Ultrafast Whole Genome Sequencing for Childhood Cancer

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age0-24
SponsorUniversity of Cambridge

About this trial

Cambridge University Hospitals NHS Foundation Trust (CUHNFT) is the Principal Treatment Centre for the East of England region, responsible for 120-150 patients \<16 years with a new diagnosis of paediatric malignancy annually; leukaemia comprises \~25% of these cases. Current molecular diagnosis of subgroups of childhood malignancies, particularly leukaemia, is based on flow cytometry, fluorescent in situ hybridisation (FISH) and single nucleotide polymorphim (SNP) arrays, for which the usual turnaround time (TAT) is 7-14 days. In the current era of access to targeted therapy, rapid diagnosis and treatment of patients in high-risk molecular subgroups is critical for improving outcomes. Children and adolescents with Philadelphia-chromosome positive (Ph+) acute lymphoblastic leukaemia (ALL) have significantly improved survival when treated with tyrosine kinase inhibitors (TKIs). Patients with Ph+-like mutations (10- 20% of paediatric ALL), also have a poor prognosis, requiring escalation of treatment and addition of targeted therapy. Rapidly identifying MYCN amplification is also of critical prognostic importance in embryonal tumours of childhood including neuroblastoma (25%) and medulloblastoma, and directly impacts on treatment from the outset of the patient journey. Overnight whole genome sequencing (WGS) entails taking an additional 5ml Peripheral Blood (PB) and Bone Marrow (BM) samples after samples for routine diagnostic workup have been collected, and could replace current standard of care (SOC), which has a median turnaround time (TAT) of up to 28 days, and up to 84 days for specific gene mutations, which can delay appropriate prognostication and management of high-risk patients. Rapid, point of care information on somatic and germline mutations will allow early risk stratification and expedite treatment for high-risk patients with cancer.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Have given written informed consent to participate

Be aged <25 years of age

Have confirmed or suspected malignancy

For pilot/feasibility study (first 10 patients), only haematological malignancies (ALL/AML) will be included

Disqualifiers

Inability to provide written informed consent (self or parent/guardian)

Insufficient tissue (BM/PB/tissue) available for research purposes after collection for routine diagnostic purposes

Trial population

Children with cancer at relapse or initial diagnosis

Trial design

Design model

Case-only

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

50 Participants
are grouped into 2 trial groups
Group A: Ultrafast WGS cohort, prospective patients
Group B: Ultrafast WGS, retrospective patients

Trial outcomes

Primary outcomes

1

Turnaround time from sample collection to availability of meaningful results.

Time frame
36 months

Secondary outcomes

1

Percentage of enrolled patients with available WGS results from the Ultrafast WGS pipeline.

Time frame
24 months
2

Correlation of data from Ultrafast WGS against current SOC WGS data.

Time frame
36 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of Cambridge

Lead sponsor

Illumina, Inc.

Collaborator

Cambridge University Hospitals NHS Foundation Trust

Collaborator