Personalized dosing of busulfan for children undergoing stem cell transplants
Implementing Pharmacogenetics in the Busulfan Dosing Method for Children Undergoing Hematopoietic Stem-cell Transplantation: a Prospective, Multicentric, Randomized Clinical Trial
This study is testing whether giving children and teens a personalized dose of busulfan based on their genetics can make stem cell transplants work better and cause fewer side effects.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 260 (estimated) |
| Ages | N/A to 18 Years |
| Sex | All |
| Sponsor | University Hospital, Geneva Academic / other |
| Drugs / interventions | fludarabine |
| Locations | 1 site (Geneva) |
| Trial ID | NCT04822532 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to personalize the dosing regimen of busulfan, a medication used in hematopoietic stem cell transplantation (HSCT), for children and adolescents. Participants will be randomly assigned to receive their initial dose based on either a standard method using age and weight or a method that incorporates genetic factors affecting busulfan metabolism. The goal is to improve the efficacy of HSCT while minimizing related toxicities. The study is being conducted internationally across five countries: Canada, Italy, Switzerland, France, and Denmark.
Who should consider this trial
Good fit: Ideal candidates are children and adolescents aged 0-18 years who require allogeneic or autologous HSCT and are eligible for busulfan-based conditioning regimens.
Not a fit: Patients who are scheduled to receive certain medications that interact with busulfan during the conditioning period may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could lead to more effective and safer HSCT treatments for children and adolescents.
How similar studies have performed: Other studies have shown promise in using pharmacogenetic approaches for drug dosing, suggesting potential success for this personalized method.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients must be aged from 0-18 years old on entry to the study; * Clinical indication of allogeneic or autologous hematopoietic stem cell transplantation; * The conditioning protocol must include IV Bu formulations, Busulfex® (Otsuka Pharmaceutical), Busilvex® (Pierre Fabre Pharma) or other European Medicines Agency (EMA) or Food and Drugs Administration (FDA) approved generic formulations regardless of the administration schedule (q6h, q12h, or q24h) * The expected length of time from recruitment to starting the conditioning regimen must be superior to 10 days; * Informed written consent to participate in the study signed by the participant/parent Exclusion Criteria: • At least one of the drugs listed below scheduled to be administered in the Bu administration days up to 24h after the last dose of Bu, whenever a washout is not possible: * Metronidazol (required washout: 7 days) * Nalidixic acid (required washout: 7 days) * Phenytoin (required washout: 21 days) * Itraconazole (required washout: 14 days) * Ketoconazole (required washout: 7 days) * Voriconazole (required washout: 7 days) * Deferasirox (required washout: 7 days)
Where this trial is running
Geneva
- Hôpitaux Universitaires de Genève — Geneva, Switzerland (Recruiting)
Study contacts
- Study coordinator: Marc Ansari, MD Prof
- Email: research@cansearch.ch
- Phone: +41795536100
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.