E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Inflammatory disease of the gastrointestinal tract |
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10011402 |
E.1.2 | Term | Crohn's disease (colon) |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10003816 |
E.1.2 | Term | Autoimmune disorders |
E.1.2 | System Organ Class | 10021428 - Immune system disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10064147 |
E.1.2 | Term | Gastrointestinal inflammation |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011401 |
E.1.2 | Term | Crohn's disease |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess safety and efficacy of MSC in Crohn’s disease refractory or intolerant to conventional therapies. |
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E.2.2 | Secondary objectives of the trial |
Measuring outcome of Crohn's disease treated by MSC:
1.Clinical response.
2.Remission (defined by CDAI<150)
3.CDAI level
4.CRP levels
5.Fecal calprotectin levels |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1-Age between 18 and 75 years old
2-Crohn’s disease affecting terminal ileum, colon or both with diagnosis confirmed according to Lennard Jones criteria
3-Clinically active disease with a CDAI between 220 and 450 and biologically active disease with a CRP > 5 mg/l and/or fecal calprotectin > 150 microg/g
4-Resistance or intolerance to mesalazine, steroids, purine analogues, methotrexate, infliximab and adalimumab
5-Adequate venous access (central catheter or good peripheral veins)
6-Willingness to sign the informed consent and enter the clinical trial
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E.4 | Principal exclusion criteria |
1-Any condition not fulfilling inclusion criteria
2-Indication for surgery
3-Symptomatic stricture
4-Undrained perianal or intraabdominal abscess
5-Change in mesalazine dosage within the last 4 weeks, change in steroid dosage within the last two weeks, change in immunosuppressant dosage within the last 3 months, use of anti-TNF treatment within the last two months
6-HIV positive
7-Uncontrolled infection, arrhythmia or hypertension
8-Terminal organ failure:
a.Renal: anuria, serious fluid overload, GFR < 30 ml/min, dialysis;
b.Pulmonary: DLCO < 35% and/or receiving supplementary continuous oxygen;
c.Hepatic: Fulminant liver failure, cirrhosis of the liver with evidence of portal hypertension, alcoholic hepatitis, esophageal varices, a history of bleeding esophageal varices, hepatic encephalopathy, uncorrectable hepatic synthetic dysfunction evinced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal liver abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin >3 mg/dL, and symptomatic biliary disease;
d. Cardiac: Symptomatic coronary artery disease or other cardiac failure requiring therapy; ejection fraction <35%; uncontrolled arrhythmia, uncontrolled hypertension
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E.5 End points |
E.5.1 | Primary end point(s) |
To assess clinical response rate (defined by a 100 points decrease in CDAI).
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
To assess clinical response rate (defined by a 100 points decrease in CDAI) at week 8.
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E.5.2 | Secondary end point(s) |
1.Clinical response.
2.Remission (defined by CDAI<150).
3.CDAI level at week.
4.CRP levels.
5.Fecal calprotectin levels. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1.Clinical response. at week 2, 4, 8 and 12,
2.Remission (defined by CDAI<150) at week 2, 4, 8 and 12,
3.CDAI level at week 2, 4, 8 and 12,
4.CRP levels at week 2, 4, 8 and 12,
5.Fecal calprotectin levels at week 2, 4, 8 and 12.
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | |