Clinical trials: Sickle Cell Disorder
Sickle Cell Disease, Vaso-occlusive Crisis
Adaptive Platform Trial for Pain Management in Sickle Cell Vaso-Occlusive Crisis
Sickle cell disease (SCD) is a severe hemoglobinopathy, characterized by recurrent vaso-occlusive crises (VOC) causing intense pain and frequent hospitalizations in adult patients.
- Trial name
- PAMAVOC (NCT07826988)
- Testing
- Group 02 Paracetamol + Morphine + Ketamine, Group 01 Control: Paracetamol + Morphine, Group 03 : Paracetamol + Morphine + Nefopam
- Volunteers
- 400 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Drepanocytosis, Chronic Neurological Deficiency
Transfusion Exchanges and Cognition in Sickle Cell Disease
DREPA-COG is an observational study evaluating the effect of exchange transfusions on cognitive function in adults with severe sickle cell disease (SS or Sβ0).
- Trial name
- DREPA-COG (NCT07168447)
- Where
- 6 sites in France
- Volunteers
- 85 people
- Sponsor
- Centre Hospitalier St Anne
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Sickle Cell Disease
Evaluation of Knowledge Among Adolescents With Sickle Cell Disease.
Evaluation of knowledge about contraception in sickle cell adolescents.
- Trial name
- SEXODREP (NCT03235011)
- Where
- France
- Volunteers
- 99 people
- Sponsor
- Adele CARLIER-GONOD
Sickle Cell Disease
Zinc for Infection Prevention in Sickle Cell Anemia (ZIPS)
A randomized double-blinded placebo-controlled trial of zinc to reduce the incidence of severe or invasive infections in Ugandan children with sickle cell anemia (SCA).
- Trial name
- ZIPS (NCT03528434)
- Testing
- Zinc
- Where
- Uganda
- Volunteers
- 250 people
- Sponsor
- Indiana University
Sickle Cell Disease
Ketamine as an Adjuvant Therapy for Acute Vaso Occlusive Crisis in Pediatric Patients With Sickle Cell Disease
The primary objective of the proposed study is to determine the potential role of Ketamine as an analgesic agent in pediatric sickle cell disease patients with refractory symptoms in acute (VOC).
- Trial name
- KSickle (NCT02801292)
- Testing
- Ketamine
- Volunteers
- 20 people
- Sponsor
- Augusta University
Pain, Sickle Cell Disease
Intranasal Fentanyl Versus Intravenous Morphine in the Treatment of Severe Painful Sickle Cell Crises in Children
Sickle cell anaemia is an inherited blood disorder which results in abnormal sickle shaped red blood cells which do not fit well through small blood vessels.
- Trial name
- NCT03682211
- Testing
- Fentanyl Citrate, Morphine sulphate
- Where
- Ireland
- Volunteers
- 31 people
- Sponsor
- University College Dublin
Sickle Cell Disease
Evaluating Thromboelastography (TEG) and ETP in Sickle Adults
The primary aim of this study is to investigate the reported enhanced coagulation status (prothrombotic status) in patients with sickle cell disease using 2 laboratory tests; thromboelastography (TEG) and Endogenous Thrombin…
- Trial name
- NCT02770534
- Testing
- blood test
- Where
- United Kingdom
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Pulmonary Hypertension
Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients Diagnosed With Pulmonary Hypertension (PH)
The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in Sickle Cell Disease (SCD) patients diagnosed with Pulmonary Hypertension.
- Trial name
- ASSET-2 (NCT00313196)
- Testing
- bosentan
- Volunteers
- 12 people
- Sponsor
- Actelion
Sickle Cell Anemia, Sickle Cell Disease
Sickle Cell Disease - Stroke Prevention in Nigeria Trial
Given large absolute numbers of individuals with sickle cell disease in Nigeria, hydroxyurea therapy for all individuals with sickle cell disease may not be initially feasible; however, a targeted strategy of hydroxyurea use for…
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Stroke
- Trial name
- SPIN (NCT01801423)
- Testing
- Hydroxyurea
- Where
- Nigeria
- Volunteers
- 29 people
- Sponsor
- Vanderbilt University Medical Center
Bone Marrow Failure Syndromes, Erythrocyte Disorder
Investigation of the Genetics of Hematologic Diseases
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases.
- Conditions
- Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis
- Trial name
- NCT02720679
- Where
- United States
- Volunteers
- 1,716 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Evaluation of the Prevalence of Pulmonary Hypertension in Adult Patients With Sickle Cell Disease
Recent data show that pulmonary hypertension (PH), defined by a tricuspid regurgitation jet (TRJ) velocity > or equal at 2.5m/s on Doppler echocardiography, is present in about 30% of adults with sickle cell disease (SCD) and is…
- Trial name
- ETENDARD (NCT00434902)
- Where
- France
- Volunteers
- 700 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Hemolysis Related Complications in SCD. A Phase II Study With Voxelotor
Intro: Sickle cell disease is a genetic disorder caused by a mutation of the β hemoglobin called HbS, which causes red blood cell (RBC) abnormalities responsible for hemolysis, mainly intravascular, leading to chronic anemia.
- Trial name
- HEMOPROVE (NCT05199766)
- Testing
- Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell disease
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Evaluation of an Information Management and Communication System for Population-wide Point-of-Care Infant Sickle Cell Disease Screening
A cluster randomized trial (CRT) of the novel sickle cell disease (SCD), M-health based SCD Information Management and Communication system SIMCS vs routine new born screening strategy in health care centres in Uganda to evaluate…
- Trial name
- SMICS (NCT07610239)
- Testing
- Sickle Cell Disease screening information management and communication system
- Where
- Uganda
- Volunteers
- 24,000 people, healthy volunteers welcome
- Sponsor
- Makerere University
Cystic Fibrosis, Hemophilia A
Medication Adherence and Non-adherence in Adults With Rare Disease
The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary…
- Conditions
- Cystic Fibrosis, Hemophilia A, Hemophilia B, Idiopathic Pulmonary Fibrosis
- Trial name
- NCT04541875
- Testing
- MAR-Scale
- Volunteers
- healthy volunteers welcome
- Sponsor
- Xperiome
Sickle Cell Disease
Prevalence of Osteoporosis in Sickle Cell Disease
Sickle cell disease is the most common single-gene disease in the world. Its prevalence is increasing in France, with patients' life expectancy increasing into developed countries.
- Trial name
- DREPAN'OS (NCT04299594)
- Testing
- Questionnaire
- Where
- France
- Volunteers
- 142 people
- Sponsor
- Hospices Civils de Lyon
Sickle Cell Disease, Hemolytic Anemia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Background: Sickle cell disease (SCD) is a disorder that causes episodes of acute pain and progressive organ damage. Ways to manage SCD have evolved slowly. Treatments do not always work.
- Trial name
- NCT04610866
- Testing
- Mitapivat
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Priapism, Sickle Cell Disease
Androgen Regulation of Priapism in Sickle Cell Disease
It is believed that when androgen (testosterone) levels are below normal there is a disturbance of normal bodily functioning that is associated with priapism in some men.
- Conditions
- Priapism, Sickle Cell Disease, Hypogonadism
- Trial name
- NCT01940718
- Testing
- Transdermal Androgel
- Where
- United States
- Sponsor
- Johns Hopkins University
Moya Moya Disease
Moya Moya Syndrome With or Withtout Sickle Cell Disease
Moya Moya disease or syndrome ar characterized by a progressive or occlusion of the intracranial carotid arteries and their mainproximal branches, followed by the development of fragile neovessels at the base of the skull, leding…
- Trial name
- BMM-ScD (NCT05050344)
- Where
- France
- Volunteers
- 50 people
- Sponsor
- University Hospital, Montpellier
Sickle Cell Disease
L-Arginine and Sickle Cell Disease
One of the main problems in sickle cell disease is the decreased bioavailability of nitric oxide and arginine. This study was designed to assess if treating sickle cell disease patients with L-arginine would improve pulmonary…
- Trial name
- NCT01142219
- Testing
- L-arginine
- Where
- Brazil
- Volunteers
- 40 people
- Sponsor
- Hospital de Clinicas de Porto Alegre
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.