E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Congenital cytomegalovirus infection.
Sensorineural Hearingloss. |
Congenitale cytomegalovirus infectie.
Sensorineurale Gehoorverlies. |
|
E.1.1.1 | Medical condition in easily understood language |
Congenitale cytomegalovirus infection.
Hearingloss. |
Aangeboren (via de baarmoeder) cytomegalovirus infectie.
Gehoorverlies. |
|
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 | 10010420 |
E.1.2 | Term | Congenital CMV infection |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic 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 |
Investigate whether early valganciclovir treatment of children with SNHL of ≥ 20 dB, unilateral or bilateral, and a confirmed congenital CMV infection can prevent deterioration of the hearing loss at 1 year follow-up. |
Onderzoeken of vroege behandeling met valganciclovir bij kinderen met gehoorverlies van ≥ 20 dB, unilateraal of bilateraal, en een congenitale CMV infectie de verergering van het gehoorverlies bij 1 jaar follow-up kan tegegaan. |
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E.2.2 | Secondary objectives of the trial |
1) Investigate whether early valganciclovir treatment of children with SNHL of ≥ 20 dB, unilateral or bilateral, and a confirmed congenital CMV infection can prevent cognitive and motor retardation. Communicative and speech development will be extensively assessed.
2) Investigate whether early valganciclovir treatment of children with SNHL of ≥ 20 dB, unilateral or bilateral, and a confirmed congenital CMV infection reduces the CMV viral load in urine and blood samples 6 weeks treatment and one week and one year after completion of treatment. At 1 year follow-up solely the urine sample will be investigated for the viral load. |
1) Onderzoeken of vroege behandeling met valganciclovir bij kinderen met gehoorverlies van ≥ 20 dB, unilateraal of bilateraal, en een congenitale CMV infectie cognitieven en bewegings retardatie kan voorkomen. Communicatieve en spraak ontwikkeling worden getest bij 1 jaar follow-up.
2) Onderzoeken of vroege behandeling met valganciclovir bij kinderen met gehoorverlies van ≥ 20 dB, unilateraal of bilateraal, en een congenitale CMV infectie de CMV virale load zal verlagen in urine en bloed dat wekelijks wordt afgenomen gedurende de 7 weken na inclusie, en 1 jaar na inclusie. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Infants with congenital CMV infection, and hearing loss (≥20 dB, in one or both ears).
• Age at time of inclusion is < 12 weeks after birth.
• >37 weeks gestational age.
• Birth weight >2500 gram.
• Parental signed informed consent.
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Kinderen met congenitaal CMV en gehoorverlies (≥20 dB, in een of beide oren).
• Leeftijd bij inclusie < 12 weken na geboorte.
• >37 weken na conceptie.
• Geboorte gewicht >2500 gram.
• Toestemmingsverklaring door ouders ondertekend.
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|
E.4 | Principal exclusion criteria |
• Indications for symptomatic congenital CMV infection based on diagnostics carried out prior to the inclusion of the child in the trial.
• In case during the house visit the presence of a symptomatic CMV infection is doubted, inclusion will be discussed. Depending on the medical history taking, physical examination and laboratory tests inclusion will be decided upon.
• Treatment with other antiviral agents or immunoglobulins.
• Leucopenia < 0,5 x 10*9/L (blood sample tested at t=0).
|
• Indicaties voor een symptomatische congenitale CMV infectie gebaseerd op diagnostiek uitgevoerd voor inclusie van het kind in de studie.
• In geval dat tijdens het huisbezoek een symptomatische CMV infectie mogelijk wordt geacht zal inclusie worden besproken. Afhankelijk van de medische geschiedenis, lichamelijk onderzoek en labuitslagen zal er worden besloten of inclusie mogelijk is.
• Behandeling met andere antiviral middelen of immunoglobulines.
• Leucopenie < 0,5 x 10*9/L (bloed getest bij t=0).
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the status of the sensorineural hearing loss expressed in dB, in children with congenital CMV at 1 year follow-up. |
Het primaire eindpunt is de status van het gehoorverlies (in dB) bij kinderen met congenitaal CMV bij 1 jaar follow-up. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The hearing loss will be evaluated at 1 year follow-up. |
Het gehoorverleis wordt onderzocht bij 1 jaar follow-up. |
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E.5.2 | Secondary end point(s) |
The secondary endpoint is the development of the infants.
The tertiary endpoint is the viral load (in dried blood spots, blood and urine) during 7 weeks after inclusion and at 1 year follow-up. |
Het secundaire eindpunt is de ontwikkeling van de kinderen.
Het tertiaire eindpunt is de viral load (in hielprikkaart, bloed en urine) in de eerste 7 weken na inclusie en bij 1 jaar follow-up. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
The development of the infants will be tested at 1 year follow-up.
The viral load will be determined at inclusion and weekly in urine during the first 7 weeks after inclusion (total 8 moments). For treated infants it will be determined at inclusion and weekly in blood samples (total 8 moments). For untreated infants it will be determined twice in blood (at inclusion and in week 6). At 1 year follow-up the viral load will be determined in urine for all infants. |
De ontwikkeling van de kinderen wordt bij 1 jaar follow-up getest.
De virale load wordt bij inclusie en wekelijks in de urine getest in de eerste 7 weken na inclusie (totaal 8 momenten). Voor behandelde kinderen wordt de virale load bij inclusie en wekelijks beoordeeld in bloed (totaal 8 momenten). Voor onbehandelde kinderen wordt de virale load tweemaal bepaald in het bloed (bij inclusie en in week 6). |
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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 | 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 | 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 | 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 | Yes |
E.8.2.3.1 | Comparator description |
Reguliere behandeling |
Standard care |
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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
| 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 | 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
|
The last visit of the last subject. |
Het laatste bezoek van de laatste proefpersoon. |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |