Pharmacogenetics of VOD in Children With HSCT

NCT03664427 | Completed

• 1 other identifier: NI16025J
• Study Type: observational
• Target: 436
• Locations: 1 country
• Sites: 1

Brief Summary

This project aims to identify common pharmacogenetic biomarkers predisposing children with cancer to develop hepatic VOD during their cancer treatment including HSCT. The impact of VOD occurrence and significant biomarkers will also be evaluated on outcome at day 100 and one year after HSCT. It should help to highlight factors that can contribute to the initiation of hepatic VOD. Understanding mechanisms of this toxicity and to know individual parameters of disease susceptibility becomes an important issue in the care of these children. The ultimate goal of research in this area would be to develop a personalized predictive medicine and, hopefully, prevent the occurrence of VOD from a therapeutic adaptation to each patient according to his pharmacogenetic profile (adapted prophylaxis, dose adjustment, drug combinations ...). A prospective identification of patients at risk of hepatic VOD will increase the safe use of anticancer.

Conditions

Cancer

Outcome Measures

Primary Outcomes (1)

• Pharmacogenetic biomarkers

  The pharmacogenetic analysis will be conducted by a whole exome genotyping approach with Microarrays Illumina "Human Omni2.5-8 v1.3" (exploring more than 2,600,000 genetic variants covering the entire genome with more than 300,000 genetic biomarkers within exons).

  12 months

Secondary Outcomes (2)

• Survival status at 100 days post HSCT

  100 days

• Survival status at 1 year post HSCT

  12 months

Study Arms (2)

Group 1: Patients with VOD

paediatric patients with Veno-Occlusive Disease (VOD) complicating haematological stem cell transplantation

Group 2: matched controls

Paediatric patients defined as matched controls without veno-occlusive disease complicating haematological stem cell transplantation

Eligibility Criteria

• Age: 6 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

This study concerns children with cancer aged less than 18 years treated for their first HSCT between 2000 and 2011 in France.

You may qualify if:

• Children with cancer aged less than 18 years old treated for their first HSCT between 2000 and 2011 in France.
• Patients are selected from the database ProMise regarding pediatric patients treated in any center of the French Society of Stem Cell transplantation (SFGM).
• Clinical data (age, sex, initial pathology, conditioning treatment, type of graft cells, VOD occurence or not, survival status at 100 days and 1 year after transplantation) were extracted from this database.

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Robert Debre Hospital

Paris, 75019, France

Location

MeSH Terms

Conditions

Neoplasms

Study Officials

• Evelyne Jacqz-Aigrain, MD, PhD

  APHP

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2018
• First Posted: September 10, 2018
• Study Start: January 15, 2019
• Primary Completion: January 15, 2020
• Study Completion: January 15, 2021
• Last Updated: April 18, 2022

Record last verified: 2021-10

Locations

FR (1)