe ulcerative colitis.
- Subjects receiving or who are expected to receive Infliximab or other biologic treatment within 8 weeks of the Day 1 study visit.
- Subjects receiving or who are expected to receive Cyclosporine, mycophenolate, or tacrolimus within 4 weeks of the Day 1 study visit.
Data sourced from ClinicalTrials.gov (NCT01240915). 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. Informational only — not medical advice.