MDS Clinical Trial
— PALMOfficial title:
Precision Therapy Versus Standard Therapy in Acute Myeloid Leukaemia and Myelodysplastic Syndrome in Elderly
NCT number | NCT05025098 |
Other study ID # | PALM |
Secondary ID | |
Status | Recruiting |
Phase | Phase 2 |
First received | |
Last updated | |
Start date | June 22, 2021 |
Est. completion date | June 10, 2027 |
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.
Status | Recruiting |
Enrollment | 36 |
Est. completion date | June 10, 2027 |
Est. primary completion date | June 10, 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 60 Years and older |
Eligibility | Inclusion Criteria: - 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) - WBC = 25 x109/L (prior hydroxyurea allowed for a maximum of 14 days, stopped before start of treatment) - Adequate renal and hepatic functions unless clearly disease related as indicated by the following laboratory values: - WHO performance status 0, 1 or 2 for subjects = 75 years of age OR 0 to 3 for subjects = 60 to 74 years of age. - Life-expectancy above 3 months - Signed Informed Conscent - Male Subjects Only: Male subjects who are sexually active, must agree, from Study Day 1 through at least 90 days after the last dose of study drug, to practice contraception with condom. Male subjects must agree to refrain from sperm donation from initial study drug administration until at least 90 days after the last dose of study drug. Exclusion Criteria: - 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". - Diagnosis of any previous or concomitant malignancy is an exclusion criterion: except when the patient completed successfully treatment (chemotherapy and/or surgery and/or radiotherapy) with curative intent for this malignancy at least 6 months prior to randomization. - Blast crisis of chronic myeloid leukemia. - Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, pulmonary disease etc.) - Cardiac dysfunction as defined by: - Unstable angina or - New York Heart Association Class = 2. Class 2 is defined as cardiac disease in which patients are comfortable at rest but ordinary physical activity results in fatigue,palpitations, dyspnea, or angina pain or - Unstable cardiac arrhythmias - Patients with a history of non-compliance to medical regimens or who are considered unreliable with respect to compliance with the study protocol and follow-up schedule. - Patients who have senile dementia, mental impairment or any other psychiatric disorder that prohibits the patient from understanding and giving informed consent. - Patients who do not understand the Written Informed Consent (e.g., language problems) - Current concomitant chemotherapy, radiation therapy, or immunotherapy; other than hydroxyurea. - Subject is known to be positive for HIV (HIV testing is not required). - Subject is known to be positive for hepatitis B or C infection with the exception of those with an undetectable viral load within 3 months. (Hepatitis B or C testing is not required). - AML subjects that has received strong and/or moderate CYP3A inducers within 7 days prior to the initiation of venetoclax treatment. - Subject has a malabsorption syndrome or other condition that precludes enteral route of administration. Additional exclusion criteria at the time of randomization - WHO performance status > 2 for subjects = 75 years of age OR > 3 for subjects = 60 to 74 years of age. - Progressive disease according to ELN criteria (see chapter 12 Response evaluation) - Initial treatment has made the patient eligible for allogeneic stemcell transplantation - In addition, it will be specific exclusion criteria for the patients receiving targeted therapies related to the Summary of Product Characteristics (SPC) of each drug. |
Country | Name | City | State |
---|---|---|---|
Norway | Akershus Universitetssykehus | Lørenskog |
Lead Sponsor | Collaborator |
---|---|
University Hospital, Akershus |
Norway,
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* Note: There are 33 references in all — Click here to view all references
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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) | 4-5 years | |
Secondary | 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 | 4-5 years | |
Secondary | Overall survival | Median overall survival | 4-5 years | |
Secondary | Adverse events | The number of adverse events. | 4-5 years | |
Secondary | Toxicity | The number of adverse events with CTCAE grade 3 or more | 4-5 years | |
Secondary | Transfusion independence | The number of erythrocyte- or thrombocyte-transfusionsCR: Bone marrow blasts < 5%; absence of blasts with Auer rods; absence of extramedullary disease; absolute neutrophil count >1.0 x 109/L; platelet count >100 x 109/L
CR with incomplete recovery (CRi): All CR criteria except for residual neutropenia (<1.0 x 109/L) or thrombocytopenia (<100 x 109/L) PR:All hematologic criteria of CR; decrease of bone marrow blast percentage to 5% to 25%; and decrease of pretreatment bone marrow blast percentage by at least 50% |
4-5 years | |
Secondary | Budget impact | Budget impact of providing targeted treatment to all eligible patients. | 4-5 years | |
Secondary | Overall response rate | For AML overall response rate is defined as Complete remission (CR), CR with incomplete recovery (CRi), Morphologic leukemia-free state, or Partial remission. For MDS overall response rate is defined as Complete remission, Marrow Complete Response, or Partial remission. | 4-5 years | |
Secondary | Quality of life | Difference in quality of life using EQ-5D questionnaire between treatment arms | 4-5 years | |
Secondary | Total costs | Total costs of precision therapy. | 4-5 years | |
Secondary | Progression | Median number of days to progression of MDS to AML. | 4-5 years | |
Secondary | Sequencing quality | The fraction of sequencing results with quality good enough to make a decision. | 4-5 years | |
Secondary | Dry tap | The fraction of patients who were excluded because of dry-tap. | 4-5 years | |
Secondary | Person staff time | Person-time used to sequence | 4-5 years | |
Secondary | Person-time used to find possible precision therapy | Person time on tumour board and preparations | 4-5 years | |
Secondary | Patients with actionable targets | The fraction of patients with actionable targets. | 4-5 years | |
Secondary | MDS progression to AML | The median time to AML progression for patients with MDS | 4-5 years |
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