Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

Light Exposure Patterns and Symptoms Among Patients With Erythropoietic Protoporphyria

NCT03682731 ·Status: COMPLETED

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

NCT04819841 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype

NCT07282210 ·Status: NOT_YET_RECRUITING ·Phase: PHASE2

Sorbent Therapy of the Cutaneous Porphyrias

NCT01422915 ·Status: COMPLETED ·Phase: PHASE2/PHASE3

Dose-Escalation Study of SCD-101 in Sickle Cell Disease

NCT02380079 ·Status: UNKNOWN ·Phase: PHASE1

Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease

NCT00842088 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

NCT00006136 ·Status: COMPLETED ·Phase: PHASE2

PK and PD Responses to Oral L-Citrulline in Patients With Sickle Cell Disease

NCT00343954 ·Status: UNKNOWN ·Phase: PHASE1

A Dose-Finding Study of AG-348 in Sickle Cell Disease

NCT04000165 ·Status: COMPLETED ·Phase: EARLY_PHASE1

A Study to Access the Safety/Efficacy of Thalidomide in the Treatment of Anemia in Patients With Myelodysplastic Syndromes

NCT00050843 ·Status: COMPLETED ·Phase: PHASE3

Protection Against Visible Light by Dihydroxyacetone in Erythropoietic Protoporphyria

NCT05780840 ·Status: UNKNOWN ·Phase: NA

A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease

NCT03132324 ·Status: TERMINATED ·Phase: PHASE1

Relative Contributions of Red Cell Catabolism and Dietary Absorption to Fetal Iron Accretion During Pregnancy

NCT01588665 ·Status: COMPLETED

Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease

NCT01000155 ·Status: TERMINATED ·Phase: PHASE2

Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.

NCT01571635 ·Status: TERMINATED ·Phase: PHASE2

Oral Iron for Erythropoietic Protoporphyrias

NCT02979249 ·Status: COMPLETED ·Phase: NA

Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias

NCT00004396 ·Status: COMPLETED ·Phase: PHASE2

Effects of HQK-1001 in Patients With Sickle Cell Disease

NCT01601340 ·Status: TERMINATED ·Phase: PHASE2

Low Dose Iron Chelation as TReatment of Oxidative Damage in Sickle Cell Disease

NCT05392101 ·Status: COMPLETED ·Phase: PHASE2

A Study of the Effect of IW-1701 (Olinciguat), a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD)

NCT03285178 ·Status: COMPLETED ·Phase: PHASE2

Iron Therapy in Erythropoietic Protoporphyria

NCT05572125 ·Status: COMPLETED

Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

NCT01709032 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Longitudinal Study of the Porphyrias

NCT01561157 ·Status: RECRUITING

Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact

NCT01688895 ·Status: COMPLETED

Effectiveness of Arginine as a Treatment for Sickle Cell Anemia

NCT00513617 ·Status: COMPLETED ·Phase: PHASE2