MedPath

Prenatal Treatment of Congenital Cytomegalovirus Infection With Letermovir Versus Valaciclovir

Phase 3
Recruiting
Conditions
CMV Infected Fetuses
Pregnant Women
Registration Number
NCT05446571
Lead Sponsor
Assistance Publique - Hôpitaux de Paris
Brief Summary

The investigators' hypothesis is that maternal treatment with Letermovir will inhibit fetal CMV replication better than Valaciclovir in infected fetuses and lead to a higher proportion of negative CMV PCR at birth in cord blood.

The main objective is to demonstrate that Letermovir administered to women carrying a CMV infected fetus following a maternal infection of the first trimester increases the proportion of neonates with a negative CMV PCR in neonatal blood collected in the first day of life or in cord blood in case of termination of pregnancy (TOP) compared to Valaciclovir.

In each group , the proportion of asymptomatic neonates and the number and type of long-term sequelae at 2 years will also be assessed and compared.

Detailed Description

15-20% of CMV infected fetuses are symptomatic and up-to 60% of those symptomatic fetuses have postnatal sequelae. Long-term sequelae are essentially neurological deficiencies and hearing loss. Long-term sequelae are mainly seen in fetuses infected following a maternal infection in the first trimester. The physiopathology of brain and inner ear lesions is not completely elucidated but the viral lesions and viral replication play a major role in this altered neurodevelopment. Fetuses with the most severe brain lesions are also those presenting with high CMV replication in the brain and in all other organs. Moreover, placenta infection affects fetal growth causing growth restriction and therefore affects fetal development in that way. Finally, infected fetuses with high blood viral load at diagnosis (around 22 weeks) are more likely to be symptomatic at birth (OR=5.7 IC95% 2.02-16.53). This correlation between symptoms and high levels of viral replication suggests that an antiviral treatment that could efficiently inhibit viral replication could be beneficial.

Neonatal antiviral treatment with Ganciclovir or Valganciclovir has been used for more than 20 years and is recommended for infected neonates that are symptomatic. Two randomized studies demonstrated that this treatment improves hearing and intellectual capacities of symptomatic neonates with central nervous system involvement. However, this improvement is only modest. This modest benefit can probably be explained by the fact that cerebral lesions developed in utero are already fixed in the neonatal period. The investigators' hypothesis is that early prenatal antiviral therapy for infected fetuses at high risk of cerebral lesions will be more efficient to alleviate long-term sequelae than neonatal treatment. The prognosis of fetal infection can now be established upon fetal imaging by ultrasound (US) and MRI, combined with fetal laboratory tests (fetal platelets count and viral load). The prognosis is poor for severe brain lesions and good when imaging and laboratory parameters are normal. In between these extremes, symptomatic fetuses with extra-cerebral or mild cerebral features are an appropriate target for antiviral therapy with the aim to prevent the development of irreversible cerebral injury.

The 3 antiviral drugs (Ganciclovir, Foscarnet and Cidofovir) that are licensed to treat CMV infection and disease in immunosuppressed patients are nucleotide inhibitors and because of their potential carcinogenicity and teratogenicity, they should be avoided in pregnancy. Valaciclovir is efficient to prevent CMV infection in transplanted patients, is safe in pregnancy and crosses the placenta efficiently. The investigators carried a phase II, not randomized, open label clinical trial to test the efficacy of Valaciclovir in infected fetuses. Valaciclovir was given to women carrying a fetus with at least 1 non-severe ultrasound feature from prenatal diagnosis up until delivery. This led to 79% asymptomatic neonates compared to 43% following natural history of the disease. However, the efficacy of Valaciclovir seemed only partial. First, the antiviral effect was partial: although fetal blood viral load decreased with treatment, 90% of treated fetuses still had detectable CMV DNA in cord blood at birth and all had detectable CMV DNA in neonatal saliva and urine. And second, the clinical efficacy was not optimal since only 57% of fetuses with more than 1 ultrasound feature were born asymptomatic, suggestive of Valaciclovir lower efficacy in such cases.

The investigators therefore looked at new anti CMV drugs. Among them only Letermovir has been licensed to prevent CMV disease in transplanted patients in 2018 and will be available in 2019. Letermovir is not a nucleotide inhibitor and has specific anti-CMV activity. In preclinical toxicity studies it was not genotoxic, not teratogenic and did not impair fertility at the recommended human doses. Besides, no specific concern arises from its safety profile in humans. It controls CMV infection and disease in bone marrow transplant patients by achieving blood viral load clearance in 50-80% of cases.

The investigators' hypothesis is that maternal treatment with Letermovir will inhibit fetal CMV replication better than Valaciclovir in symptomatic infected fetuses and lead to a higher proportion of negative CMV PCR at birth in cord blood. Since severity is largely related to viral replication, clearance of viral replication is a valid surrogate endpoint for clinical outcome in such rare and phenotypically variable cases

The investigators' main objective is to demonstrate that Letermovir administered to women carrying a CMV infected fetus following a maternal infection of the first trimester increases the proportion of neonates with a negative CMV PCR in neonatal blood collected in the first day of life or in cord blood in case of termination of pregnancy (TOP) compared to Valaciclovir. The primary endpoint is the proportion of negative CMV PCR (\<500 IU/ml) in neonatal blood collected in the first day of life or in cord blood at termination of pregnancy

The following will also to be compared between the 2 arms : the proportion of asymptomatic neonates, the overall growth, the proportion of long-term sequelae at 2 years of age, the tolerance of treatment for mothers, fetuses and neonates, the maternal adherence to treatment, the evolution of ultrasound features between Day0 and Week 2, Week 4, and Week 6 of treatment, the changes in cerebral and placental features between Day 0 and Week 6 of treatment, using magnetic resonance imaging (MRI), the post-mortem examination in cases with medical termination of pregnancy (TOP).

Recruitment & Eligibility

Status
RECRUITING
Sex
Female
Target Recruitment
46
Inclusion Criteria
  • Pregnant woman ≥ 18 years old,
  • CMV infection in the 1st trimester
  • with an infected fetus at 15 -28 weeks (positive CMV PCR in the amniotic fluid) With a fetus presenting without any severe cerebral ultrasound feature (ventriculomegaly ≥15 mm, hydrocephalus, periventricular hyperechogenicity, microcephaly<-3SD, vermian hypoplasia, porencephaly, lissencephaly, corpus callosum dysgenesis, cystic leukomalacia)
  • affiliation to a social security regime//health insurance
  • Given consent for the study
  • Patient must be able and willing to comply with study visits and procedures

Exclusion Criteria

  • Participation to another interventional drug trial (category 1)
  • Subject protected by law under guardianship or curatorship
  • Maternal CMV infection after 15 weeks'
  • Creatinine clearance <50 ml/mn/1,73m²
  • Liver insufficiency (Child Pugh grade C), AST, ALT 5 x ULN, bilirubin 2 x ULN.
  • Woman with known allergy to Letermovir or Valaciclovir
  • Contraindication for the administration of Letermovir and Valaciclovir listed in the SmPC of Prevymis® and Zelitrex®
  • Women with hypersensitivity to aciclovir
  • Concomitant administration of St John's wort
  • Woman treated by pimozide, ergot alkaloids, dabigatran, atorvastatin, simvastatin, rosuvastatin, pitavastatin or cyclosporin.
  • Woman with hereditary intolerance to galactose, with lactose lapp deficiency, glucose or galactose malabsorption syndrome
Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Primary Outcome Measures
NameTimeMethod
CMV PCR in neonatal blood collectedAt Termination of pregnancy

Negative CMV PCR (\<500 IU/ml) in cord blood

Secondary Outcome Measures
NameTimeMethod
Birthweightat birth
complianceup to 39 weeks

valaciclovir or letermovir concentrations in maternal blood during pregnancy Every 2 follow-up visits and at birth or TOP

fetal assessmentup to 39 weeks

Classification after pathological cerebral examination in severe and non-severe cases during pregnancy

CMV DNA load in fetal bloodup to 39 weeks

in fetal blood by quantitative PCR in IU/mL

CMV DNA load in cord bloodup to 39 weeks

cord blood by quantitative PCR in IU/mL

CMV DNA load in neonatal bloodup to 3 days of life

neonatal blood by quantitative PCR in IU/mL

CMV DNA load in amniotic fluidup to 39 weeks

amniotic fluid by quantitative PCR in IU/mL

CMV DNA load in salivaup to 3 days of life

saliva by quantitative PCR in IU/mL during pregnancy and first days of life

CMV DNA load in urineup to 3 days of life

urine by quantitative PCR in IU/mL during pregnancy and first days of life

parenchymal abnormalities during pregnancyup to 39 weeks

fetal assessment

Number of asymptomatic neonatesin the first day of life
placental weightat birth
number of long-term sequelaeat 2 years of life
type of long-term sequelaeat 2 years of life
maternal full blood countup to 39 weeks

during pregnancy

maternal renal functionup to 39 weeks

during pregnancy

maternal liver functionup to 39 weeks

measurements of liver enzyme (ALAT ASAT GCT PAL) and bilirubin during pregnancy

gestational age at deliveryat birth
neonatal defects non related to infectionin the first day of life
neonatal full blood countin the first day of life
neonatal renal functionin the first day of life
neonatal liver functionin the first day of life
changes in ultrasound featuresup to 39 weeks

changes in ultrasound features as per 4 groups : 1) stable, 2) disappearance or decrease in symptoms, 3) increase or new non-severe symptoms 4) appearance of severe cerebral symptoms during pregnancy and at birth or the end of trial

gyration disorders during pregnancyup to 39 weeks

fetal assessment

white matter abnormalities during pregnancyup to 39 weeks

fetal assessment

ventriculomegaly during pregnancyup to 39 weeks

fetal assessment

changes in placental features on MRIup to 39 weeks

changes in placental features on MRI, measuring placental T2 relaxation time, diffusion parameters and IVIM

brain biometrics during pregnancyup to 39 weeks

fetal assessment

hepatomegaly during pregnancyup to 39 weeks

fetal assessment

splenomegaly during pregnancyup to 39 weeks

fetal assessment

intestinal abnormalities during pregnancyup to 39 weeks

fetal assessment

abnormal amniotic fluid volume during pregnancyup to 39 weeks

fetal assessment

Letermovir concentration in cord bloodat birth or TOP

in cord blood

Letermovir concentration in amniotic fluidat birth or TOP

in amniotic fluid

Letermovir concentration in placentaat birth or TOP

in placenta

Letermovir concentration in neonatal bloodin the first day of life

in neonatal blood

Sequencing of CMV UL56 and UL89 genesin the first day of life

Sequencing of CMV UL56 and UL89 genes in positive neonates for CMV PCR

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