Clinical trial NCT04168788: Pharmacogenetic Study of Antimitotic Therapies Involved in Hepatic VOD in Children With Nephroblastoma or ALL
Official title: Pharmacogenetic Study of Antimitotic Therapies Involved in Hepatic Veno-occlusive Disease in Children With Nephroblastoma or Acute Lymphoblastic Leukemia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Hepatic Veno-Occlusive Disease
Nephroblastoma
Acute Lymphoblastic Leukemia
Keywords: Pharmacogenetics
Lead sponsor: University Hospital, Angers
Sponsor class: OTHER_GOV
First posted: 2019-11-19
Enrollment: 150
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: BASIC_SCIENCE

Brief summary: Hepatic veno-occlusive diseases (VOD) during cancer treatment in children are serious toxicities that have occurred with interruptions of chemotherapy and risk of relapse. In addition, these toxicities have a negative impact on the patient's quality of life, serious long-term sequelae and are potentially fatal in children.

The risk factors associated with the occurrence of these complications are, to date, unknown, at the exception to the exposition to certain treatments (6-thioguanine, busulfan, actinomycin D, radiotherapy, etc.). To understand the effects of this toxicity and those of susceptibility to the disease becomes a major issue in the treatment of these children.

Detailed description: Case-control study, nested in two French multicenter cohorts, on pharmacognenetic, biological and clinical susceptibility factors associated with the occurrence of hepatic veno-occlusive disease during the anticancer treatment for nephroblastoma or acute lymphoblastic leukemia, with centralized genetic analysis.

After obtaining consent (patient or parents for minor patients), a blood sample is collected during the routine follow-up consultation and tubes are sent directly to Paris for the pharmacogenetic analysis at the end of the study.

Eligibility criteria: Inclusion Criteria:

* Children aged \< 18 years old at the time of cancer diagnosis
* Having been treated with a single line of treatment for nephroblastoma or ALL, in France between 2000 and 2018, and who did not receive allogeneic hematopoietic stem cell transplantation
* Weight greater than 5 kg at inclusion
* Informed consent dated and signed by the holder of the parental authority (if minor) or by the patient (if major) to take part in the study
* Affiliated to a Social Security scheme

Exclusion Criteria:

* Unavaibility of constitutional DNA
* Person who receive more than one treatment line for nephroblastoma or ALL in childhood or adolescence
* Pregnant, lactating or parturient women
* Person deprived of their liberty by judicial or administrative decision
* Person under psychiatric care under duress
* Person subject to legal protection
* Person unable to express their consent

Primary outcomes:
measure: Correlate pharmacogenetic analysis with veno-occlusive disease.
timeFrame: One day
Secondary outcomes:
measure: Participant characteristics.
timeFrame: One day
Other outcomes:
Locations:
city: Amiens
country: France
facility: Univesity Hostipal of Amiens
city: Bordeaux
country: France
facility: University Hospital of Bordeaux
city: Brest
country: France
facility: University of Brest
city: Dijon
country: France
facility: University Hospital of Dijon
city: Lille
country: France
facility: Centre Oscar Lambret
city: Limoges
country: France
facility: University Hospital of Limoges
city: Marseille
country: France
facility: Hôpital La Timone
city: Nantes
country: France
facility: University Hospital of Nantes
city: Nice
country: France
facility: University Hospital of Nice
city: Paris
country: France
facility: Institut Curie
city: Paris
country: France
facility: Hôpital Trousseau
city: Poitiers
country: France
facility: University Hospital of Poitiers
city: Rennes
country: France
facility: University Hospital of Rennes
city: Saint-Denis
country: France
facility: University Hospital of La Réunion
city: Tours
country: France
facility: University Hospital of Tours
city: Villejuif
country: France
facility: Gustave Roussy
Study officials:
affiliation: University Hospital, Angers
name: Isabelle Pellier, MD
role: PRINCIPAL_INVESTIGATOR
References: