Acute Myeloid LeukemiaNCT05312112
Real World Outcomes Using Novel Agents for AML in the UK
An observational study of Acute Myeloid Leukemia, run by Guy's and St Thomas' NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
This project will collect data on patients with acute myeloid leukemia in the United Kingdom who were treated with two new targeted therapies during the coronavirus pandemic Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Venetoclax and Gilteritinib
- How many people
- 1,000 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 1 May 2022to 1 Oct 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- Child, Adult, Older adult
Study population: The NHS criteria for access to venetoclax was that a patient was fit for IC and was: * Aged >16y with NPM1 mutation without FLT3 internal tandem duplication (ITD) * Aged >50y with NPM1, IDH1 or IDH2 mutations (regardless of FLT3 status) * Patients aged >60y without favourable-risk cytogenetics Gilteritinib was made available to all patients aged >16y with relapse or refractory FLT3 mutated AML. Other FLT3 inhibitors are available to patients through various access schemes
Venetoclax cohort Inclusion criteria
- Newly diagnosed acute myeloid leukaemia
- No prior therapies for AML, apart from hydroxyurea (or similar) for cytoreduction. Previous treatments for MDS or other conditions are allowed
- Treated with venetoclax in combination with either azacitidine or LDAC No exclusion criteria
Gilteritinib/FLT3 cohort Inclusion criteria
- Relapsed acute myeloid leukaemia, including molecular relapse
- Treated with FLT3 inhibitor No exclusion criteria
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site, 1 recruiting
- Recruiting
Guy's and St Thomas' NHS Foundation Trust
London, SE1 9RT
Richard Dillon
About this study
Official title: Real World Outcomes Using Novel Agents for Acute Myeloid Leukaemia in the United Kingdom
This project will collect data on patients with acute myeloid leukemia in the United Kingdom who were treated with two new targeted therapies during the coronavirus pandemic
Read the detailed description
Acute myeloid leukaemia (AML) is a blood cancer which in fit young adults is typically treated with intensive chemotherapy. While this is potentially curative, it is associated with significant side effects and the requirement for long hospital admissions. Infection is a major issue during AML treatment, as both the disease and the chemotherapy impair the immune system.
Early data suggested that COVID-19 is associated with a very high rate of death in AML patients undergoing intensive chemotherapy. Because of this, and the need for significant hospital resources to deliver intensive chemotherapy, the NHS made available two new, less intensive, targeted therapies for the treatment of AML during the COVID-19 pandemic - venetoclax and gilteritinib. The aim was to reduce mortality and healthcare resource use.
Many hundreds of patients across the UK have been treated with these two medications on the temporary access scheme. The research aims to collect de-identified data from treating patients to describe the outcomes of patients treated with these approaches, both in terms of the safety and effectiveness.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Venetoclax, Gilteritinib
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VenetoclaxDrug
Also called Venclyxto
Observational study of venetoclax in AML
GilteritinibDrug
Also called Xospata
Observational study of gilteritinib in AML
| Study group | Role | What they receive |
|---|---|---|
| Venetoclax | Venetoclax in newly diagnosed AML Receives: Drug: Venetoclax | |
| FLT3 inhibitors | FLT3 inhibitors including gilteritinib in relapsed AML Receives: Drug: Gilteritinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
Overall survival measured from time of treatment initiation
Measured: 1 year
Early death rate
Early death rate measured at day 60 after treatment initiation
Measured: Day 60 after starting treatment
Secondary outcomes (7)
Response rate
Response rate as defined by ELN 2017
Measured: After 2 cycles of therapy (each cycle is 28 days although may be extended if recovery is delayed)
Incidence of relapse in patients achieving remission
Relapse incidence measured from the time of achieving remission
Measured: 1 year
Relapse-free survival
RFS as defined by ELN
Measured: 1 year
Treatment toxicity 1
Number of days in hospital and number of days of intensive care
Measured: During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)
Treatment toxicity 2
Duration of neutropenia and thrombocytopenia
Measured: During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)
Treatment toxicity 3
Number of blood and platelet transfusions
Measured: During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)
Comparison of survival between patient sub-groups
Overall survival compared between disease groups
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2022
- Study start1 May 2022
- Primary completion1 Oct 2023 (estimated)
- Study completion1 Oct 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Guy's and St Thomas' NHS Foundation TrustThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guy's and St Thomas' NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- King's College London
- Responsible party
- Sponsor
- Investigators
- Richard Dillon, Principal investigator, King's College London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05312112
- Sponsor's protocol ID
- 305432
- Other IDs
- 305432
- Status last verified
- Feb 2023
- First submitted
- 9 Mar 2022
- Last updated
- 2 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real World Outcomes Using Novel Agents for Acute Myeloid Leukaemia in the United Kingdom
- ClinicalTrials.gov ID
- NCT05312112
- Acronym / sponsor ID
- 305432
- Conditions
- Acute Myeloid Leukemia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- Guy's and St Thomas' NHS Foundation TrustUniversity, hospital or other
- Collaborators
- King's College London
- Study start
- 1 May 2022 (actual)
- Primary completion
- 1 Oct 2023 (estimated)
- Study completion
- 1 Oct 2023 (estimated)
- First posted
- 5 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This project will collect data on patients with acute myeloid leukemia in the United Kingdom who were treated with two new targeted therapies during the coronavirus pandemic
Detailed description
Acute myeloid leukaemia (AML) is a blood cancer which in fit young adults is typically treated with intensive chemotherapy. While this is potentially curative, it is associated with significant side effects and the requirement for long hospital admissions. Infection is a major issue during AML treatment, as both the disease and the chemotherapy impair the immune system.
Early data suggested that COVID-19 is associated with a very high rate of death in AML patients undergoing intensive chemotherapy. Because of this, and the need for significant hospital resources to deliver intensive chemotherapy, the NHS made available two new, less intensive, targeted therapies for the treatment of AML during the COVID-19 pandemic - venetoclax and gilteritinib. The aim was to reduce mortality and healthcare resource use.
Many hundreds of patients across the UK have been treated with these two medications on the temporary access scheme. The research aims to collect de-identified data from treating patients to describe the outcomes of patients treated with these approaches, both in terms of the safety and effectiveness.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Venetoclax | – | Drug: VenetoclaxVenetoclax in newly diagnosed AML |
| FLT3 inhibitors | – | Drug: GilteritinibFLT3 inhibitors including gilteritinib in relapsed AML |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- The NHS criteria for access to venetoclax was that a patient was fit for IC and was: * Aged >16y with NPM1 mutation without FLT3 internal tandem duplication (ITD) * Aged >50y with NPM1, IDH1 or IDH2 mutations (regardless of FLT3 status) * Patients aged >60y without favourable-risk cytogenetics Gilteritinib was made available to all patients aged >16y with relapse or refractory FLT3 mutated AML. Other FLT3 inhibitors are available to patients through various access schemes
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VenetoclaxAlso: VenclyxtoObservational study of venetoclax in AML | Drug | Venetoclax |
| GilteritinibAlso: XospataObservational study of gilteritinib in AML | Drug | FLT3 inhibitors |
Primary outcome
Overall survival
Time frame: 1 year
Overall survival measured from time of treatment initiation
Early death rate
Time frame: Day 60 after starting treatment
Early death rate measured at day 60 after treatment initiation
7 secondary outcomes
Response rate
Time frame: After 2 cycles of therapy (each cycle is 28 days although may be extended if recovery is delayed)
Incidence of relapse in patients achieving remission
Time frame: 1 year
Relapse-free survival
Time frame: 1 year
Treatment toxicity 1
Time frame: During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)
Treatment toxicity 2
Time frame: During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)
Treatment toxicity 3
Time frame: During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)
Comparison of survival between patient sub-groups
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2023.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Richard Dillon principal investigator
King's College London
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Myeloid Leukemia, run by Guy's and St Thomas' NHS Foundation Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 2 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Richard Dillon (020 7188 257, richard.dillon@kcl.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Mar 2023.