Clinical trials: Pyruvate Kinase Deficiency
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period
ACTIVATE-KidsT (AG348-C-022) is a multicenter study designed to evaluate the efficacy and safety of treatment with mitapivat compared with placebo in pediatric participants with pyruvate kinase deficiency (PK deficiency) who are…
- Trial name
- ACTIVATE-KidsT (NCT05144256)
- Testing
- Mitapivat
- Where
- 22 sites in United States, Canada and 8 more
- Volunteers
- 49 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency
Pyruvate Kinase Deficiency Epidemiological Study (PIECE)
Pyruvate kinase deficiency (PKD) is the most common red cell glycolytic enzyme defect causing hereditary non-spherocytic hemolytic anemia, caused by mutations in the PKLR gene.
- Trial name
- PIECE (NCT03866590)
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- CENTOGENE GmbH Rostock
Pyruvate Kinase Deficiency
Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
This is an open-label Phase II trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
- Trial name
- NCT06422351
- Testing
- RP-L301
- Where
- 3 sites in United States, Spain
- Volunteers
- 10 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Pyruvate Kinase Deficiency
Pyruvate Kinase Deficiency Global Longitudinal Registry
This study is an observational (ie, noninterventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare nonspherocytic hemolytic anemia.
- Trial name
- PEAK Registry (NCT03481738)
- Where
- 52 sites in United States, Canada and 14 more
- Volunteers
- 500 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period
Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not regularly receiving…
- Trial name
- ACTIVATE-Kids (NCT05175105)
- Testing
- Mitapivat
- Where
- 19 sites in United States, Canada and 5 more
- Volunteers
- 30 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency, Congenital Non-Spherocytic Hemolytic Anemia
Pyruvate Kinase Deficiency Natural History Study
The purpose of this study is to describe the range and incidence of symptoms, treatments, and complications related to pyruvate kinase deficiency (PKD).
- Trial name
- PKD NHS (NCT02053480)
- Where
- 30 sites in United States, Canada and 4 more
- Volunteers
- 254 people
- Sponsor
- Boston Children's Hospital
Pyruvate Kinase Deficiency
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
- Trial name
- NCT04105166
- Testing
- RP-L301
- Where
- 3 sites in United States, Spain
- Volunteers
- 4 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
- Conditions
- Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia
- Trial name
- NCT05777993
- Testing
- Mitapivat
- Where
- 23 sites in Belgium, Brazil and 6 more
- Volunteers
- 55 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency
Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008
This is a substudy of Study AG348-C-008, an observational, longitudinal, multicenter, global registry that aims to better understand the longitudinal clinical implications of pyruvate kinase deficiency (PK deficiency).
- Trial name
- NCT04995315
- Where
- 3 sites in United States, Italy and 1 more
- Volunteers
- 13 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study
This study is testing whether high-dose Vitamin C is safe and well-tolerated in patients with two inherited red blood cell disorders - Pyruvate Kinase Deficiency (PKD) and Class A Glucose-6-Phosphate Dehydrogenase Deficiency…
- Trial name
- G6PDA PKD (NCT07612345)
- Testing
- Vitamin C
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of Utah
Sickle Cell Disease, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Pyruvate Kinase Deficiency
A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency
Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 (mitapivat) in participants with PK deficiency.
- Trial name
- NCT02476916
- Testing
- AG-348
- Where
- 13 sites in United States, Canada and 4 more
- Volunteers
- 52 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
Study AG348-C-006 evaluated the efficacy and safety of orally administered AG-348 as compared with placebo in participants with pyruvate kinase (PK) deficiency, who were not regularly receiving blood transfusions.
- Trial name
- NCT03548220
- Testing
- AG-348
- Where
- 46 sites in United States, Brazil and 14 more
- Volunteers
- 80 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
Study AG348-C-007 was a multicenter study designed to evaluate the efficacy and safety of treatment with AG-348 in a minimum of 20, with up to 40, participants with pyruvate kinase (PK) deficiency, who were regularly receiving…
- Trial name
- NCT03559699
- Testing
- AG-348
- Where
- 19 sites in United States, Canada and 7 more
- Volunteers
- 27 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia
Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
The primary objective of this study is to understand and characterize the health-related quality of life (HRQoL) and disease burden of adult participants with PK deficiency receiving routine clinical care.
- Conditions
- Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Anemia, Anemia, Hemolytic
- Trial name
- PKD (NCT04964323)
- Where
- 5 sites in United States, Italy and 3 more
- Volunteers
- 15 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia
Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
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.
- Conditions
- Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Hereditary Hemolytic Anemia, Myelodysplastic Syndromes
- Trial name
- NCT04902833
- Testing
- Blood Draw
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Massachusetts General Hospital
Pyruvate Kinase Deficiency
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
This is an open-label, multicenter, extension study to evaluate the long-term safety, tolerability, and efficacy of treatment with mitapivat in participants who were previously enrolled in Study AG348-C-006 or Study AG348-C-007.
- Trial name
- NCT03853798
- Testing
- Mitapivat
- Where
- 42 sites in United States, Brazil and 14 more
- Volunteers
- 90 people
- Sponsor
- Agios Pharmaceuticals, Inc.