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
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
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
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.