Precision Therapy Versus Standard Therapy in AML and MDS in Elderly
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age60+
SponsorUniversity Hospital, Akershus
About this trial
This is a randomized clinical trial that randomizes between treatment principles. The study will investigate if precision therapy determined by a tumour board is better than standard treatment for acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) in elderly. The tumour board will decide the precision therapy based on identified genetic changes that can guide customized therapy. There are currently 40-50 targeted therapies approved for various cancers in Norway. The precision therapy will be given in addition to the standard treatment.
The primary study objective will be to evaluate the cost-effectiveness of a precision therapy strategy compared with standard treatment. Other objectives will mesaure efficacy and satety of the treatment, and impact on life quality of the patients.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Patients with a diagnosis of AML and related precursor neoplasms according to WHO 2016 classification (excluding acute promyelocytic leukemia) including secondary AML (after an antecedent hematological disease and therapy-related AML (not if they have received antileukemic/mds treatment)), or
acute leukemias of ambiguous lineage according to WHO 2016 or a diagnosis of myelodysplastic syndrome (MDS) with IPSS-R > 4.5.1
Patients 60 years and older.
Patients must NOT be eligible for intensive chemotherapy or allogeneic stem cell therapy (See Chapter 23.3)
Disqualifiers
Acute promyelocytic leukemia.
AML with favourable cytogenetic or genetic changes in patients who are fit for intensive chemotherapy. Favourable genetics are: t(8;21)(q22;q22.1); RUNX1-RUNX1T1; inv(16)(p13.1q22) or t(16;16)(p13.1;q22); CBFB-MYH11; mutated NPM1 without FLT3-ITD or with FLT3-ITDlow†; Biallelic mutated CEBPA
Patients previously treated for AML or MDS (any antileukemic therapy or MDS treatment including investigational agents).
Patients where it is not possible to get bone-marrow for NGS, i.e., "dry tap".
Trial design
Design model
Parallel
Treatments tested in this trial
Standard therapy
DrugFor AML patients: Venetoclax will be administered orally once daily Days 1 through 28, of a 28-day cycle, with a designated dose of 400 mg daily after ramp up in Cycle 1. During Cycle 1 Days 1 - 3, the dose of Venetoclax will ramp up from 100 mg on Day 1, 200 mg on Day 2, and 400 mg on Day 3. Azacitidine (100 mg/m2) is given once daily following administration of venetoclax for 5 days of every cycle, starting on Day 1 of each cycle. For MDS patients: Azacitidine (100 mg/m2) is given once daily for 5 days of every 28 day cycle, starting on day 1 of each cycle.
Precision therapy
DrugPrecision therapy for both AML and MDS patients is standard therapy + tumor board determined precision therapy. The tumor board determined precision therapy can be all available drugs with a marketing authorization in Norway.
Treatment groups
36 Participants
are divided into 2 treatment groupsGroup A: Standard therapyActive comparator 1 intervention
Group B: Precision therapyExperimental treatment 2 interventions
Trial outcomes
Primary outcomes
1
cost-effectiveness of a precision therapy
To evaluate the cost-effectiveness of a precision therapy strategy compared with standard treatment. The total cost of the treatment will be calculated and divided on health related quality of life (HRQoL)
Time frame
4-5 yearsSecondary outcomes
1
Event free survival
Median event free survival, i.e., the number of days to death or progression of the disease (whichever comes first)with AML or MDS
Time frame
4-5 years2
Overall survival
Median overall survival
Time frame
4-5 years3
Adverse events
The number of adverse events.
Time frame
4-5 years4
Toxicity
The number of adverse events with CTCAE grade 3 or more
Time frame
4-5 yearsLocations
1Map coordinates are unavailable for these locations. Locations are shown below instead.
Akershus UniversitetssykehusRecruiting Lørenskog
Norway
NorwaySponsors and contacts
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Source ClinicalTrials.gov