Clinical trial NCT05556642: Relapsed and Refractory Hepatoblastoma: A Prospective Registry and Liver Tumor Research Consortium Initiative
Official title: Relapsed and Refractory Hepatoblastoma: A Prospective Registry and Hepatic Tumors Research Consortium Initiative
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Relapsed Hepatoblastoma
Refractory Hepatoblastoma
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
First posted: 2022-09-27
Enrollment: 500
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with relapsed or refractory Hepatoblastoma

Brief summary: There are limited data regarding the biology and treatment of relapsed/refractory hepatoblastoma (rrHBL). This project provides the infrastructure for acquisition of biological specimens, imaging, and correlative clinical data to facilitate biology studies and characterization of rrHBL. This registry will collect clinical, demographic, and pathological data, specimens (as available) and imaging from patients with rrHBL, prospectively. Cases are identified through:

1. Existing clinical and/or cancer registry databases
2. Referrals from clinicians, surgeons, or pathologists
3. Families initiating contact with Registry staff directly

Detailed description: The following data/materials will be collected:

Clinical Data: Demographic data, date of diagnosis, pathology, radiological imaging data, signs and symptoms at diagnosis, molecular and biological data, staging details including sites of disease, detailed treatment data (e.g. types and dates of surgeries/interventional therapy (if any), medical/systemic therapy, radiotherapy), response to treatment (imaging and tumor markers), dates of progression, types of progression (local or metastatic), and follow-up data. The demographic and clinical data collected are abstracted and entered into an electronic data system secured by password protection. Collection of existing molecular and/or genomic data or analysis that has been performed will also be included.

Research Specimens:

Tissue (fresh/frozen/FFPE) from diagnosis and all subsequent timepoints where tissue was acquired (for example at each relapse and/or progression) will be requested for all enrolled patients and sent for central review to the study pathologist and stored in the rrHBL biorepository within the Cancer and Blood Disease Institute at Cincinnati Children's Hospital Medical Center. Future research testing may be conducted on this tissue.

Optional research specimens:

Pending consent specification of family/patient, and resources, specimens may be sent directly to collaborating laboratories to proceed with tumor modeling and drug testing, or other translational research with PI or designee via an Institutional Review Board approved project.

* Tumor Modeling, Drug Testing, or Other Applicable Cancer-Related Research (Fresh, frozen,): Prospectively collected or retained left-over tumor tissue samples and normal tissue (background liver) as available, for research purposes.
* Blood: prospectively collected at time of enrollment and at time of subsequent relapse
* Urine: prospectively collected at time of enrollment and at time of subsequent relapse
* Saliva: prospectively collected at time of enrollment or at one time point after enrollment

Registry clinical data will be correlated with biological/bioinformatic and genomic data. Data stored in the Registry may be used to provide statistical data for scientific presentations and for preparation of peer- reviewed manuscripts.

Eligibility criteria: Inclusion Criteria:

* All patients of any age with a suspected diagnosis (per treating oncologist/surgeon) or confirmed diagnosis of a rrHBL and all patients with Hepatocellular Malignant Neoplasm- Not Otherwise Specified (HCN-NOS) who are \<6 years of age at the time of initial diagnosis
* To allow for tumor modelling to be performed with fresh tissue from these cases, patients with suspected rrHBL are eligible to enroll on study
* Unless the patient is deceased, all patients and/or one parent or legal guardian must provide written informed consent as well as HIPAA/release of information consent

Exclusion Criteria:

* None

Primary outcomes:
measure: Characterization of relapsed or refractory Hepatoblastoma
timeFrame: From enrollment until data analysis is complete - assessed up to 40 years
measure: Create biorepository of specimens
timeFrame: From enrollment until data analysis is complete - retained up to 40 years
measure: Clinical data and biological data correlation
timeFrame: From enrollment until data analysis is complete - assessed up to 40 years
Secondary outcomes:
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Katherine Somers, MD
role: STUDY_CHAIR
References: