Clinical trials: Erythrocyte Disorder
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.
Anemia, Chronic Kidney Disease
Peginesatide for Anemia in Chronic Hemodialysis Patients
The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and pharmacokinetics (PK) of multiple intravenous doses of peginesatide in participants with chronic kidney disease (CKD) who are on hemodialysis.
- Conditions
- Anemia, Chronic Kidney Disease, Chronic Renal Failure
- Trial name
- NCT00228449
- Testing
- peginesatide
- Where
- 14 sites in United States
- Volunteers
- 165 people
- Sponsor
- Affymax
Anemia, Leukemia, Lymphocytic, Chronic
Epoetin Alfa for the Treatment of Anemia Resulting From Chronic Lymphocytic Leukemia
The purpose of this study is to evaluate the effect of treatment with epoetin alfa versus placebo on the percentage of red blood cells in anemic patients with chronic lymphocytic (white blood cell) leukemia and its effect on the…
- Conditions
- Anemia, Leukemia, Lymphocytic, Chronic, Leukemia, Lymphocytic, Cancer
- Trial name
- NCT00270049
- Testing
- epoetin alfa
- Volunteers
- 195 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Myeloproliferative Disorders, Myelofibrosis
An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions
The purpose of this Phase 3 study is to evaluate the efficacy and safety of Luspatercept compared with placebo in subjects with myeloproliferative neoplasm (MPN)-associated Myelofibrosis (MF) and anemia on concomitant Janus…
- Conditions
- Myeloproliferative Disorders, Myelofibrosis, Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis
- Trial name
- INDEPENDENCE (NCT04717414)
- Testing
- ACE-536
- Where
- 185 sites in United States, Argentina and 24 more
- Volunteers
- 313 people
- Sponsor
- Celgene
Immune Thrombocytopenic Purpura
A Safety Study of Intravenous Immunoglobulin in Patients With Chronic Immune Thrombocytopenic Purpura (ITP)
It is known that intravenous immunoglobulins can induce hemolysis, but the mechanism is not known in detail. The primary objective of this study was to investigate the specificity of antigens on red blood cells in patients with…
- Trial name
- NCT01390649
- Testing
- IgPro10
- Where
- 17 sites in Bulgaria, Romania and 1 more
- Volunteers
- 57 people
- Sponsor
- CSL Behring
Parkinson's Disease
Study to Investigate the Tolerability, Steady-state Pharmacokinetics and Erythrocyte COMT Inhibition of BIA 3-202
The purpose of this study is to investigate the tolerability and safety of three multiple dose regimens of nebicapone (BIA 3-202 100 mg, 200 mg, and 300 mg 6 times daily) in healthy volunteers.
- Trial name
- NCT02772627
- Testing
- Nebicapone
- Where
- Portugal
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Bial - Portela C S.A.
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Myelodysplastic Syndromes, Anemia
A Study to Evaluate Ofirnoflast in Adults With Very Low- to Intermediate-risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions
The primary objective of this study is to two part. First, dose selection for dose optimization based on safety of ofirnoflast. Second, to evaluate the efficacy of ofirnoflast administered in the study population.
- Trial name
- NCT07738510
- Testing
- Ofirnoflast
- Volunteers
- 70 people
- Sponsor
- Halia Therapeutics, Inc.