Tim Hassall
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View article: CTP-02. A Phase 1 trial of Deflexifol™ in pediatric patients with relapsed or refractory brain tumors
CTP-02. A Phase 1 trial of Deflexifol™ in pediatric patients with relapsed or refractory brain tumors Open
BACKGROUND Deflexifol is a novel combination of 5-fluorouracil (5-FU) and leucovorin, allowing for co-delivery in one infusion. 5-FU has documented activity in pediatric ependymoma. It is hypothesized that this novel formulation will enhan…
View article: PTHP-15. Enhancing Medulloblastoma Classification: Integrating IHC and DNA Methylation Data from the SJMB12 Clinical Trial
PTHP-15. Enhancing Medulloblastoma Classification: Integrating IHC and DNA Methylation Data from the SJMB12 Clinical Trial Open
The SJMB12 trial used immunohistochemistry (IHC)-based molecular grouping to separate medulloblastoma (MB) into WNT, SHH, and non-WNT/non-SHH (NWNS) groups for risk and treatment stratification. IHC was selected due to its rapid turnaround…
View article: Disparities in Quality of Life and Family Functioning 24 Months After Diagnosis of a Childhood Brain Tumour: A Longitudinal Cohort Study
Disparities in Quality of Life and Family Functioning 24 Months After Diagnosis of a Childhood Brain Tumour: A Longitudinal Cohort Study Open
Background Increases in survival for childhood brain tumour are evidence of improved care; however, survival is not the only outcome that matters. There has been little scholarly investigation into the psychosocial wellbeing of the diagnos…
View article: Suppressing recurrence in Sonic Hedgehog subgroup medulloblastoma using the OLIG2 inhibitor CT-179
Suppressing recurrence in Sonic Hedgehog subgroup medulloblastoma using the OLIG2 inhibitor CT-179 Open
OLIG2-expressing tumor stem cells have been shown to drive recurrence in Sonic Hedgehog (SHH)-subgroup medulloblastoma (MB) and patients urgently need specific therapies to target this tumor cell population. Here, we investigate the therap…
View article: Radiographic and visual response to the type II RAF inhibitor tovorafenib in children with relapsed/refractory optic pathway glioma in the FIREFLY-1 trial
Radiographic and visual response to the type II RAF inhibitor tovorafenib in children with relapsed/refractory optic pathway glioma in the FIREFLY-1 trial Open
Background Due to their anatomical locations, optic pathway gliomas (OPGs) can rarely be cured by resection. Given the importance of preserving visual function, we analyzed radiological and visual acuity (VA) outcomes for the type II RAF i…
View article: CTNI-09. TYPE II RAF INHIBITOR TOVORAFENIB IN RELAPSED/REFRACTORY (R/R) PEDIATRIC LOW-GRADE GLIOMA (PLGG): RESULTS FROM PATIENTS ON A DRUG HOLIDAY (DH) IN THE PHASE 2 FIREFLY-1 TRIAL
CTNI-09. TYPE II RAF INHIBITOR TOVORAFENIB IN RELAPSED/REFRACTORY (R/R) PEDIATRIC LOW-GRADE GLIOMA (PLGG): RESULTS FROM PATIENTS ON A DRUG HOLIDAY (DH) IN THE PHASE 2 FIREFLY-1 TRIAL Open
BACKGROUND Tovorafenib, a selective, CNS-penetrant, type II RAF inhibitor, received US FDA accelerated approval for r/r BRAF-altered pLGG. Results from the ongoing FIREFLY-1 (NCT04775485) phase 2 study (Kilburn LK, et al. Nat Med. 2023) in…
View article: NCOG-05. HEALTH-RELATED QUALITY OF LIFE (HRQOL) IN THE PHASE 2 FIREFLY-1 (PNOC026) TRIAL OF THE TYPE II RAF INHIBITOR TOVORAFENIB IN RELAPSED/REFRACTORY (R/R) PEDIATRIC LOW-GRADE GLIOMA (PLGG)
NCOG-05. HEALTH-RELATED QUALITY OF LIFE (HRQOL) IN THE PHASE 2 FIREFLY-1 (PNOC026) TRIAL OF THE TYPE II RAF INHIBITOR TOVORAFENIB IN RELAPSED/REFRACTORY (R/R) PEDIATRIC LOW-GRADE GLIOMA (PLGG) Open
BACKGROUND Despite favorable long-term survival, ongoing disease and treatment-related morbidity substantially impact the HRQOL of patients with pLGG. Tovorafenib is a selective, CNS-penetrant, type II RAF inhibitor. Results from the ongoi…
View article: Suppl Table S2 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency
Suppl Table S2 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency Open
Results of time-dependent co-variate and landmark analyses at time-points 3 and 6 months of the trial
View article: Suppl Figure S1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency
Suppl Figure S1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency Open
Pathological complete remission in metastatic colorectal adenocarcinoma. (A) Baseline (hematoxylin-eosin, CD8 and PD-L1). (B) Post-nivolumab: no detectable tumor cells, increased inflammation, and increase in CD8 and PD-L1 expression. All …
View article: Trial protocol 1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency
Trial protocol 1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency Open
Full trial protocol NCT02992964
View article: Data from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency
Data from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency Open
Purpose: Checkpoint-inhibitors have limited efficacy for children with unselected solid and brain tumors. We report the first prospective pediatric trial (NCT02992964) using nivolumab exclusively for refractory non-hematological cancers ha…
View article: Suppl Figure S1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency
Suppl Figure S1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency Open
Pathological complete remission in metastatic colorectal adenocarcinoma. (A) Baseline (hematoxylin-eosin, CD8 and PD-L1). (B) Post-nivolumab: no detectable tumor cells, increased inflammation, and increase in CD8 and PD-L1 expression. All …
View article: Suppl Table S2 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency
Suppl Table S2 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency Open
Results of time-dependent co-variate and landmark analyses at time-points 3 and 6 months of the trial
View article: Supplementary Table S1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency
Supplementary Table S1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency Open
Representativeness of Study Participants
View article: Supplementary Table S1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency
Supplementary Table S1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency Open
Representativeness of Study Participants
View article: Trial protocol 1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency
Trial protocol 1 from Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency Open
Full trial protocol NCT02992964
View article: Quality of life and family functioning 12 months after diagnosis of childhood brain tumour: A longitudinal cohort study
Quality of life and family functioning 12 months after diagnosis of childhood brain tumour: A longitudinal cohort study Open
Background The wellbeing of a child with brain tumour is affected by several factors. We present the first investigation of quality of life and family functioning in a parent and child across the first 12 months after diagnosis, examining …
View article: MDB-65. RESULTS FROM THE SJ-ELIOT PHASE 1 CLINICAL TRIAL EVALUATING PREXASERTIB (LY2606368) IN COMBINATION WITH CYCLOPHOSPHAMIDE OR GEMCITABINE FOR CHILDREN AND ADOLESCENTS WITH REFRACTORY OR RECURRENT MEDULLOBLASTOMA
MDB-65. RESULTS FROM THE SJ-ELIOT PHASE 1 CLINICAL TRIAL EVALUATING PREXASERTIB (LY2606368) IN COMBINATION WITH CYCLOPHOSPHAMIDE OR GEMCITABINE FOR CHILDREN AND ADOLESCENTS WITH REFRACTORY OR RECURRENT MEDULLOBLASTOMA Open
BACKGROUND SJ-ELIOT (NCT04023669) was a phase 1 trial that explored the combination of the checkpoint kinase inhibitor, prexasertib with the DNA-damaging agents, cyclophosphamide and gemcitabine, in recurrent or refractory medulloblastoma.…
View article: DIPG-67. RE-IRRADIATION PRACTICES AND OUTCOMES IN PATIENTS WITH DIPG/DMG: A REPORT FROM THE INTERNATIONAL DIPG REGISTRY
DIPG-67. RE-IRRADIATION PRACTICES AND OUTCOMES IN PATIENTS WITH DIPG/DMG: A REPORT FROM THE INTERNATIONAL DIPG REGISTRY Open
BACKGROUND Given that radiation therapy is the only treatment modality demonstrated to result in any clinical benefit for children with DIPG, re-irradiation therapy has been explored as a treatment option for progressive DIPG. Several stud…
View article: LGG-40. TYPE II RAF INHIBITOR TOVORAFENIB IN RELAPSED/REFRACTORY PEDIATRIC LOW-GRADE GLIOMA (PLGG): REVERSIBLE DECREASES IN GROWTH VELOCITY IN THE PHASE 2 FIREFLY-1 TRIAL
LGG-40. TYPE II RAF INHIBITOR TOVORAFENIB IN RELAPSED/REFRACTORY PEDIATRIC LOW-GRADE GLIOMA (PLGG): REVERSIBLE DECREASES IN GROWTH VELOCITY IN THE PHASE 2 FIREFLY-1 TRIAL Open
BACKGROUND: Tovorafenib is an investigational, selective, CNS-penetrant, type II RAF inhibitor. The ongoing FIREFLY-1 (NCT04775485) phase 2 study (Kilburn LK, et al. Nat Med. 2023) of tovorafenib in BRAF-altered pLGG resulted in antitumor …
View article: DIPG-84. PHASE 1 DOSE-ESCALATION STUDY OF ACT001 IN PEDIATRIC PATIENTS WITH ADVANCED BRAIN AND SOLID TUMORS SHOWS ACTIVITY IN DIFFUSE MIDLINE GLIOMA (DMG)
DIPG-84. PHASE 1 DOSE-ESCALATION STUDY OF ACT001 IN PEDIATRIC PATIENTS WITH ADVANCED BRAIN AND SOLID TUMORS SHOWS ACTIVITY IN DIFFUSE MIDLINE GLIOMA (DMG) Open
BACKGROUND ACT001, an oral parthenolide derivative targeting STAT3 and NF-κB pathways, has shown activity in preclinical models of DMG. We evaluated safety, toxicity and activity of ACT001 in this recently completed, first-in-child study o…
View article: MDB-92. EFFECT OF REDUCED-DOSE CRANIOSPINAL IRRADIATION AND REDUCED-DOSE ADJUVANT CHEMOTHERAPY ON CHILDREN AND ADOLESCENTS WITH WNT MEDULLOBLASTOMA WITHOUT RESIDUAL OR METASTATIC DISEASE: RESULTS FROM THE SJMB12 CLINICAL TRIAL
MDB-92. EFFECT OF REDUCED-DOSE CRANIOSPINAL IRRADIATION AND REDUCED-DOSE ADJUVANT CHEMOTHERAPY ON CHILDREN AND ADOLESCENTS WITH WNT MEDULLOBLASTOMA WITHOUT RESIDUAL OR METASTATIC DISEASE: RESULTS FROM THE SJMB12 CLINICAL TRIAL Open
BACKGROUND In response to studies showing medulloblastoma (MB) survival is not only contingent on clinical factors (e.g. metastases, residual disease) but also on molecular factors (e.g. molecular group, gene aberrations), we launched the …
View article: Spatial Transcriptomic Sequencing of a DIPG-infiltrated Brainstem reveals Key Invasion Markers and Novel Ligand-Receptor Pairs contributing to Tumour to TME Crosstalk
Spatial Transcriptomic Sequencing of a DIPG-infiltrated Brainstem reveals Key Invasion Markers and Novel Ligand-Receptor Pairs contributing to Tumour to TME Crosstalk Open
Emerging spatially-resolved sequencing technologies offer unprecedented possibilities to study cellular functionality and organisation, transforming our understanding of health and disease. The necessity to understand healthy and diseased …
View article: Management of patients with diffuse intrinsic pontine glioma in Australia and New Zealand: Australian and New Zealand Children's Haematology/Oncology Group position statement
Management of patients with diffuse intrinsic pontine glioma in Australia and New Zealand: Australian and New Zealand Children's Haematology/Oncology Group position statement Open
Introduction The main mission of the Australian and New Zealand Children's Haematology and Oncology Group (ANZCHOG) is to develop and facilitate local access to the world's leading evidence‐based clinical trials for all paediatric cancers,…
View article: Goal-directed therapeutic exercise for paediatric posterior fossa brain tumour survivors: a qualitative analysis of experiences
Goal-directed therapeutic exercise for paediatric posterior fossa brain tumour survivors: a qualitative analysis of experiences Open
Purpose To explore child and parent experiences of a 12-week goal-directed therapeutic exercise intervention in paediatric posterior fossa brain tumours survivors and to identify features of the program that influenced program adherence an…
View article: Trial protocol 1 from Efficacy of Nivolumab in Pediatric Cancers with High Mutation Burden and Mismatch Repair Deficiency
Trial protocol 1 from Efficacy of Nivolumab in Pediatric Cancers with High Mutation Burden and Mismatch Repair Deficiency Open
Full trial protocol NCT02992964