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Active clinical trials for "Anemia, Sickle Cell"

Results 61-70 of 922

Intravenous Gammaglobulin for Sickle Cell Pain Crises

Sickle Cell DiseasePain

The purpose of this study is to determine whether intravenous immune globulin is safe and effective in the acute treatment of pain crises in sickle cell disease. Funding Source: Food and Drug Administration (FDA), Office of Orphan Products Development (OOPD)

Recruiting16 enrollment criteria

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other...

Sickle Cell AnemiaBeta-thalassemia Major1 more

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.

Recruiting32 enrollment criteria

Age of Blood in Sickle Cell Transfusion

Sickle Cell Disease

The Investigators hypothesize that older red cell units trigger phagocytosis and activation of circulating macrophages with a downstream immunomodulatory cascade and release of excess Non Transferrin Bound Iron(NTBI) that leads to increased rates of infection in adults with Sickle Cell Disease(SCD). To test this hypothesis, the study staff will perform a randomized prospective clinical trial. In aim 1, the study staff will determine the biochemical differences between ≥30 day-old versus ≤10 day-old units. In aim 2, the study staff will determine the physiologic effects of the transfused blood in a patient with SCD. Lastly, in aim 3, the study staff will explore the clinical implications of receiving older red cells over a 3 month period.

Recruiting10 enrollment criteria

RH Genotype Matched RBC Transfusions

Sickle Cells Disease

To determine the feasibility of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD.

Recruiting7 enrollment criteria

RHD Genotype Matching for Anti-D

Sickle Cell DiseaseAnti-D Antibodies

This is a pilot study to evaluate the feasibility and safety of providing RH genotype matched D+ Red Blood Cells (RBCs) to chronically transfused patients with sickle cell disease (SCD) who type D+ but have formed anti-D and are currently transfused with D- RBC (Red Blood Cell) units.

Recruiting7 enrollment criteria

A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell...

Sickle Cell Disease

A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).

Recruiting27 enrollment criteria

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in...

Healthy VolunteersAnemia1 more

The purpose of the study is to assess the safety and tolerability of AG-946 in healthy volunteers after oral administration of single ascending doses (SAD) and multiple ascending doses (MAD) of AG-946 over 14 or up to 28 days of dosing, and to identify a range of doses that are safe and pharmacologically active in participants with sickle cell disease. The SAD and MAD parts of the study will be randomized and double-blinded, and will assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of AG-946 as well as the effect of food (SAD only) on the pharmacokinetics (PK) of AG-946. The sickle cell disease (SCD) part of the study will be non-randomized and open-label, and is designed to identify 1 or more safe and tolerable dose(s) of AG-946 with potential activity in the treatment of participants with sickle cell disease (SCD).

Recruiting80 enrollment criteria

L-citrulline Injection in Patients Aged 6-21 Years Old With Sickle Cell Disease Presenting With...

Acute Vaso Occlusive Crisis (VOC)

The purpose of this study is to determine if intravenous L-citrulline can abrogate an active vaso-occlusive crisis in sickle cell disease, resulting in decreased pain, reduction or elimination of opiate usage, and reduction or elimination of hospital admission. The applicant is developing intravenous L-citrulline (Turnobi™) for treatment of sickle cell disease (SCD). The current development program targets treatment of sickle cell-associated vaso-occlusive crisis (VOC) specifically. The aim of Part 1 is to identify the optimum dose regimens for the Part 2 of the trial which is a double-blind, placebo controlled adaptive 'pick-the-winner' design. This study will allow assignment of more subjects to the better treatment arm/s based on emerging data. The study, initially, will evaluate efficacy and tolerability of incremental doses of intravenous (IV) L-citrulline (Turnobi™) in patients with SCD while receiving standard of care therapy for VOC.

Recruiting29 enrollment criteria

AB1 in Adult Patients With Sickle Cell Disease (SCD)

Sickle Cell Disease

This will be an open-label, dose escalating study with a starting dose of 2mg. Up to 4 additional cohorts will be enrolled at subsequently higher doses of 4mg, 8mg, 16mg, and 32mg. Each dose will be taken orally, once daily, for 8 weeks.

Recruiting28 enrollment criteria

Interest of Famotidine in Children With Sickle Cell Disease

Sickle Cell Disease

The purpose of this study is to determine whether oral famotidine, a histamine type 2 receptor antagonist already widely used with very few side effects in other indications in children, is effective in reducing endothelial expression of P-selectin in children with sickle cell disease (SCD). This pilot study will constitute the essential prerequisite for a randomized clinical trial comparing the efficacy of famotidine with that of placebo in the prevention of vaso-occlusive crises in SCD patients.

Recruiting27 enrollment criteria
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