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.
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