Clinical trials: Sickle Cell Disorders
Healthy, Sickle Cell Anemia
Dose Escalation Study of PF-07209326 in Healthy Participants and Participants With Sickle Cell Disease
This Phase 1 first-in-human, first-in-patient, single ascending dose and multiple dose study will be a randomized, double-blind, placebo-controlled investigation of the safety, tolerability, and pharmacokinetics of PF-07209326 in…
- Trial name
- NCT04255875
- Testing
- PF-07209326
- Where
- 16 sites in United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Pfizer
Sickle Cell Disease, Kidney Injury
Acute Kidney Injury in Patients With Sickle Cell Disease
Patients with sickle cell disease may be at risk for acute kidney injury (AKI)during sickle cell crisis (pain or acute chest syndrome).
- Conditions
- Sickle Cell Disease, Kidney Injury, Kidney Diseases, Kidney Disease, Chronic
- Trial name
- NCT03105271
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Sickle Cell Disease
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD
- Trial name
- BEACON (NCT05456880)
- Testing
- BEAM-101
- Where
- 19 sites in United States
- Volunteers
- 15 people
- Sponsor
- Beam Therapeutics Inc.
Sickle Cell Disease
Sickle-cell Disease Registry of the GPOH
Sickle cell disease is one of the most common hereditary diseases. Most severe complications can be avoided if the disease is detected early and treated appropriately.
- Trial name
- SichReg (NCT03327428)
- Where
- Germany
- Volunteers
- 1,000 people
- Sponsor
- University Hospital Heidelberg
Sickle Cell Disease, Sickle Cell Anemia
Alendronate for Osteonecrosis in Adults With Sickle Cell Disease
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Osteonecrosis, Ischemic Necrosis
- Trial name
- NCT06016634
- Testing
- Alendronate Sodium
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, Davis
Sickle Cell Disease
A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe 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…
- Trial name
- NCT03653247
- Testing
- Plerixafor, Busulfan, BIVV003
- Where
- 5 sites in United States
- Volunteers
- 7 people
- Sponsor
- Sangamo Therapeutics
Sickle Cell Anemia, Beta-thalassemia Major
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
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…
- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Trial name
- NCT03653338
- Testing
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Paul Szabolcs
Healthy Adult Subjects, Sickle Cell Disease
Safety, Tolerability and Pharmacokinetics of FTX-6058
This is a study to evaluate the safety, tolerability and pharmacokinetics of FTX-6058 in healthy adult subjects and adult subjects with sickle cell disease (SCD).
- Trial name
- NCT04586985
- Testing
- FTX-6058 - Two Dosing Periods, FTX-6058 / Midazolam Syrup
- Where
- 2 sites in United States
- Volunteers
- 109 people, healthy volunteers welcome
- Sponsor
- Fulcrum Therapeutics
Pulmonary Hypertension, Sickle Cell Anemia
A Study of Patients Having Pulmonary Hypertension Associated With Sickle Cell Disease and Completing an ASSET Study
This study will assess the safety and efficacy of bosentan therapy (in a study known as ASSET) for patients who have high blood pressure in the lungs associated with sickle cell disease.
- Trial name
- ASSET-3 (NCT00360087)
- Testing
- Bosentan
- Volunteers
- 236 people
- Sponsor
- Actelion
Hematologic Malignancies, Nonmalignant Diseases
Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)
Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609814
- Testing
- Clofarabine, Fludarabine Injection
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, San Francisco
Sickle Cell Disease
Sleep Study in Adult Patients With Major Sickle Cell Disease With Paroxysmal Nocturnal Events
Hypothesis is that the occurrence of nocturnal Vaso-Occlusive Crisis (VOC) and priapism in adults might be related to episodes of nocturnal desaturation secondary to a sleep apnea syndrome.
- Trial name
- DREPASOM (NCT02539771)
- Testing
- Polysomnography and blood sample
- Where
- France
- Volunteers
- 114 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Vaso-occlusive Crisis
Study of Erythrocyte Parameters and Hypercoagulability in Sickle Cell Disease (SCD-TGA)
Sickle cell disease (SCD) is an inherited haemoglobinopathy disorder caused by mutations in HBB gene with amino-acid substitution on β globin chain.
- Trial name
- SCD-TGA (NCT05376046)
- Testing
- Erythrocytic parameters and thrombin generation assay measurement
- Where
- France
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- BILLOIR
Sickle Cell Anaemia
Prevention Of Morbidity In Sickle Cell Disease Pilot Phase
The hypothesis is that in sickle cell anaemia, nocturnal oxyhaemoglobin desaturation, is associated with low processing speed index, and this morbidity can be reduced with overnight auto Continuous Positive Airways Pressure…
- Trial name
- NCT00415727
- Testing
- auto Continuous Positive Airways Pressure (CPAP) with oxygen supplementation
- Where
- 2 sites in United Kingdom
- Volunteers
- 22 people
- Sponsor
- Institute of Child Health
Sickle Cell Disease
Contraception in Women With Sickle Cell Disease
Sickle cell anemia is a homozygous genetic disease with high prevalence in Brazil. There are changes in conformation and physicochemical properties of red cells that generate varied clinical manifestations among which is chronic…
- Trial name
- NCT02594462
- Testing
- etonogestrel-releasing implant contraceptive
- Where
- 2 sites in Brazil
- Volunteers
- 29 people
- Sponsor
- University of Sao Paulo
Sickle Cell Anemia, Thalassemia
L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia. as evaluated by the number of occurrences of sickle cell crises.
- Trial name
- NCT00125788
- Testing
- L-glutamine
- Where
- 5 sites in United States
- Volunteers
- 70 people
- Sponsor
- Emmaus Medical, Inc.
Sickle Cell Leg Ulcer
Test of the Safety, Effectiveness, & Acceptability of An Improvised Dressing for Sickle Cell Leg Ulcers in the Tropics
One in 300 Jamaicans have HbSS sickle cell disease, and of these, up to 70% will suffer from sickle cell leg ulcers (SCLUs). Of these, 24% will have a chronic SCLU (one lasting longer than 6 months).
- Trial name
- SCLUJamaica (NCT04479618)
- Testing
- Usual Practice, Improvised dressing, Advanced dressing
- Where
- Jamaica
- Volunteers
- 48 people
- Sponsor
- Benskin, Linda, PhD, RN, SRN (Ghana), CWCN, CWS, DAPWCA
Sickle Cell Disease
Topical Sodium Nitrite in Sickle Cell Disease and Leg Ulcers
The investigators are conducting a Phase II prospective and placebo controlled study of a topical cream containing sodium nitrite compared to the current standard of care.
- Trial name
- NCT02863068
- Testing
- Topical Sodium Nitrite
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- caterina P minniti
Sickle Cell Disease and Pregnancy
Multi Disciplinary Sickle Cell Disease Obstetrics Care Program in Ghana
The goal of this observational study is to determine the feasibility and effectiveness of initiating a multidisciplinary sickle cell disease (SCD) obstetrics program for women with SCD in a non-academic hospital.
- Trial name
- SCOB III (NCT07488091)
- Testing
- Multidisciplinary sickle cell disease (SCD) obstetrics program for women with SCD in a non-academic hospital.
- Where
- Ghana
- Volunteers
- 198 people
- Sponsor
- University of Ghana Medical School
Sickle Cell Anemia in Children, Malaria
Dihydroartemisinin-Piperaquine or Sulphadoxine-Pyrimethamine for the Chemoprevention of Malaria in Sickle Cell Anaemia
Sickle Cell Anaemia (SCA) is an inherited disease that makes the body produce red blood cells with abnormal sickle-shaped cells. The sickle-shaped cells are rigid, not flexible and break up easily resulting in anaemia.
- Trial name
- CHEMCHA (NCT04844099)
- Testing
- Dihydroartemisinin Piperaquine
- Where
- 3 sites in Malawi, Uganda
- Volunteers
- 723 people
- Sponsor
- Liverpool School of Tropical Medicine
Sickle Cell Disease
Added Value of Speckle Tracking in the Evaluation of Patients With Sickle Cell Disease
Sickle Cell Disease is a serious disease that is life-threatening for patients being homozygous for the SS form or heterozygous for the SC or βthal forms.
- Trial name
- NCT02394431
- Testing
- Cardiac echography, Biological parameters, Clinical parameters
- Where
- Belgium
- Volunteers
- 62 people
- Sponsor
- Brugmann University Hospital
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