Clinical trials: Pyruvate Kinase Deficiency
- Condition Pyruvate Kinase Deficiency
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
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.