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RecruitingNCT05722886DETERMINEUpdated Jul 1, 2026

DETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial) - Master Screening Protocol

A Phase 2/3 interventional study of Alectinib and Atezolizumab in Haematological Malignancy and Solid Tumour, sponsored by Cancer Research UK. Recruiting at 27 sites in United Kingdom. Per ClinicalTrials.gov, last updated 2026-07-01.

Sponsored by Cancer Research UK · Phase 2/3, Interventional, and Treatment

Phase
Phase 2/3
Study type
Interventional
Enrollment
825
Allocation
Non-randomized
Sex
All
01

Study summary

DETERMINE is an open-label phase II/III trial. It will look at targeted treatments in rare cancers or common cancers with rare genetic change (mutation). Patients must have a cancer with an identified mutation. This could be found during routine testing or as part of another research programme. The DETERMINE trial will recruit adults, teenagers and children. If a drug is found to benefit a new patient group, the study team will work with the NHS and the Cancer Drugs Funds to see if these drugs can be available for patients in the future. This clinicaltrials.gov record refers to the Overall Trial Protocol (Master Screening Record), additional records will be added to clinicaltrials.gov for each treatment arm.

Read the detailed description

DETERMINE is an umbrella-basket platform trial to evaluate the efficacy of licensed targeted therapies in rare* adult, paediatric and teenage/young adult (TYA) cancers with actionable genomic alterations, including common cancers with rare actionable alterations.

*Rare is defined generally as incidence less than 6 cases in 100,000 patients (includes paediatric and TYA cancers) or common cancers with rare alterations.

The number of treatment arms opened will depend on the number of licensed medicines identified for inclusion. Each trial cohort has a target sample size of 30 evaluable patients. Sub-cohorts may be defined and further expanded to a target of 30 evaluable patients each.

This clinicaltrials.gov record refers to the Overall Trial Protocol (Master Screening Record), please refer to the references section for links to the individual treatment arm records.

The main aims of the clinical trial arms are:

  • To describe the anti-cancer activity of licensed targeted drugs outside their licensed indication.
  • To assess the safety and adverse event (AE) profile of licensed, targeted anti-cancer drugs in the target population.
  • To understand biological mechanisms for response and resistance to targeted therapies.
  • To evaluate the quality of life (QoL) of target populations receiving the licensed, targeted anti-cancer drugs.

This Master Screening Record will capture the number of patients with a cancer containing the appropriate genetic alteration that have been successfully allocated and consented to each arm. The trial results (according to the protocol defined outcome measures) will be reported per-arm for each treatment arm.

The ultimate aim is to translate positive clinical findings to the NHS to provide new treatment options for rare adult, paediatric and TYA cancers.

02

Conditions studied

  • Haematological Malignancy
  • Solid Tumour

Keywords

  • Adult
  • Alectinib
  • ALK Tyrosine Kinase Receptor
  • Antineoplastic Agents
  • Atezolizumab
  • BRAF Kinase
  • Cancer
  • Child
  • CMMRD
  • Cobimetinib
  • Entrectinib
  • Genes, HER2
  • Immune Checkpoint Inhibitors
  • Immunological
  • Lymphoproliferative Disorders
  • Malignancy
  • Malignant Neoplasms
  • MSI
  • Molecular Targeted Therapy
  • Mutation
  • Neoplasms by Histologic Type
  • Neoplasms by Site
  • Paediatric
  • Pertuzumab
  • Precision Medicine
  • Protein Kinase Inhibitors
  • Rare
  • ROS1 protein, human
  • TMB
  • Trastuzumab
  • Tumour-agnostic
  • Vemurafenib
  • Young adult
  • Capmatinib
  • MET
  • Dabrafenib
  • Trametinib
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

THE PATIENT MUST FULFIL THE ELIGIBILITY CRITERIA OUTLINED BELOW AND WITHIN THE SPECIFIC TREATMENT ARM APPENDIX TO WHICH THEY ARE ENROLLED.

Core Inclusion Criteria:

  1. Any patient (adult patients or children and TYA as defined in each treatment arm appendix) with histologically proven locally advanced or metastatic cancer (solid tumour or haematological malignancy) who has:

    1. exhausted (or declined) standard-of-care treatment options.
    2. or for whom no effective standard treatment is available.
    3. and whose disease has progressed or is refractory. Exceptional circumstances may apply as described in the protocol.
  2. Diagnosis of a rare cancer harbouring an actionable genomic alteration, or common cancer types with rare actionable genomic alterations, that has been identified using a validated next-generation sequencing method and for which there is a relevant open treatment arm within the DETERMINE trial.
  3. Life expectancy of at least three months.
  4. Patients are able to provide written (signed and dated) informed consent and be capable of co-operating with treatment and follow-up. For patients under 16 years old, the parent or legal guardian will be asked to provide written informed consent and the patient will be asked to provide age-appropriate assent (written or verbal, commensurate with age and level of understanding).
  5. Patients with objectively evaluable or measurable disease, according to an assessment method appropriate for their cancer type.
  6. Patients must provide a fresh tissue biopsy at baseline and blood samples for translational research. Note that for patients with haematological malignancies or neuroblastomas, blood, bone marrow and/or trephine, lymph node or lump biopsy samples may be taken. For patients with haematological malignancies, a skin punch biopsy may also be taken.
  7. Eastern Cooperative Oncology Group (ECOG) performance status 0-1 (ECOG performance status 2 may be considered on an individual basis) (adults), Karnofsky score ≥50% (TYA) or Lansky Play scales ≥50% (\<12 years). Please see specific treatment arm appendices for any variations on this criterion and for definitions of adult and paediatric populations. Note: Paediatric patients: patients with Central Nervous System (CNS) tumours and a stable neurological deficit may be eligible with a performance status below 50%, at the discretion of the Investigator. In such cases, the deficit must be stable for at least 7 days prior to trial enrolment and be assessed by the local investigator as due to tumour or due to a post-surgical AE.
  8. Women of childbearing potential are eligible provided that they meet the following criteria:

    • Have a negative serum or urine pregnancy test before enrolment and
    • Agree to the birth control methods and duration of use of those methods, as specified in each treatment arm appendix.
  9. Male patients with partners of childbearing potential are eligible provided that they agree to the birth control methods and duration of use of those methods, as specified in each treatment arm appendix.

Core exclusion criteria:

  1. Ongoing AEs Common Terminology Criteria of Adverse Events (CTCAE) Grade ≥2 attributable to previous anti-cancer treatments. Exceptions to this are any clinically stable AEs, which in the opinion of the Investigator should not exclude the patient.
  2. At high medical risk, in the opinion of the Investigator, because of non-malignant systemic disease (including active uncontrolled infection).
  3. Female patients who are pregnant, breastfeeding or planning to become pregnant or male patients with a partner who is a woman of childbearing potential and is planning to become pregnant during the trial or following the last dose of IMP, as specified in each treatment arm appendix.
  4. Is (or plans to be) a patient in another interventional clinical trial, whilst taking part in this trial. Participation in an observational trial which does not involve administration of an Investigational Medicinal Product (IMP) and which, in the opinion of the local Investigator, would not place an unacceptable burden on the patient would be acceptable e.g. sample collection* or QoL studies.

    *for paediatric patients participating in other studies involving tissue/circulating tumour (ct) DNA/other blood collection, consideration would need to be given to the total blood volumes collected (as per the European Medicines Agency blood volume limits for children).

  5. Co-administration of anti-cancer therapies other than those administered in this trial (with the exception of lifelong hormone suppression such as luteinising hormone agonists/analogues in prostate cancer).
  6. Radiotherapy (except for palliative reasons) or chemotherapy, endocrine therapy (except when given for conditions other than malignant disease; e.g. thyroid replacement for hypothyroidism, hydrocortisone for cortisol deficiency/panhypopituitarism), nitrosoureas, mitomycin-C, immunotherapy and molecularly targeted agents or other IMPs within 4 weeks or 5 half-lives (whichever is the shorter).
  7. Rapidly progressing or symptomatically deteriorating brain metastases. Patients with previously treated brain metastases are eligible, provided the patient has not experienced a seizure or had a clinically significant change in neurological status within the 14 days (for adult patients) or 7 days (for paediatric patients) prior to the start of IMP administration. Such patients must be non-dependent on steroids or on a stable or reducing dose of steroid treatment for at least 14 days (or 7 days for paediatric patients) prior to the start of IMP administration. Primary brain or CNS malignancies are allowed providing the patient is clinically stable (if requiring corticosteroids must be at stable or decreasing doses for at least 14 days for adults and 7 days for paediatric patients prior to the start of IMP administration). Patients who have received brain irradiation must have completed whole-brain radiotherapy and/or stereotactic radiosurgery at least 14 days prior to the start of IMP administration.
  8. Any other condition which, in the opinion of the local Investigator, would not be in the best interests of the patient.
04

Study design

Phase
Phase 2 / Phase 3
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Single group
Masking
None (open label)
Enrollment
825 participants (estimated)

Study arms

  • Experimental
    Treatment Arm 1: Alectinib

    This alectinib treatment arm is for adult, paediatric and TYA patients with ALK-positive cancers.

    Drug: Alectinib

  • Experimental
    Treatment Arm 2: Atezolizumab

    This atezolizumab treatment arm is for adult, paediatric and TYA patients with cancers with high tumour mutational burden (TMB) or microsatellite instability high (MSI-high) or proven (previously diagnosed) constitutional mismatch repair deficiency (CMMRD).

    Drug: Atezolizumab

  • Experimental
    Treatment Arm 3: Entrectinib

    This entrectinib treatment arm is for adult, paediatric and TYA patients with ROS1 gene fusion-positive cancers.

    Drug: Entrectinib

  • Experimental
    Treatment Arm 4: Trastuzumab in combination with pertuzumab

    This trastuzumab and pertuzumab treatment arm is for adult, paediatric and TYA patients with cancers with HER2 amplification or activating mutations.

    Drug: Trastuzumab in combination with pertuzumab

  • Experimental
    Treatment Arm 5: Vemurafenib in combination with cobimetinib

    This vemurafenib and cobimetinib treatment arm is for BRAF V600 mutation-positive cancers occurring in adults only.

    Drug: Vemurafenib in combination with cobimetinib

  • Experimental
    Treatment Arm 6: Capmatinib

    This capmatinib treatment arm is for adult patients with cancers harbouring MET dysregulations.

    Drug: Capmatinib

  • Experimental
    Treatment Arm 7: Dabrafenib in combination with trametinib

    This dabrafenib and trametinib treatment arm is for adult, paediatric and TYA patients with cancers harbouring BRAF V600 mutations.

    Drug: Dabrafenib in combination with trametinib

Interventions

  • DrugAlectinib

    Adult patients will be administered alectinib orally at a dose of 600 mg (four 150 mg capsules) twice daily. Paediatric patients with a body weight ≥40 kg and who are able to swallow the capsules will be administered alectinib orally at a dose of 600 mg (four 150 mg capsules) twice daily. Each cycle of treatment will consist of 28 days and patients may continue on treatment until disease progression without clinical benefit, unacceptable AEs or withdrawal of consent.

    Also known as: Alecensa

  • DrugAtezolizumab

    Adult patients will receive 1200 mg of atezolizumab intravenously every 21 days. Paediatric patients will receive atezolizumab at a dose of 15 mg/kg (maximum 1200 mg) every 21 days. Patients may continue on treatment until disease progression without clinical benefit, unacceptable AEs or withdrawal of consent.

    Also known as: Tecentriq

  • DrugEntrectinib

    Adult and paediatric patients with body surface area (BSA) ≥1.51 m\^2 will receive entrectinib orally at a dose of 600 mg daily dose (three 200 mg capsules per day). Paediatric patients with BSA \<1.51 m\^2 will receive a dose adjusted for BSA. Each cycle of treatment will consist of 28 days and patients may continue until disease progression without clinical benefit, unacceptable AEs or withdrawal of consent.

    Also known as: Rozlytrek

  • DrugTrastuzumab in combination with pertuzumab

    The initial loading dose of trastuzumab is 8 mg/kg body weight followed thereafter by a maintenance dose of 6 mg/kg body weight administered intravenously every 21 days. The initial loading dose of pertuzumab is 840 mg followed thereafter by a maintenance dose of 420 mg administered intravenously every 21 days. Paediatric patients will receive a dose of pertuzumab adjusted by body weight. Patients may continue until disease progression without clinical benefit, unacceptable AEs or withdrawal of consent.

    Also known as: Herceptin in combination with Perjeta

  • DrugVemurafenib in combination with cobimetinib

    Patients will receive vemurafenib at a dose of 960 mg (four tablets of 240 mg) orally on a twice daily schedule throughout a 28-day cycle. Patients will receive cobimetinib at a dose of 60 mg (three tablets of 20 mg) to be taken orally, once daily for 21 consecutive days (days 1 to 21 in each 28-day cycle); followed by a 7-day break. Patients may continue on treatment until disease progression without clinical benefit, unacceptable AEs or withdrawal of consent.

    Also known as: Zelboraf in combination with Cotellic

  • DrugCapmatinib

    Patients will receive capmatinib orally at a daily dose of 800 mg consisting of 400 mg (two 200 mg tablets) twice daily. Each cycle of treatment will consist of 28 days and patients may continue on treatment until disease progression without clinical benefit, unacceptable AEs or withdrawal of consent.

    Also known as: Tabrecta

  • DrugDabrafenib in combination with trametinib

    Adult patients (≥18 years) will receive dabrafenib at a dose of 150 mg (two 75 mg tablets) twice daily (total daily dose 300 mg). Paediatric patients (1 to \<12 years) will receive dabrafenib dose adjusted by body weight as 10 mg dispersible tablets orally twice daily. Paediatric patients (≥12 years) and TYA patients (16 to \<18 years) will have the option to receive adult or paediatric dosing. Adult patients will receive trametinib at a daily dose of 2 mg (one 2 mg tablet) orally once daily (total daily dose 2 mg). Paediatric patients receive trametinib at a dose adjusted by body weight as 0.05 mg/m\^2 powder for oral solution twice daily. Paediatric patients (≥12 years) and TYA patients (16 to \<18 years) will have the option to receive adult or paediatric dosing. Each cycle of treatment will consist of 28 days. Patients may continue until disease progression without clinical benefit, unacceptable AEs or withdrawal of consent.

    Also known as: Tafinlar in combination with Mekinist, Finlee in combination with Spexotras

05

What researchers measure

Primary outcomes

  1. Number of patients who consent to each arm.

    This is a master screening entry with sub-study entries to capture the results of each arm. As such a primary outcome measure for this entry is not relevant, however this entry will be used to report the number of patients with a cancer containing the appropriate genetic alteration that have been successfully allocated and consented to each arm.

    Time frame: Up to 5 years.

06

Study locations

26 of 27 sites recruiting
  • Belfast City Hospital
    Belfast, BT9 7AB, United Kingdom
    • Vicky Coyle, Prof · Contact · V.Coyle@qub.ac.uk
    • Vicky Coyle, Prof · Principal investigator
    Recruiting
  • University Hospital Birmingham
    Birmingham, B15 2TT, United Kingdom
    • Gary Middleton, Prof · Contact · G.Middleton@bham.ac.uk · 0121 371 3573
    • Gary Middleton, Prof · Principal investigator
    Recruiting
  • Birmingham Children's Hospital
    Birmingham, B4 6NH, United Kingdom
    • Gerard Millen, Dr · Contact · g.millen@nhs.net · 07921843607
    • Gerard Millen, Dr · Principal investigator
    Not yet recruiting
  • Bristol Royal Hospital for Children
    Bristol, BS2 8BJ, United Kingdom
    • Antony Ng, Dr · Contact · Antony.Ng@uhbw.nhs.uk · 0117 342 8044
    • Antony Ng, Dr · Principal investigator
    Recruiting
  • Bristol Haematology and Oncology Centre
    Bristol, BS2 8ED, United Kingdom
    • Antony Ng, Dr · Contact · Antony.Ng@uhbw.nhs.uk · 0117 342 8044
    • Antony Ng, Dr · Principal investigator
    Recruiting
  • Addenbrooke's Hospital
    Cambridge, CB2 OQQ, United Kingdom
    Recruiting
  • Velindre Cancer Centre
    Cardiff, CF14 2TL, United Kingdom
    Recruiting
  • Cardiff Children's Hospital
    Cardiff, CF14 4XW, United Kingdom
    Recruiting
  • Western General Hospital
    Edinburgh, EH4 2XU, United Kingdom
    • Stefan Symeonides, Dr · Contact
    • Stefan Symeonides, Dr · Principal investigator
    Recruiting
  • The Beatson Hospital
    Glasgow, G12 OYN, United Kingdom
    Recruiting
  • Royal Hospital for Children Glasgow
    Glasgow, G51 4TF, United Kingdom
    Recruiting
  • Leicester Royal Infirmary
    Leicester, LE1 5WW, United Kingdom
    • Harriet Walter, Dr · Contact · harriet.walter1@nhs.net · 01162587602
    • Harriet Walter, Dr · Principal investigator
    Recruiting
  • Alder Hey Hospital
    Liverpool, L14 5AB, United Kingdom
    Recruiting
  • University College London Hospital
    London, NW1 2BU, United Kingdom
    • Martin Forster, Prof · Contact · M.Forster@ucl.ac.uk · 020 3447 5085
    • Martin Forster, Prof · Principal investigator
    Recruiting
  • Guy's Hospital
    London, SE1 9RT, United Kingdom
    Recruiting
  • Great Ormond Street Hospital
    London, WC1N 3JH, United Kingdom
    Recruiting
  • Royal Manchester Children's Hospital
    Manchester, M13 9WL, United Kingdom
    • Guy Makin, Dr · Contact · Guy.Makin@mft.nhs.uk · 0161 701 8419
    • Guy Makin, Dr · Principal investigator
    Recruiting
  • The Christie Hospital
    Manchester, M20 4BX, United Kingdom
    • Matthew Krebs, Dr · Contact · matthew.krebs@nhs.net · 0161 918 7672
    • Matthew Krebs · Principal investigator
    Recruiting
  • Clatterbridge Cancer Centre
    Metropolitan Borough of Wirral, CH63 4JY, United Kingdom
    Recruiting
  • Great North Children's Hospital
    Newcastle, NE1 4LP, United Kingdom
    Recruiting
  • Freeman Hospital
    Newcastle, NE7 7DN, United Kingdom
    • Alastair Greystoke, Dr · Contact · Greystoke@newcastle.ac.uk · 0191 2138476
    • Alastair Greystoke, Dr · Principal investigator
    Recruiting
  • Churchill Hospital
    Oxford, OX3 7LE, United Kingdom
    Recruiting
  • John Radcliffe Hospital
    Oxford, OX3 9DU, United Kingdom
    Recruiting
  • Weston Park Hospital
    Sheffield, S10 2SJ, United Kingdom
    Recruiting
  • Sheffield's Children's Hospital
    Sheffield, S10 2TH, United Kingdom
    • Daniel Yeomanson, Dr · Contact · danyeomanson@nhs.net · 01142717366
    • Daniel Yeomanson, Dr · Principal investigator
    Recruiting
  • Southampton General Hospital
    Southampton, SO16 6YD, United Kingdom
    • Juliet Gray, Prof · Contact · Juliet.Gray@uhs.nhs.uk · 0238 120 6639
    • Juliet Gray, Prof · Principal investigator
    Recruiting
  • The Royal Marsden Hospital
    Sutton, SM2 5PT, United Kingdom
    Recruiting
07

References and documents

08

Registry details

Key details

Study ID
NCT05722886
Lead sponsor
Cancer Research UK
Collaborators
University of Manchester, University of Birmingham, Royal Marsden NHS Foundation Trust, Hoffmann-La Roche, Novartis Pharmaceuticals
Responsible party
Sponsor
First posted
Feb 10, 2023
Start date
Mar 1, 2023
Primary completion
Oct 2029 (estimated)
Completion
Oct 2029 (estimated)
Last update
Jul 1, 2026

Study contacts

Aida Sarmiento Castro
Contact
determine@cancer.org.uk
+44 207 242 0200
Matthew Krebs, Dr
principal investigator · The Christie Hospital

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
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