ALL SCTped FORUM - Pharmacogenomic Study (add-on Study)
Allogeneic Stem Cell Transplant for Children and Adolescents With Acute Lymoblastic Leukemia FORUM - Pharmacogenomic Study (add-on Study)
3 other identifiers
interventional
1,000
1 country
4
Brief Summary
Pharmacogenomics (PG) offers the opportunity to individualize treatment according to patient genetic variations which influence activity of enzyme metabolizing or acting in the pathway of prescribed chemotherapy drugs. This add-on research aims to prospectively investigate variations in several candidate genes related to all types of chemotherapeutic drugs and TBI used in the main related study NCT 01949129, THE ALL SCTped FORUM study for their potential role as predictive biomarkers of PK variability and outcome of myeloablative therapy for pediatric patients receiving an allogeneic hematopoietic stem cell transplantation in acute lymphoblastic leukemia.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_4
Started Apr 2013
Longer than P75 for phase_4
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2013
CompletedFirst Submitted
Initial submission to the registry
July 3, 2015
CompletedFirst Posted
Study publicly available on registry
February 2, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2026
CompletedApril 16, 2019
April 1, 2019
8 years
July 3, 2015
April 15, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Genetic variants in participants as a marker of risk of Adverse events and/or Efficacy of the studied agents
Genotyping of candidates genes related to pharmacokinetics and pharmacodynamics of the studied agents. Association study between the herein genetic variants and the below mentioned phenotypes (odd ratio).
through study completion, an average of 2 years
Secondary Outcomes (10)
Number of participants with acute Graft-versus-host disease (aGvHD) according to the Glucksberg scale and Seattle criteria
18 months after inclusion of first patient, afterwards, annually up to 10 years
Number of participants with chronic Graft-versus-host disease (cGvHD) according to the Glucksberg scale and Seattle criteria
18 months after inclusion of first patient, afterwards, annually up to 10 years
Number of participants with VOD/SOS according to the Seattle criteria
18 months after inclusion of first patient, afterwards, annually up to 10 years
Number of participants with Neutrophil recovery as a measure of Safety and Tolerability
18 months after inclusion of first patient, afterwards, annually up to 10 years
Number of participants with Platelet recovery as a measure of Safety and Tolerability
18 months after inclusion of first patient, afterwards, annually up to 10 years
- +5 more secondary outcomes
Other Outcomes (8)
Administered Bu dose(mg) per day
Measures assessed at time of conditioning (up to 5days)
Target Bu plasma concentration(ng/ml)
Measures assessed at time of conditioning (up to 5days)
Target Bu Area under the plasma concentration versus time curve (AUC) (min*ng/ml)
Measures assessed at time of conditioning (up to 5days)
- +5 more other outcomes
Study Arms (3)
Total body irradiation (TBI)
EXPERIMENTALThe Pharmacogenomics add-on requires 2X 5ml blood EDTA for further DNA analyses. Note this is an add-on study to NCT 01949129, THE ALL SCTped FORUM study. Please refer to the main study for further details.
Busulfan
EXPERIMENTALThe Pharmacogenomics add-on requires 2X 5ml blood EDTA for further DNA analyses. Note this is an add-on study to NCT 01949129, THE ALL SCTped FORUM study. Please refer to the main study for further details.
Treosulfan
EXPERIMENTALThe Pharmacogenomics add-on requires 2X 5ml blood EDTA for further DNA analyses. Note this is an add-on study to NCT 01949129, THE ALL SCTped FORUM study. Please refer to the main study for further details.
Interventions
Blood samples (2x5ml EDTA tubes) should be collected just before the start of the conditioning regimen from every patient regardless of therapeutic arm by every centre and stored ≤-20°C Patient should be in remission (MRD negative) for this sampling, otherwise the sample should be taken using a mouth swab/saliva (not intravenously). For second transplant patients, please provide DNA taken before first transplant or a fresh saliva samples.
Bu PK analysis after the first dose of IV Bu (+potential subsequent ones). Blood sampling: -\>For Bu 4 X/d: Before the first Bu dose (Time 0), then straight after the end of infusion (Time 1), then at 15 min (Time 2), 30 min (Time 3), 1 hour (Time 4) and 4 hour (Time 5) after the end of infusion -\>For Bu 1 X/d: Before the first Bu dose (Time 0), then straight after the end of infusion (Time 1), then at 1 hour (Time 2), 3 hour (Time 3), 5 hour (Time 4), 7 hour (Time 5) and 11 hour (Time 6) after the end of infusion. For centers not performing BU TDM, perform Dried Blood Sampling (DBS) analysis: -\> 0.5ml blood sample should be collected and 5µl spotted onto DBS cards in duplicate. Dry them max 5 hours and then keep in a sealed envelope and store at -80°C, as below
Eligibility Criteria
You may qualify if:
- Patients with ALL (except for patients with B-ALL)
- indication for allogeneic HSCT
- complete remission (CR) before HSCT
- written consent of the parents (legal guardian) and, if necessary, the minor patient via "Informed Consent Form"
- no pregnancy
- no secondary malignancy
- no previous HSCT
- HSCT is performed in a study participating centre
You may not qualify if:
- Non Hodgkin-Lymphoma
- ALL with extramedullary involvement with indication for TBI
- CNS involvement at the timepoint of screening
- Trisomy 21
- The whole protocol or essential parts are declined either by patient himself/herself or the respective legal guardian
- No consent is given for saving and propagation of anonymous medical data for study reasons
- Severe concomitant disease that does not allow treatment according to the protocol at the investigator's discretion (e.g. malformation syndromes, cardiac malformations, metabolic disorders)
- Karnofsky / Lansky score \< 50%
- Subjects unwilling or unable to comply with the study procedures
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Swiss Pediatric Oncology Grouplead
- ALL SCTped Forumcollaborator
Study Sites (4)
Hôpital Cantonal de Genève, Département de Pédiatrie
Geneva, Cansearch Laboratory, 1211, Switzerland
Universitäts-Kinderspital beider Basel (UKBB), Onkologie/Hämatologie
Basel, 4056, Switzerland
HUG Hôpitaux Universitaire de Genève, Unité d'onco-hématologie pédiatrique
Geneva, 1211, Switzerland
Universitäts-Kinderspital
Zurich, 8032, Switzerland
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Christina Peters, Prof. MD PhD
St. Anna Kinderspital, Vienna, Austria
- STUDY CHAIR
Peter Bader, Prof. MD PhD
Klinik für Kinder- u. Jugendheilkunde III, Johann Wolfgang Goethe Universität, Frankfurt, Germany
- STUDY CHAIR
Franco Locatelli, Prof. MD PhD
Department of Pediatric Hematology and Oncology IRCCS Ospedale, Rome, Italy
- PRINCIPAL INVESTIGATOR
Marc Ansari, MD, PD
Département de Pédiatrie, Hôpital Cantonal de Genève
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- BASIC SCIENCE
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 3, 2015
First Posted
February 2, 2016
Study Start
April 1, 2013
Primary Completion
April 1, 2021
Study Completion
April 1, 2026
Last Updated
April 16, 2019
Record last verified: 2019-04