Sickle Cell Disease clinical trials near you

8 shown of 138

8 shown of 138 clinical trials.

1. RecruitingPhase 1InterventionalNCT06647979

   Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

   Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia

   A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace a diseased gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of Graft-Versus-Host Disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to fix or replace a diseased gene is called gene editing. A person's own cells are edited using a specialized biological medicine that has been formulated for use in human beings. Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. Investigators have recently discovered a gene called BCL11A that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition.

   Sites

   1

   Target

   10

   Study sites

   Boston, US

   Sponsor: Daniel Bauer

   View on ClinicalTrials.gov(opens in a new tab) Express interest
2. RecruitingPhase 1InterventionalNCT07708350

   Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease

   Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)

   A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly deoxyribonucleic acid (DNA)) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace a diseased gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of Graft-Versus-Host Disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to fix or replace a diseased gene is called gene editing. A person's own cells are edited using a specialized biological medicine that has been formulated for use in human beings. Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. Investigators have recently discovered a gene called BCL11A that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition.

   Sites

   1

   Target

   5

   Study sites

   Boston, US

   Sponsor: Daniel Bauer

   View on ClinicalTrials.gov(opens in a new tab) Express interest
3. RecruitingObservationalNCT07800247

   Hemoglobin Disorders in Egypt: Sickle Cell Disease Ain Shams University Institutional Registry

   Sickle Cell Disease (SCD)

   This is a retrospective descriptive study to create an institutional registry that will continue as a prospective follow-up non-intervention study that will be carried out at Hematology Oncology and BMT Department, Children's Hospital, Ain-Shams University, Cairo, Egypt.

   Sites

   2

   Target

   400

   Study sites

   Cairo, EG +1 more

   Sponsor: Ain Shams University

   View on ClinicalTrials.gov(opens in a new tab) Express interest
4. RecruitingInterventionalNCT03976180

   High-flow Oxygen for Vaso-occlusive Pain Crisis

   Sickle Cell Disease

   Sickle cell disease (SCD) is characterized by recurrent vaso-occlusive pain crisis (VOC), which may evolve to acute chest syndrome (ACS), the most common cause of death among adult patients with SCD. Currently, there is no safe and effective treatment to abort VOC or prevent secondary ACS. Management of VOC mostly involve a symptomatic approach including hydration, analgesics, transfusion, and incentive spirometry, which was investigated in a very limited number of patients (\ The polymerisation of HbS is one major feature in the pathogenesis of vaso-occlusion. Among factors determining the rate and extent of HbS polymer formation, the hypoxic stimulus is one of the most potent and readily alterable. Current guidelines recommend oxygen therapy in patients with VOC in order to maintain a target oxygen saturation of 95%. Low-flow nasal oxygen (LFNO) is routinely used to achieve this normoxia approach, particularly in patients at risk of secondary ACS because they may experience acute desaturation. In contrast, various case series suggest a potential beneficial role of intensified oxygen therapy targeting hyperoxia for the management of VOC, particularly with the use of hyperbaric oxygen, but the latter is difficult to implement in routine clinical practice. A recent high-flow nasal oxygen (HFNO) technology allows the delivery of humidified gas at high fraction of inspired oxygen (FiO2) through nasal cannula. The FiO2 can be adjusted up to 100% (allowing hyperoxia that may reverse sickling) and the flow can be increased up to 60 L/min (which generates positive airway pressure and dead space flushing, that may prevent evolution of VOC towards ACS by alleviating atelectasis and opioid-induced hypercapnia). In patients with acute respiratory failure, HFNO has been shown to improve patient's comfort, oxygenation, and survival as compared to standard oxygen or non-invasive ventilation. The aim of the present study is to test the efficacy and safety of HFNO for the management of VOC and prevention of secondary ACS. The investigators will use a multi-arm multi-stage (MAMS) design to achieve these goals. HFNO will be delivered through AIRVO 2 (Fisher and Paykel Healthcare, New Zealand), a device that incorporates a turbine allowing its use in hospital wards.

   Sites

   1

   Target

   350

   Study sites

   Créteil, FR

   Sponsor: Assistance Publique - Hôpitaux de Paris

   View on ClinicalTrials.gov(opens in a new tab) Express interest
5. RecruitingObservationalNCT04093986

   Hydroxyurea Exposure Limiting Pregnancy and Follow-Up Lactation

   Sickle Cell Disease, Sickle Cell Anemia

   The purpose of this research study is to document and understand the effects of hydroxyurea exposure for women with SCD and their babies, during both gestation and lactation.

   Sites

   1

   Target

   200

   Study sites

   Cincinnati, US

   Sponsor: Children's Hospital Medical Center, Cincinnati

   View on ClinicalTrials.gov(opens in a new tab) Express interest
6. RecruitingObservationalNCT07001189

   Impact of Regional Anesthesia on Inflammatory Mechanisms During Vaso-occlusive Crisis in Sickle Cell Patients

   Sickle Cell, Sickle Cell Disease, Vaso-Occlusive Crises

   Sickle cell disease is a chronic disease characterized by multiple vaso-occlusive complications. The basic treatment for patients with a vaso-occlusive crisis (VOC) is based on adequate hydration, oxygen therapy and pain control. Loco Regional Anesthesia (LRA) is one of the major treatments in resuscitation anesthesia for both anesthesia and analgesia. LRA allows effective and almost immediate pain control by blocking nerve afferents in a given area. LRA could decrease the inflammatory response during crises and accelerate resolution of the crisis.

   Sites

   1

   Target

   34

   Study sites

   Pointe-à-Pitre, GP

   Sponsor: Centre Hospitalier Universitaire de la Guadeloupe

   View on ClinicalTrials.gov(opens in a new tab) Express interest
7. RecruitingPhase 4InterventionalNCT07488520

   Integrating Point of Care Testing (POCT) For Newborn Screening and Early Care for Sickle Cell Disease in Yopougon, Côte d'Ivoire

   Sickle Cell Disease (SCD)

   The goal of this clinical trial is to learn if a multifaceted intervention composed of Gazelle-Multispectral sickle cell disease (SCD) point of care testing (POCT) and early initiation of comprehensive SCD care in children with SCD disease aged 0 - 6 months can improve their clinical outcomes. The main questions it aims to answer are: * Does the intervention lower the number of SCD-related illnesses? * Does the intervention lower the illness incidence defined as seeking health care at any health facility - with or without treatment - for any episode related to a SCD related illness? * Does the intervention lower all-cause death rate (at the end of 1, 2, 3 and 3.5 years of follow-up)? Researchers will compare the multifaceted intervention results with those of historical data (based on erratic SCD testing and treatment) from the region. Participants will undergo a SCD screening test using the Gazelle Multispectral platform and if positive they will undergo confirmatory testing with HemoTypeSC™. Participants with SCD will receive early comprehensive clinical interventions i.e. standard administration of antibacterial and antimalarial prophylaxis, vaccinations for pneumococcal and Haemophilus influenzae type b (Hib), hydroxyurea, parental education about the need for regular and, if necessary, urgent medical care.

   Sites

   2

   Target

   120

   Study sites

   Abidjan, CI +1 more

   Sponsor: Swiss Tropical & Public Health Institute

   View on ClinicalTrials.gov(opens in a new tab) Express interest
8. RecruitingObservationalNCT07206095

   Integrative Diagnosis for SCD and Other RADs

   Sickle Cell Disease, Thalassaemia, Congenital Dyserythropoietic Anemia (CDA), Enzyme Disorder; Anemia, Spherocytosis, Hereditary, Stomatocytosis, Hemoglobin Disorder, Anemia Due to Membrane Defect, Rare Anemia Disorders

   INTEGRA aims at enabling personalized medicine for RHADs patients by the establishment of an integrative diagnostic approach based on deep phenotypic and genetic characterization through combining new generation methodologies.

   Sites

   9

   Target

   200

   Study sites

   Barcelona, ES +8 more

   Sponsor: Hospital Universitari Vall d'Hebron Research Institute

   View on ClinicalTrials.gov(opens in a new tab) Express interest