Fiche traitement
Busulfan
Children's Oncology Group • Cancer, SEP
Suivi actif
Phase 3
Résumé
Busulfan est développé par Children's Oncology Group. Le traitement est actuellement en Phase 3. Il est associé à Cancer, SEP. Son objectif thérapeutique principal est Thérapie cellulaire. Le statut actuel est : suivi actif.
À vérifier
Thérapie cellulaire
Phase 3
Children's Oncology Group
United States, Australia, Canada
Background:
Some blood cancers can be caused by germline variants (changes) in a person s RUNX1 gene. Germline variants are genetic inherited changes a person is born with. Stem cell transplants are used to treat many diseases including blood cancers. Stem cell transplantation for patients with germline RUNX1 mutation driven blood cancers is standard of care and available in most major medical centers. The difference with this transplantation protocol is that it is prospective, only available to participants with germline RUNX1 variants and designed to determine the extent to which tailoring chemotherapy and supportive care medication doses for each individual patient may improve outcomes compared to data derived from retrospective transplantation protocols for patients with RUNX1 varinats which is less accurate.
Objective:
The primary objective of this protocol is to determine how tailored doses of chemotherapy and supportive care medications may improve disease free survival as compared to historical/expected disease free survival.
Eligibility:
People aged 4 to 70 years with blood cancer caused by a RUNX1 gene mutation. Other participants are also needed: (1) stem cell donors; (2) relatives who do not have a mutation in the RUNX1 gene; and (3) healthy volunteers.
Design:
Participants with blood cancer will be screened during approximately 1-3 months before transplatation. They will have blood tests and tests of their heart and lung function. A sample of bone marrow may be taken.
A flexible tube (central line) will be inserted into a vein in participants chest or lower neck. This line will remain in place during the hospitalization and be used to draw blood and administer drugs. These lines are almost always transitioned to a peripherally inserted central catheter (PICC) line at the time of hospital discharge.
Participants will be inpatient for 4 to 5 weeks. They will receive drugs to prepare their body for the stem cell transplant. Some may also receive radiation treatment. Other tests will include imaging scans. The stem cell transplant will be given through the central line.
After discharge from the clinic, participants will have follow-up visits at least once per week for approximately 100 days. Then they will have follow-up clinic visits for 3 years.
Donors, relatives, and healthy volunteers may provide samples of blood, stool, and saliva. Adults may also opt to provide samples of skin and bone marrow.
2026-07-29
Début phase actuelle
Non disponible dans les sources synchronisées
Timeline clinique
Phase 3
Début phase actuelle
Non disponible dans les sources synchronisées
Préclinique
Phase 1
Phase 2
Phase 3
Approuvé
Objectif scientifique de l’étude
classification automatique vérifiable
Thérapie cellulaire
Confiance automatique : 80%
Ce pourcentage n’est pas une probabilité de succès clinique.
Il mesure uniquement la force de la classification automatique de l’objectif scientifique. Le calcul part de 45 points, ajoute 7 points par unité de poids du mot-clé le plus fortement détecté, puis est plafonné à 95 %. Exemple : un score maximal de 2 donne 45 + (2 × 7) = 59 %.
Indices détectés : stem cell → Thérapie cellulaire
Ce score est heuristique et doit être vérifié dans le protocole ou une publication.
Aucune incohérence majeure détectée entre la maladie, l’objectif et la description.
Transparence et qualité de la fiche
données vérifiablesClassificationCohérente automatiquement
Confiance de la fiche100 %
Source principaleSource officielle ou publication indexée
Dernière vérification2026-07-29
Anomalies détectées0
Validation humaineValidation humaine non effectuée
Donnée issue d’une source
Donnée normalisée automatiquement
Interprétation algorithmique
Classification scientifique multi-axes
ne pas confondre statut et résultatÉtat de l’étudeStatut inconnu
Disponibilité des résultatsRésultats à vérifier
InterprétationImpossible à déterminer
Niveau de preuveNon évaluable
Un essai terminé n’est pas nécessairement positif. Un essai arrêté n’est classé comme échec scientifique que lorsqu’un résultat ou une raison explicite le confirme.
Indicateurs factuels
sans score subjectifPhase enregistréePhase 3
Études associées1
Publications associées9
Statut consolidéSuivi actif
Aucune chance de succès, note d’innovation, potentiel thérapeutique ou probabilité de commercialisation n’est calculé tant qu’un modèle validé et des données suffisantes ne sont pas disponibles.
Historique factuel de la molécule
13 événement(s) daté(s)Cette liste reprend uniquement les dates trouvées dans les registres et publications liés. Elle ne constitue pas une prévision.
| Date | Événement | Source |
|---|---|---|
| 2026-07-12 | PublicationPrecision myeloablation with TDM-guided busulfan in pediatric primary immunodeficiencies: a real-world study of 28 patients. | Translational pediatrics |
| 2026-07-14 | PublicationEfficacy and safety of mitoxantrone hydrochloride liposome/ thiotepa/ busulfan/fludarabine (MTBF) conditioning regimen for salvage allogeneic hematopoietic stem cell transplantation in patients with relapsed or refractory acute myeloid leukemia: A single - Arm prospective clinical trial. | Cell transplantation |
| 2026-07-22 | PublicationSuccessful allogeneic hematopoietic stem cell transplantation in children with myeloid malignancies secondary to GATA2 deficiency using decitabine-containing busulfan conditioning. | Bone marrow transplantation |
| 2026-07-29 | Dernière mise à jour des donnéesBusulfan | Biomedical Watch |
| 2026-08-01 | Début de l’étudeNCT07762508 | Registre d’essai clinique |
| 2026-08-13 | Création de l’enregistrementNCT07762508 | Registre d’essai clinique |
| 2026-08-16 | PublicationReinterpreting the matched-sibling relapse penalty in ATG-based allogeneic transplantation for myelodysplastic neoplasms: a question of dose, not donor. | Annals of hematology |
| 2026-08-16 | PublicationRestoration of spermatogenesis is dependent on activation of a SPRY4-ERK checkpoint following germline stem cell damage†. | Biology of reproduction |
| 2026-08-16 | PublicationImpact of conditioning regimen on outcomes of young AML patients ( < 40 years) undergoing HCT in complete remission, with transplant conditioning intensity score of 3.5-4.0. A study from the acute leukemia working party of the European Society for Blood and Marrow Transplantation. | Bone marrow transplantation |
| 2026-08-16 | PublicationAssociation of Busulfan Exposure with Event-Free Survival in a Chinese Pediatric Cohort: A Multicenter, Prospective Cohort Study. | Drug design, development and therapy |
| 2026-08-16 | PublicationA Novel Modified Bu/Vp16/cy/Flu/Ara-C Conditioning Regimen Enhances Outcomes for High-Risk Acute Lymphoblastic Leukemia Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation. | Cancer medicine |
| 2026-09-11 | PublicationFludarabine, Busulfan, Thiotepa, and Melphalan: A Novel Low-Toxicity Myeloablative Conditioning Regimen for Haploidentical HSCT in Children and Adolescents Aged ≥7 Years With Transfusion-Dependent β-Thalassemia. | Pediatric blood & cancer |
| 2029-07-01 | Fin de l’étude prévue / enregistréeNCT07762508 | Registre d’essai clinique |
Début phase actuelle
Non disponible dans les sources synchronisées
Essais cliniques liés
1 essai(s)
Début phase actuelle
Non disponible dans les sources synchronisées
| NCT | Titre | Phase | Statut administratif | Pays | Sponsor |
|---|---|---|---|---|---|
| NCT07762508 | A Prospective Single-Arm Study of VABu Conditioning Regimen in Allo-HSCT for Low-Performance or High-Comorbidity AML Patients in CR1 | Non applicable | NOT_YET_RECRUITING | China | The First Affiliated Hospital of Soochow University |
Publications liées
9 publication(s)Publications liées
9 publication(s)| Titre | PMID | DOI | Journal | Date |
|---|---|---|---|---|
| Fludarabine, Busulfan, Thiotepa, and Melphalan: A Novel Low-Toxicity Myeloablative Conditioning Regimen for Haploidentical HSCT in Children and Adolescents Aged ≥7 Years With Transfusion-Dependent β-Thalassemia. Voir source | 42489576 | 10.1002/1545-5017.70563 | Pediatric blood & cancer | |
| Reinterpreting the matched-sibling relapse penalty in ATG-based allogeneic transplantation for myelodysplastic neoplasms: a question of dose, not donor. Voir source | 42525122 | 10.1007/s00277-026-07209-4 | Annals of hematology | |
| Restoration of spermatogenesis is dependent on activation of a SPRY4-ERK checkpoint following germline stem cell damage†. Voir source | 41852347 | 10.1093/biolre/ioag063 | Biology of reproduction | |
| Impact of conditioning regimen on outcomes of young AML patients ( < 40 years) undergoing HCT in complete remission, with transplant conditioning intensity score of 3.5-4.0. A study from the acute leukemia working party of the European Society for Blood and Marrow Transplantation. Voir source | 42469396 | 10.1038/s41409-026-02991-1 | Bone marrow transplantation | |
| Association of Busulfan Exposure with Event-Free Survival in a Chinese Pediatric Cohort: A Multicenter, Prospective Cohort Study. Voir source | 42504202 | 10.2147/DDDT.S593835 | Drug design, development and therapy | |
| A Novel Modified Bu/Vp16/cy/Flu/Ara-C Conditioning Regimen Enhances Outcomes for High-Risk Acute Lymphoblastic Leukemia Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation. Voir source | 41761760 | 10.1002/cam4.71669 | Cancer medicine | |
| Successful allogeneic hematopoietic stem cell transplantation in children with myeloid malignancies secondary to GATA2 deficiency using decitabine-containing busulfan conditioning. Voir source | 42471475 | 10.1038/s41409-026-02978-y | Bone marrow transplantation | |
| Efficacy and safety of mitoxantrone hydrochloride liposome/ thiotepa/ busulfan/fludarabine (MTBF) conditioning regimen for salvage allogeneic hematopoietic stem cell transplantation in patients with relapsed or refractory acute myeloid leukemia: A single - Arm prospective clinical trial. Voir source | 42446104 | 10.1177/09636897261469013 | Cell transplantation | |
| Precision myeloablation with TDM-guided busulfan in pediatric primary immunodeficiencies: a real-world study of 28 patients. Voir source | 42433921 | 10.21037/tp-2026-0344 | Translational pediatrics |
Sources officielles
liens de rechercheDernière mise à jour des données : 2026-07-29
Ces sources sont proposées pour vérification. Les informations de la fiche doivent être confirmées dans les registres officiels ou les publications originales.