Acute Myeloid Leukemia Clinical Trial
Official title:
Characterization Of Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
NCT number | NCT04902833 |
Other study ID # | 21-187 |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | February 1, 2022 |
Est. completion date | December 31, 2025 |
This cross-sectional prevalence assessment study involves a single blood draw in specific patient populations to assess for enzymatic and genomic evidence for acquired pyruvate kinase deficiency.
Status | Recruiting |
Enrollment | 100 |
Est. completion date | December 31, 2025 |
Est. primary completion date | April 30, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Cohort 1 - Capable and willing to provide informed consent for participation in the study. - Diagnosis of clonal cytopenia of undetermined significance (CCUS), myelodysplastic syndrome (MDS) or myelodysplastic/myeloproliferative neoplasm (MDS/MPN syndrome) according to 2016 World Health Organization (WHO) classification system. - Anemia secondary to underlying clonal cytopenia of undetermined significance (CCUS), MDS or MDS/MPN syndrome, defined as a hemoglobin <11.0 g/dL measured within 30 days of study enrollment. Anemia should not be related to nutritional deficiency (such as iron, cobalamin, folate, or copper deficiencies), peripheral immune or non-immune hemolysis, or renal disease, in the opinion of the investigator. - Age >18 years. - Cohort 2 - Capable and willing to provide informed consent for participation in the study. - Diagnosis of a clonal myeloid neoplasm, such as MDS, MDS/MPN syndrome, myeloproliferative neoplasm (MPN), acute myeloid leukemia (AML), clonal cytopenia of undetermined significance (CCUS), or other clonal myeloid neoplasm according to 2016 World Health Organization (WHO) classification system. - A diagnosis of an otherwise unexplained Coombs-negative non-immune hemolytic anemia, according to the clinical judgement of the investigator. Some form of objective laboratory evidence must be present, including one or more of the following: negative direct antiglobulin (Coombs) test, reduced haptoglobin, elevated indirect bilirubin, elevated lactate dehydrogenase, elevated aspartate aminotransferase, or compatible findings on peripheral blood film. Results of all of these tests are not required to satisfy this criterion. - Age >18 years. Exclusion Criteria: - Cohort 1 - Receipt of red cell transfusion within 60 days of study enrollment. - Have a known untreated nutritional anemia or acquired disorder resulting in hemolysis, such as paroxysmal nocturnal hemoglobinuria (PNH). A known hereditary anemia (such as thalassemia trait) is not exclusionary if the patient's baseline hemoglobin has worsened significantly (in the opinion of the investigator) after development and diagnosis of MDS. - Cohort 2 - Have a known hereditary anemic disorder, such as thalassemia, sickle cell disease, or hereditary enzyme deficiency, with the exception of hereditary X-linked glucose-6-phosphate dehydrogenase deficiency known not to cause chronic baseline hemolysis. Testing for these diagnoses is not required unless deemed clinically necessary. - Have a known untreated nutritional anemia or acquired disorder resulting in hemolysis, such as paroxysmal nocturnal hemoglobinuria (PNH). |
Country | Name | City | State |
---|---|---|---|
United States | Massachusetts General Hospital | Boston | Massachusetts |
Lead Sponsor | Collaborator |
---|---|
Massachusetts General Hospital | Agios Pharmaceuticals, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Characterization of other possible factors involved in acquired PKD, including acquired epigenetic or gene expression factors | Day 1 | ||
Primary | Overall prevalence of possible or likely acquired pyruvate kinase deficiency | defined by PK enzyme activity or PK:HK ratio >1 SD below the control mean (healthy subject mean) as measured by enzyme assay, or potentially pathogenic mutations in the PKLR gene as found on PKLR sequencing | Day 1 | |
Secondary | Overall prevalence of definite acquired pyruvate kinase deficiency | Defined by PK enzyme activity below normal (or a PK:HK ratio <8.7) as measured by enzyme assay, or known pathogenic mutations in the PKLR gene as found on PKLR sequencing | Day 1 | |
Secondary | Red cell pyruvate kinase enzyme activity | Red cell pyruvate kinase enzyme activity in patients not receiving red cell transfusion in the 60 days prior to blood draw | 60 days | |
Secondary | Red cell pyruvate kinase | hexokinase enzyme activity ratio in patients not receiving red cell transfusion in the 60 days prior to blood draw | 60 Day | |
Secondary | Somatic mutations in PKLR (and other genes associated with acquired PKD) detected in the hematopoietic clone | Day 1 | ||
Secondary | Somatic mutations in other genes associated with hemolytic anemia detected in the hematopoietic clone | Day 1 | ||
Secondary | Characterization of pyruvate kinase-related red cell metabolites (ATP, 2,3-DPG) and pyruvate kinase-R protein in patients with clonal myeloid disorders | Day 1 | ||
Secondary | Impact of pyruvate kinase activators on PK activity in vitro | Day 1 |
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