To analyze the number of patients with (germline/somatic) actionable molecular aberrations in patients with relapsed/refractory pediatric tumors for whom no standard treatment or study protocol is available.
ID
Source
Brief title
Condition
- Miscellaneous and site unspecified neoplasms malignant and unspecified
Synonym
Health condition
relapse refractory, pediatric oncology, molecular profiling, actionable lesions recidief refractair, kinderoncologie, moleculaire profilering, behandelbare laesies
Research involving
Sponsors and support
Intervention
- Other intervention
Outcome measures
Primary outcome
To define the number of patients with relapsed/refractory pediatric tumors for whom no standard protocol treatment is available, in whom actionable lesions are identified
Secondary outcome
• To determine the number of patients with relapsed/refractory pediatric tumors in the Netherlands each year for whom no standard protocol or treatment is available (per protocol population)
• The percentage of patients who are able to undergo a diagnostic tumor biopsy (as standard of care)
• The percentage of tumor biopsies associated with procedure-related complications
• The percentage of cases in which tumor material was obtained of sufficient quality for molecular profiling
• The percentage of cases in which one or more actionable lesions are identified
• The percentage of cases in which a mutation in a cancer predisposition gene is identified
• The percentage of cases in which the molecular tumor board is able to provide a treatment advice to the treating physician
• The time frame between the date of the biopsy and the date of the treatment advice by the molecular tumor board
• The percentage of patients treated according to the treatment recommendation
• Major side-effects and disease responses observed in the patients treated
• Survival parameters (overall and progression-free survival) in the entire cohort of patients with a follow-up duration of 1 year following enrolment.
Background summary
Significant progress has been made in the cure of pediatric cancer through treatment optimization (chemotherapy, radiotherapy and surgery) and improvement of supportive care. Despite major advances, 25% of children with cancer ultimately die due to lack of effective treatment. New treatment modalities are urgently needed. The most promising option is the development of targeted therapy in which a genetic aberration in the tumor is targeted by small molecules. This however requires that the tumor biology is deciphered to identify tumor-driving genetic aberrations. Another strategy is to perform compound screening on organoids grown from tumor tissue.
Study objective
To analyze the number of patients with (germline/somatic) actionable molecular aberrations in patients with relapsed/refractory pediatric tumors for whom no standard treatment or study protocol is available.
Study design
This is a non-randomized single-arm observational study aimed at molecular profiling of tumor biopsy samples and germline tissue, taken during a standard of care biopsy procedure to confirm relapsed or refractory malignancy, from patients with relapsed/refractory pediatric tumors.
Intervention
Molecular profiling
Study burden and risks
The main aim of this study is to identify actionable lesions in cancers arising in children to develop personalized medicine. Therefore minors have to be included in the study. There are no risks associated with participation in this study, as the biopsy will be performed as standard of care.
Erasmus MC-Sophia Children's Hospital<br>
POB 2060
C.M. Zwaan
Rotterdam 3000 CB
The Netherlands
+31 (0)10 7036691/6130
c.m.zwaan@erasmusmc.nl
Erasmus MC-Sophia Children's Hospital<br>
POB 2060
C.M. Zwaan
Rotterdam 3000 CB
The Netherlands
+31 (0)10 7036691/6130
c.m.zwaan@erasmusmc.nl
Age
Inclusion criteria
-relapsed/refractory pediatric cancer, which was established at initial diagnosis by standard diagnostic methods
-no available standard treatment or study protocol
-life-expectancy of at least 10 weeks
-written informed consent according to local law and legislation
-age <30 years
Exclusion criteria
-biopsy considered unsafe
-severe organ toxicity precluding undergoing any of the procedures mentioned in this protocol
-any other condition that may hamper participation according to the treating physician
Design
Recruitment
IPD sharing statement
Followed up by the following (possibly more current) registration
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL5728 |
| NTR-old | NTR5915 |
| CCMO | NL56826.078.16 |
| OMON | NL-OMON55694 |
Summary results
Nowadays, all children with newly diagnosed, relapsed or refractory tumours are offered WES and RNA-seq in our center as standard of care, complemented by DNAmethylation for CNS tumours and sarcomas, to facilitate precision diagnostics and improve cure rates.
Standardisation of data analysis and target prioritisation as well as improved access to targeted treatments within combination trials are required to translate findings from precision medicine programs into clinical care and eventually improve survival."