E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Subjects must have steroid refractory treatment of Grades B-D acute GVHD secondary to allogeneic HSCT (stem cells sources: bone marrow, peripheral blood stem cells, or cord blood cells) or DLI. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10018651 |
E.1.2 | Term | Graft versus host disease |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the efficacy of Prochymal in subjects experiencing steroidrefractory Grades B-D acute GVHD |
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E.2.2 | Secondary objectives of the trial |
To gather additional information on the safety of Prochymal in subjects experiencing steroid-refractory Grades B-D acute GVHD. |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Subjects must be 6 months to 60 years of age, inclusive ユ Subjects who were diagnosed with steroid refractory Grades B-D acute GVHD post HSCT or post DLI ユ Subjects must be able to be treated within 3 days (+ 1) of diagnosis of steroid-refractory acute GVHD ユ Subjects must have adequate renal function as defined by: Calculated Creatinine Clearance of >30mL/min using the Cockroft-Gault equation ユ Subjects who are women of childbearing potential, must be non-pregnant, not breast-feeding, and use adequate contraception. Male subjects must use adequate contraception ユ Subject must have a minimum Karnofsky/Lansky Performance Level of at least 30 at the time of study entry ユ Subject (or legal representative where appropriate) must be capable of providing written informed consent. |
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E.4 | Principal exclusion criteria |
Subject has started treatment with second line therapy prior to randomization. ユ Subject has any underlying or current medical or psychiatric condition that, in the opinion of the Investigator, would interfere with the evaluation of the subject including uncontrolled infection, heart failure, pulmonary hypertension, etc. ユ Subjects may not receive any other investigational agents (not approved by the FDA) concurrently during study participation or within 30 days of randomization. ユ Subject has a known allergy to bovine or porcine products. ユ Subject has received a transplant for a solid tumor disease. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Complete Response of major or equal to 28 days duration |
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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 | No |
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 | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 | 0 |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 12 |