A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）

Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT03439839 ·Status: COMPLETED ·Phase: PHASE2

Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH

NCT03866681 ·Status: UNKNOWN ·Phase: PHASE4

Hydroxyurea in the Treatment of Sickle Cell Disease

NCT02709681 ·Status: COMPLETED

Safety Evaluation Study for Patients With Polycythemia Vera

NCT05074550 ·Status: UNKNOWN ·Phase: PHASE1

Real-life Treatment Outcomes of Ravulizumab in PNH

NCT06633536 ·Status: ACTIVE_NOT_RECRUITING

Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria

NCT01642979 ·Status: UNKNOWN ·Phase: PHASE2

A Study of Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 9MW3011 in Patients With Polycythemia Vera

NCT06752746 ·Status: RECRUITING ·Phase: PHASE1

A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

NCT06546670 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Hydroxyurea in Young Children With Sickle Cell Anemia

NCT00519701 ·Status: COMPLETED ·Phase: NA

Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia

NCT00001197 ·Status: COMPLETED ·Phase: PHASE2

Safety, Tolerability and Pharmacokinetics of FTX-6058

NCT04586985 ·Status: COMPLETED ·Phase: PHASE1

REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria

NCT07229235 ·Status: RECRUITING

Global PNH Patient Registry

NCT05755867 ·Status: RECRUITING

Multicenter Retrospective Analysis About the Clinical Characteristics of Korean Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

NCT01224483 ·Status: UNKNOWN

Multicenter Study of Hydroxyurea in Patients With Sickle Cell Anemia (MSH)

NCT00000586 ·Status: COMPLETED ·Phase: PHASE3

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years

NCT05660265 ·Status: TERMINATED ·Phase: PHASE1

Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure Syndromes

NCT01192425 ·Status: COMPLETED

A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)

NCT04983264 ·Status: COMPLETED ·Phase: PHASE1

Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease

NCT02225132 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease

NCT06578507 ·Status: RECRUITING ·Phase: PHASE2

Identification of Factors Associated With Treatment Response in Patients With Polycythemia Vera, Essential Thrombocythemia, and Pre-myelofibrosis.

NCT05440838 ·Status: RECRUITING

Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease

NCT05565092 ·Status: TERMINATED ·Phase: PHASE2

Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron Overload

NCT00560820 ·Status: COMPLETED ·Phase: PHASE1

Study of Hydroxyurea to Treat Sickle Cell Disease

NCT02868138 ·Status: COMPLETED

Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia

NCT06761560 ·Status: NOT_YET_RECRUITING ·Phase: PHASE2/PHASE3