Clinical trials: Hemolytic Disease of the Newborn
Sickle Cell Disease
A Stratified Sickle Event Randomized Trial (ASSERT)
The purpose of this study is to compare the effects of ICA-17043 to placebo with or without hydroxyurea (an oral drug used for treatment of sickle cell disease) in patients with sickle cell disease who have had 2 or more acute…
- Trial name
- ASSERT (NCT00102791)
- Testing
- ICA-17043
- Where
- 49 sites in United States, Brazil and 4 more
- Volunteers
- 297 people
- Sponsor
- Icagen
Sickle Cell Disease
Safety and Efficacy of Sodium Nitrite in Sickle Cell Disease
This study will determine if administration of sodium nitrite is safe and can improve small vessel blood flow and tissue oxygenation when given as an additional treatment in patients with acute vaso-occlusive crisis (pain crisis)…
- Trial name
- NCT01033227
- Testing
- sodium nitrite injection, usp
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital Los Angeles
Thalassemia (Transfusion Delendent)
Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia
The CENTAurus trial is a prospective clinical study designed to address systematically some of the relevant endocrine complications in an iron overloaded thalassemic population, primary objective being the assessment of the…
- Trial name
- CENTAurus (NCT02069886)
- Testing
- deferasirox
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
- Trial name
- NCT05632354
- Testing
- Osivelotor
- Where
- 9 sites in United States, Nigeria
- Volunteers
- 47 people
- Sponsor
- Pfizer
Other Sickle-cell Disorders With Crisis, Unspecified
Quality of Life Study for Sickle Cell Patients Treated With Jobelyn (Sorghum Bicolor Extract)
The purpose of this study is to determine the antioxidant effect of prolonged use of sorghum bicolor (jobelyn) to increase the level of plasma superoxide dismutase and glutathione reductase in patients with sickle cell disease…
- Trial name
- NCT01704794
- Testing
- Folic Acid + Paludrine + Jobelyn (500mg), Folic Acid + Paludrine + Jobelyn (250mg), Folic Acid + Paludrine + Jobelyn (2mg)
- Where
- Nigeria
- Volunteers
- 96 people
- Sponsor
- Lagos State University
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease.
- Trial name
- HOPE Kids 2 (NCT04218084)
- Testing
- Voxelotor
- Where
- 35 sites in United States, Egypt and 7 more
- Volunteers
- 236 people
- Sponsor
- Pfizer
Transfusion-dependent Beta-Thalassemia
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem…
- Trial name
- NCT05860595
- Testing
- KL003 cell injection Drug Product
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Vaso-Occlusive Pain Episode in Sickle Cell Disease, Sickle Cell Disease (SCD)
Low Dose Bolus Ketamine For Use In Sickle Cell Pain Crisis
The goal of this study is to learn if Ketamine works more efficiently, as compared to Opioids, for Sickle Cell Pain The main questions it aims to answer are: Does Ketamine lower the number of times participants need to be…
- Trial name
- NCT07682662
- Testing
- Standardized Low-dose bolus IV Ketamine within 30 minutes of arrival and every 1 hour as needed for max of 3 doses., Standard Care (in control arm)
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- University of Mississippi Medical Center
Myelodysplastic Syndromes, Beta-thalassemia
Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL.
- Trial name
- NCT04973280
- Where
- 4 sites in Austria, Belgium and 2 more
- Volunteers
- 187 people, healthy volunteers welcome
- Sponsor
- Celgene
Fetal Hemoglobin, Thalassemia Major
Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing…
- Conditions
- Fetal Hemoglobin, Thalassemia Major, Genetic Disease
- Trial name
- BTM (NCT06490627)
- Testing
- Thalidomide
- Where
- Pakistan
- Volunteers
- 54 people
- Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Sickle Cell Disease
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).
- Trial name
- PRECISE (NCT06923111)
- Testing
- Xromi
- Where
- 12 sites in Germany, United Kingdom
- Volunteers
- 180 people
- Sponsor
- Nova Laboratories Limited
Sickle Cell Disease
Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials
- Trial name
- 034OLE (NCT03573882)
- Testing
- Voxelotor
- Where
- 60 sites in United States, Canada and 9 more
- Volunteers
- 179 people
- Sponsor
- Pfizer
Diabetes Mellitus, Heart Disease
Deferoxamine for the Treatment of Hemochromatosis
When patients receive repeated blood transfusions the level of iron in the patient s blood can rise. When iron is processed in the body a protein known as hemosiderin can begin collecting in the organs.
- Conditions
- Diabetes Mellitus, Heart Disease, Hemochromatosis, Thalassemia
- Trial name
- NCT00001203
- Where
- United States
- Volunteers
- 151 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Anemia, Sickle Cell Thalassemia
Clinical and Laboratory Characteristics of Sickle Cell Anemia Patients Admitted With Fever
This study will summarized the clinical and laboratory data and the outcome of all the patients suffering from Sickle Cell Anemia (Including Sickle cell thalassemia) admitted to the pediatric ward.
- Trial name
- NCT00736060
- Testing
- Observational
- Where
- Israel
- Volunteers
- 60 people
- Sponsor
- HaEmek Medical Center, Israel
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with…
- Conditions
- Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia
- Trial name
- NCT04965597
- Testing
- Treosulfan, Fludarabine Phosphate, Tacrolimus
- Where
- 24 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
Vaso-occlusive Crisis, Sickle Cell Disease
Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia
The aim of the present study is comparing the effectiveness of different treatment regimens for investigating the therapeutic potential for each one in management of Vaso-occlusive pain in pediatric sickle cell disease.
- Conditions
- Vaso-occlusive Crisis, Sickle Cell Disease, Sickle Cell Anemia in Children
- Trial name
- NCT04301336
- Testing
- Omega 3, Vit D, Zinc sulfate
- Where
- 4 sites in Egypt, Saudi Arabia
- Volunteers
- 350 people
- Sponsor
- Beni-Suef University
Hemolytic Disease of the Newborn, Fetal Anemia
Neonatal Outcomes on Direct Fetal Intravenous Immunoglobulin During Intrauterine Transfusion for Rhesus Isoimmunization
The goal of this clinical trial is to learn if giving intravenous immunoglobulin directly to the fetus during intrauterine transfusion can improve newborn outcomes in pregnancies affected by rhesus isoimmunization.
- Conditions
- Hemolytic Disease of the Newborn, Fetal Anemia, Rhesus Isoimmunization, Erythroblastosis Fetalis Due to RH Antibodies
- Trial name
- NCT07885592
- Testing
- Intrauterine Transfusion, Intravenous immunoglobulin (IVIG)
- Where
- India
- Volunteers
- 30 people
- Sponsor
- Post Graduate Institute of Medical Education and Research, Chandigarh
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Disease
A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke
The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke.
- Trial name
- HIBISCUS 3 (NCT05953584)
- Testing
- Etavopivat, Hydroxyurea
- Where
- 9 sites in India, Nigeria and 1 more
- Volunteers
- 27 people
- Sponsor
- Forma Therapeutics, Inc.
Priapism, Anemia, Sickle Cell
Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence
Priapism, a prolonged erection of the penis, is a medical issue that often affects men with sickle cell disease. The purpose of this study is to collect demographic and clinical information on priapism by interviewing men with…
- Trial name
- NCT00300235
- Where
- 27 sites in United States
- Volunteers
- 1,464 people
- Sponsor
- University of Texas Southwestern Medical Center