Chart review assessing venetoclax treatment outcomes for AML - AML Real World Evidence Initiative (ARC)
| ISRCTN | ISRCTN16805365 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN16805365 |
| IRAS number | 326050 |
| Secondary identifying numbers | H18.Oncology12-SR1812, IRAS 326050, CPMS 55421 |
- Submission date
- 29/09/2023
- Registration date
- 10/11/2023
- Last edited
- 20/08/2024
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Cancer
Plain English summary of protocol
Background and study aims
This study investigates venetoclax use in the treatment of acute myeloid leukaemia (AML) in Great Britain (GB). Venetoclax is a treatment for AML that was recently approved in GB. Venetoclax is given as a combination with other medications, with different combinations available, for AML patients who have never received a previous treatment and who cannot receive intensive chemotherapy. Clinical trials have shown venetoclax works well compared to other medications, but currently, there is limited information on how it works in everyday clinics. This study will help provide information on how this treatment is used in the real world. The study aims to describe how well this treatment works, including survival, the time the treatment continues to work for the patient, how often patients have to visit their doctor or hospital when taking the treatment, and how different treatment combinations are used in GB overall.
Who can participate?
Adult patients with AML aged 18 years or older at AML diagnosis who are taking a venetoclax combination treatment (for at least 28 days) because they cannot receive chemotherapy
What does the study involve?
This multi-centre study will be run across eight to nine sites. Doctors will review medical records for AML patients treated with different venetoclax combinations and enter patient information into an online form. Patients themselves are not directly involved, and there will be no changes to their care.
Patient demographics (e.g. age, sex), clinical characteristics (e.g. other diseases or medical conditions a patient has at the time), treatment patterns (e.g. when the treatment was taken and how much was taken), effectiveness (how well the medicine works including survival), and how often patients visit their doctor or hospital whilst taking the treatment will be collected. Information that cannot reveal the patient’s identity will be collected, and this will be anonymised in the final database.
What are the possible benefits and risks of participating?
Patients will not directly participate in this study but their medical records will be reviewed by hospital staff, who will identify which records are eligible for the study. Waiver for Informed Consent has been granted by HRA, as no personal or sensitive data is collected as part of this research. Any medical record data collected will be pseudonymised.
Where is the study run from?
AbbVie (UK)
When is the study starting and how long is it expected to run for?
November 2022 to May 2024
Who is funding the study?
AbbVie Inc (US)
Who is the main contact?
Abbvie UK Medical Information, ukmedinfo@abbvie.com
Contact information
Principal Investigator
MRC Molecular Haematology Unit
Weatherall Institute of Molecular Medicine
Oxford
OX3 9DS
United Kingdom
 ORCID ID |
0000-0003-3931-0914 |
|---|---|
| Phone | +44 (0)1865 222309, +44 (0)1865 22489 |
| paresh.vyas@imm.ox.ac.uk |
Public, Scientific
AbbVie House
Vanwall Business Park
Vanwall Road
Maidenhead, Berkshire
SL6 4UB
United Kingdom
| Phone | +44 (0)1628494026 |
|---|---|
| ukmedinfo@abbvie.com |
Study information
| Study design | Non-interventional retrospective multicentre cohort study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Retrospective chart review |
| Study setting(s) | Medical and other records |
| Study type | Other, Efficacy |
| Participant information sheet | No participant information sheet available |
| Scientific title | A centre-based chart review study to assess treatment outcomes of venetoclax for the treatment of acute myeloid leukemia (AML) |
| Study objectives | To describe the real world outcomes and treatment patterns in newly diagnosed AML patients who are ineligible for intensive chemotherapy receiving venetoclax-based combinations in Great Britain |
| Ethics approval(s) |
1. Approved 13/04/2023, HRA (Ground Floor, Skipton House, 80 London Road, London, SE1 6LH, United Kingdom; +44 (0)207 104 8000; approvals@hra.nhs.uk), ref: 23/PR/0342 2. Approved 13/04/2023, Health and Care Research Wales (HCRW) (Ground Floor, Skipton House, 80 London Road, London, SE1 6LH, United Kingdom; +44 (0)207 104 8000; HCRW.approvals@wales.nhs.uk), ref: 23/PR/0342 |
| Health condition(s) or problem(s) studied | Acute myeloid leukaemia |
| Intervention | Patients receiving venetoclax combination therapies for their acute myeloid leukaemia (AML) will be eligible for inclusion in the study, as per the inclusion criteria. This study is retrospective in design and so patients will not be consented to participate, instead under the NHS guidance, their data will be accessed by their direct care team only. Once a patient has been identified for inclusion, they will be enrolled and their data will be accessed and entered into the electronic data capture (EDC) system. As the study is retrospective, the patients will not be followed up for any prespecified amount of time. The observation period for the study is from diagnosis of AML up to the most recently available data at the time of data entry. Their data will simply be entered into the EDC and once all data has been entered for each patient, and all data queries have been answered, the study will close. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | Not Applicable |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | Venetoclax |
| Primary outcome measure | 1. Overall survival (OS) will be measured as the number of days between the initiation of the studied line of therapy (i.e., venetoclax-based regimen) until death (event) or end of follow-up (censoring). 2. Event-free survival (EFS) will be measured as the number of days between initiating the studied line of therapy and disease progression, refractory disease, or death; both outcomes will be censored at the end of follow-up. |
| Secondary outcome measures | 1. AML healthcare resource utilisation (HRU) measured using data entered into the electronic data capture (EDC) system at one timepoint 2. AML treatment patterns for the venetoclax cohort measured using data entered into the electronic data capture (EDC) system at one timepoint |
| Overall study start date | 01/11/2022 |
| Completion date | 06/05/2024 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Upper age limit | 99 Years |
| Sex | Both |
| Target number of participants | 150-200 |
| Total final enrolment | 168 |
| Key inclusion criteria | 1. The patient received a diagnosis for AML, if available, suggested diagnosis codes: ICD-9 205.0x or ICD-10 C92.0x, C92.4x, C92.5x 2. The patient was at least 18 years old at AML diagnosis date 3. The patient was initiated on the studied line of therapy (see Table 1 for the list of eligible treatments included based on NCCN guidelines and clinical input) for previously untreated AML: 3.1. Venetoclax in combination with an HMA (on or after 28th May 2021) or LDAC (on or after 25th February 2022), the date of MHRA approvals of venetoclax (for the treatment of adult patients with newly diagnosed AML who are ineligible for intensive chemotherapy) 4. The patient was treated with the studied line of therapy at least 28 days prior to the date of data collection 5. Information on the patient's treatments, selected (or important) clinical characteristics, and outcomes is available from the start of the studied line of therapy onwards |
| Key exclusion criteria | 1. The patient received the studied line of therapy as part of a clinical trial 2. The patient received prior lines of therapy for AML 3. The patient has a history of malignancies within 2 years prior to the studied line of therapy, other than AML, and with the exception of: 3.1. Myelodysplastic syndromes (MDS), myeloproliferative neoplasm (MPN) or chronic myelomonocytic leukemia (CMML) 3.2. Adequately treated in situ carcinoma of the cervix uteri or carcinoma in situ of the breast 3.3. Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin 3.4. Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent |
| Date of first enrolment | 31/10/2023 |
| Date of final enrolment | 08/04/2024 |
Locations
Countries of recruitment
- England
- United Kingdom
- Wales
Study participating centres
OX3 7LE
United Kingdom
CF14 4XW
United Kingdom
LS9 7TF
United Kingdom
HA1 3UJ
United Kingdom
NG7 2UH
United Kingdom
B15 2GW
United Kingdom
NW1 2BU
United Kingdom
PO6 3LY
United Kingdom
S10 2JF
United Kingdom
Sponsor information
Industry
Abbvie House
Vanwall Business Park
Vanwall Rd
Maidenhead
SL6 4UB
England
United Kingdom
| Phone | +44 (0)1628 561090 |
|---|---|
| ukmedinfo@abbvie.com | |
| Website | http://www.abbvie.co.uk/ |
"ROR" |
https://ror.org/04tnbfn25 |
Funders
Funder type
Industry
Government organisation / For-profit companies (industry)
- Alternative name(s)
- AbbVie Inc., AbbVie U.S., AbbVie US, Allergan
- Location
- United States of America
Results and Publications
| Intention to publish date | 06/05/2025 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not expected to be made available |
| Publication and dissemination plan | Intend to publish the data through conference posters and a manuscript approximately 1 year after the end date of the study |
| IPD sharing plan | For data privacy reasons, there are no plans to share individual patient level data (IPD). AbbVie will only have access to summary data tables. |
Editorial Notes
20/08/2024: The total final enrolment was added.
01/12/2023: Internal review.
08/10/2023: Study's existence confirmed by HRA and Health and Care Research Wales (HCRW) (UK).
