E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
The proposed indication for Prochymal™ is for treatment of subjects with steroid-refractory acute graft versus host disease (GVHD).
|
|
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.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
1) To evaluate the efficacy of Prochymal in subjects experiencing steroid-refractory acute GVHD, Grades II-IV. |
|
E.2.2 | Secondary objectives of the trial |
2) To gather additional information on the safety of Prochymal in subjects experiencing steroid-refractory acute GVHD, Grades II-IV. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Subjects must be 6 months to 60 years of age, inclusive • Subjects who were diagnosed with acute GVHD before day 100 post transplant • Subjects must have Grade II-IV acute GVHD subsequent to allogeneic HSCT or donor leukocyte infusion that has failed to respond to corticosteroid therapy • Subjects must be able to begin Prochymal™ infusion within 3 days of determination of steroid refractory disease. • Subjects must have adequate renal function as defined by: Calculated Creatinine Clearance of >30mL/min using the Cockroft Gault equation • Subjects who are women of childbearing potential, must be non-pregnant, not breast-feeding, and use adequate contraception. Male subjects must use adequate contraception. • Subject must have a minimum Karnofsky Performance Level of at least 30 at the time of study entry. • Subject (or legal representative where appropriate) must be capable of providing written informed consent.
|
|
E.4 | Principal exclusion criteria |
• 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 (e.g., infection, heart failure, pulmonary hypertension, etc.). • Subjects may not receive any other investigational agents to treat acute GVHD concurrently during study participation. • Subject has a known allergy to bovine or porcine products. • Subjects who are not expected to survive at least four weeks (eg, due to ongoing, active infections or other comorbidities).
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary Efficacy Endpoint Complete response with >/= 28 days duration
|
|
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 | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | Yes |
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) | 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
| Information not present in EudraCT |
E.8.4 | The trial involves multiple sites in the Member State concerned | Information not present in EudraCT |
E.8.5 | The trial involves multiple Member States | Information not present in EudraCT |
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 |
|
E.8.7 | Trial has a data monitoring committee | Yes |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 9 |
E.8.9.2 | In all countries concerned by the trial days | 0 |