E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment and prevention of CMV disease in kidney transplant recipients |
|
E.1.1.1 | Medical condition in easily understood language |
Treatment and prevention of Human cytomegalovirus (CMV), a serious infection complicating kidney transplantation |
|
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 | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10049107 |
E.1.2 | Term | CMV viraemia |
E.1.2 | System Organ Class | 100000004862 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10009703 |
E.1.2 | Term | CMV infection |
E.1.2 | System Organ Class | 100000004862 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 15.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10009701 |
E.1.2 | Term | CMV |
E.1.2 | System Organ Class | 100000004848 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10060577 |
E.1.2 | Term | CMV viremia |
E.1.2 | System Organ Class | 100000004862 |
|
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 |
• The primary objective of this study is to describe the tolerability profile of up to 200 days prophylaxis of valganciclovir oral solution and tablets in pediatric kidney transplant recipients. |
|
E.2.2 | Secondary objectives of the trial |
• To describe the incidence of CMV infection (viremia) and disease (CMV syndrome or tissue invasive CMV) within the first 52 weeks post transplant.
• To describe the incidence and nature of CMV resistance to ganciclovir (mutations in UL97 and/or UL54). |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Parent or guardian of patient willing and able to give written informed consent; the written assent from the child is also required if he/she is old enough to understand the risks and benefits of the study.
2. Patient has received a kidney transplant.
3. Males or females aged between 4 months and ≤ 16 years.
4. Patient at risk of developing CMV disease (including R+ who are at
risk of CMV due to other factors determined by the treating
physician).
5. Patient has adequate hematological and renal function defined as:
• Absolute neutrophil count (ANC) >1300 cells/μL
• Platelet count >40,000 cells/μL
• Hemoglobin >8.0 g/dL
• Estimated Schwartz creatinine clearance (>15 mL/min) to allow for dosing according to algorithm
6. Patient is able to tolerate oral medication (any standard practice tube
feeding is acceptable).
7. Negative pregnancy test (blood or urine) for females of child bearing
potential before initiation of valganciclovir treatment.
8. Patients of reproductive potential agree to utilize an effective method
of contraception throughout the study period and for 90 days following discontinuation of study drug (abstinence is a valid method of contraception). |
|
E.4 | Principal exclusion criteria |
1. Patient has exhibited an allergic or other significant adverse reaction to acyclovir, valaciclovir, ganciclovir or valganciclovir (or excipients) in the past.
2. Patient has severe, uncontrolled diarrhea (more than 5 watery stools
per day).
3. Patient has liver enzyme elevation of more than five times the upper
limit of normal for AST (SGOT) or ALT (SGPT).
4. Patient requires use of any protocol prohibited concomitant
medications.
5. Patient has previously participated in this clinical trial.
6. Patient is a lactating female who will not discontinue nursing prior to
study entry.
7. Patient is simultaneously participating in another clinical trial except as approved by the Sponsor. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
To describe the tolerability profile of up to 200 days prophylaxis of valganciclovir oral solution and tablets in pediatric kidney transplant recipients |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Up to 52 weeks post-transplant |
|
E.5.2 | Secondary end point(s) |
To describe the incidence of CMV infection and disease within the first 52 weeks post-transplant
To describe the incidence and nature of resistance to ganciclovir |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Up to 52 weeks post-transplant
|
|
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 | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
|
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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.8.3 |
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 | 12 |
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 |
Australia |
Brazil |
France |
Germany |
Mexico |
Spain |
Sweden |
United Kingdom |
United States |
|
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
|
The end of the study is defined as the last patients’ last visit. |
|
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 | 10 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 11 |
E.8.9.2 | In all countries concerned by the trial days | 15 |