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Active clinical trials for "Anemia, Sickle Cell"

Results 1-10 of 922

L-Arginine in Children Having Sickle Cell Disease With Increased Tricuspid Regurgitant Jet Velocity...

Sickle Cell Disease

This study aims to investigate the possible efficacy and safety of L-Arginine in children having Sickle Cell Disease with increased Tricuspid Regurgitant Jet Velocity

Recruiting8 enrollment criteria

GBT021601-021: A Study in Adult and Pediatric Participants With SCD

Sickle Cell Disease

The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of GBT021601.

Recruiting12 enrollment criteria

Hyperbaric-oxygen Therapy (HBOT) Versus Placebo for Treating Vaso-Occlusive Crisis (VOC) in Sickle...

Sickle Cell DiseaseHyperbaric Oxygen Therapy1 more

This is a randomised, controlled, double-blind, placebo trial of HBOT (intervention) superiority in the treatment of VOC in SCD, to demonstrate the effectiveness of HBOT for the decrease in pain level in the treatment of SCD-VOC.

Recruiting12 enrollment criteria

Sleep and Pain in Sickle Cell Disease

Sickle Cell DiseaseSleep Disturbance1 more

This is a study testing the effects of behavioral sleep interventions on pain and brain function in sickle cell disease.

Recruiting9 enrollment criteria

Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation...

Sickle Cell Disease

Background: Peripheral blood stem cell transplantation procedures are used for people with sickle cell disease. Researchers want to improve the success and reduce the complications for these procedures. This might allow more people to have a transplant. Objective: To see if a new transplant regime is effective, safe and well tolerated in people with sickle cell disease. Eligibility: Adults at least 18 years old with sickle cell disease and certain complications. A relative who is a half tissue match. Design: Participants will be screened with medical history, physical exam, and blood tests. Recipients will also have: Heart, lung, and mental health tests Chest x-rays Bone marrow taken from the pelvic bone Eyes and teeth checked Recipients will have a large central line inserted into a vein for up to 6 months. Donors will have their veins tested and have an IV inserted for 1 day or on rare occasions 2 days. Donors will get a drug to activate bone marrow. It will be injected for about 6 days. Donors will have at least 1 five-hour procedure where bone marrow stem cells will be collected. Blood will be taken from a vein in one arm or in rare cases from a groin vein and put through a machine. Some blood will be saved and the rest will be returned. Stem cells will be taken from the saved blood in a lab and frozen until ready to give to the recipient. Recipients will have: Stems cells collected and frozen Hygiene lessons Bone density scans Low-dose radiation Drugs for their immune system Donor cells infused through their central line Transfusions After about 30 days, recipients will leave the hospital. They must stay near NIH for 3 months after the transplant and have frequent visits. After returning home, they will have 8 visits over 5 years, then be contacted yearly. ...

Recruiting24 enrollment criteria

Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible...

Sickle Cell DiseaseStem Cell Transplant Complications2 more

The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression. This study will expand the access of HSCT for patients with SCD who are currently not eligible because of donor restrictions.

Recruiting18 enrollment criteria

Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease

Sickle Cell Disease

This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years. Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent participants with Sickle Cell Disease ages 12 to 17 years. Part C is a multiple dose, safety, tolerability, and PK study, which includes the assessment of hematological effects and the effect on TCD flow velocity of voxelotor in pediatric participants with Sickle Cell Disease ages 4 to 17 years. Part D is a multiple dose, safety, tolerability, and PK study, which examines the hematological effects of voxelotor in pediatric participants with Sickle Cell Disease ages 6 months to < 4 years.

Recruiting22 enrollment criteria

A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic...

Sickle Cell Disease

This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell transplantation using BIVV003.

Recruiting32 enrollment criteria

Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients...

Sickle Cell Disease (SCD)

The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 6 months to <18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years. The efficacy and safety of crizanlizumab was already demonstrated in adults with sickle cell disease. The approach is to extrapolate from the PK/pharmacodynamics (PD) already established in the adult population. The study is designed as a Phase II, multicenter, open-label study.

Recruiting34 enrollment criteria

Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease...

Sickle Cell Disease

The purpose of this study is to investigate the effect of VIT-2763 on markers of hemolysis (breakdown in red blood cells) in sickle cell disease (SCD). The safety, tolerability and clinical beneficial effects of VIT-2763 for the treatment of SCD are also explored.

Recruiting22 enrollment criteria
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