Ignite Creation Date:
2025-12-25 @ 2:54 AM
Ignite Modification Date:
2025-12-26 @ 1:35 AM
Study NCT ID:
NCT04902833
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-11-18
First Post:
2021-05-21
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
Sponsor:
Massachusetts General Hospital
Organization:
Study Overview
Official Title:
Characterization Of Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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.
Detailed Description:
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.
* Red cell pyruvate kinase enzyme activity and next-generation sequencing (NGS) hereditary hemolytic anemia panels will be performed on samples from all recruited participants.
* The study will recruit patients to two separate cohorts.
* Cohort 1 will recruit approximately 75 anemic (Hgb \<11.0 g/dL) MDS participants without overt clinical evidence of hemolysis.
* Cohort 2 will recruit approximately 25 participants with clonal myeloid disorders of any type with evidence of non-immune, otherwise unexplained hemolytic anemia
* Participation in the study involves a single blood draw. Basic information about the participant's blood disorder will also be collected.
It is expected that about 100 people will take part in this research study
* Red cell pyruvate kinase enzyme activity and next-generation sequencing (NGS) hereditary hemolytic anemia panels will be performed on samples from all recruited participants.
* The study will recruit patients to two separate cohorts.
* Cohort 1 will recruit approximately 75 anemic (Hgb \<11.0 g/dL) MDS participants without overt clinical evidence of hemolysis.
* Cohort 2 will recruit approximately 25 participants with clonal myeloid disorders of any type with evidence of non-immune, otherwise unexplained hemolytic anemia
* Participation in the study involves a single blood draw. Basic information about the participant's blood disorder will also be collected.
It is expected that about 100 people will take part in this research study
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: