E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
human cytomegalovirus (HCMV) in stem cell transplant patients |
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E.1.1.1 | Medical condition in easily understood language |
human cytomegalovirus (HCMV) in stem cell transplant patients |
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E.1.1.2 | Therapeutic area | Diseases [C] - Virus Diseases [C02] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10009703 |
E.1.2 | Term | CMV infection |
E.1.2 | System Organ Class | 100000004862 |
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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 |
1. To assess the efficacy of CSJ148 on preventing active HCMV infection during the first 98 days after stem cell transplant.
2. To assess the safety and tolerability of CSJ148 when administered to stem cell transplant recipients. |
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E.2.2 | Secondary objectives of the trial |
1. To assess if CSJ148 can increase the time to start preemptive therapy.
2. To assess if CSJ148 can reduce the number of times that preemptive therapy is required.
3. To assess if CSJ148 can reduce the proportion of patients developing HCMV disease.
4. To assess the PK of CSJ148 in stem cell transplant recipients. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inclusion Criteria:
- Male and female patients at least 18 years of age scheduled to undergo allogeneic bone marrow, peripheral blood stem cell, or cord blood transplantation and begin conditioning chemotherapy within 24-48 hours of planned dosing day.
- Subject seropositive for HCMV before transplantation; donor can be seropositive or seronegative for HCMV (donor positive/recipient positive or donor negative/recipient positive).
“other protocol-defined inclusion criteria may apply” |
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E.4 | Principal exclusion criteria |
Exclusion Criteria:
- Have had HCMV-related organ disease within 6 months prior to enrollment.
- Detectable HCMV infection (positive pp65 antigenemia or plasma HCMV DNA polymerase chain reaction (PCR) assays prior to enrollment
- Received any of the following within 30 days prior to enrollment: ganciclovir, valganciclovir, foscarnet, cidofovir, acyclovir (>25 mg/kg/day IV), valacyclovir (>3 gm/day oral), famciclovir (>1500 mg/day oral), HCMV immune globulin, immune globulin (>500 mg/kg), or any other medication with anti-HCMV activity.
- Require mechanical ventilation within 7 days prior to enrollment.
- Received any vasopressors or other agents for hemodynamic support within 7 days prior to enrollment.
- Impaired renal function requiring dialysis.
“other protocol-defined exclusion criteria may apply” |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Number of participants who require preemptive HCMV therapy
2. Number of participants with adverse events as a measure of safety and tolerability |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1. Time to initiation of preemptive HCMV therapy
2. Number of times that preemptive HCMV therapy is required
3. Number of participants developing HCMV disease
4. Area under the serum concentration-time curve during the dosing interval (AUCtau)
5. Maximum serum concentration during the dosing interval (Cmax)
6. Trough serum concentration (Ctrough) prior to the next dose administration |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
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 | 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.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 6 |
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 | No |
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 |
Belgium |
Germany |
United States |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |