Clinical trials: Anemia, Neonatal
Sickle Cell Disease, Vaso-occlusive Crisis
Treatment of Sickle Cell Patients Hospitalized in Pain Crisis With Prophylactic Dose Low-molecular-weight Heparin (LMWH) Versus Placebo
Sickle cell disease (SCD) is one of the most common inherited diseases worldwide and exhibits highest frequency in people of African descent.
- Trial name
- NCT01419977
- Testing
- Dalteparin
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Duke University
Thalassemia
Zinc & Bone Health in Thalassemia: The Think Zinc Study
The purpose of this study is to test whether zinc can improve bone health in young patients with thalassemia.
- Trial name
- ThinkZn (NCT00459732)
- Testing
- Zinc
- Where
- 3 sites in United States
- Volunteers
- 45 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
A Relative Bioavailability Study of a Prasugrel Orally Disintegrating Tablet
This study compares the clinical tablet formulation of prasugrel taken orally with an orally disintegrating tablet (ODT) taken orally.
- Trial name
- NCT01430091
- Testing
- Prasugrel (clinical formulation), Prasugrel (Orally Disintegrating Tablet [ODT])
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Eli Lilly and Company
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia in Remission
Vorinostat, Tacrolimus, and Methotrexate in Preventing GVHD After Stem Cell Transplant in Patients With Hematological Malignancies
This pilot phase II trial studies how well giving vorinostat, tacrolimus, and methotrexate works in preventing graft-versus-host disease (GVHD) after stem cell transplant in patients with hematological malignancies.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT01789255
- Testing
- vorinostat, tacrolimus, cyclosporine
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease
Building Adaptive Coping and Knowledge to Improve Daily Life
The purpose of this study is to find out how teenagers with chronic pain and sickle cell disease respond to a new training program called Back2Life and get their feedback about how to modify the program to best fit their needs.
- Trial name
- Back2Life (NCT04602728)
- Testing
- Back2Life
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Emory University
Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Busulfan, Etoposide, and Intensity-Modulated Radiation Therapy Followed By Donor Stem Cell Transplant in Treating Patients With Advanced Myeloid Cancer
RATIONALE: Giving chemotherapy drugs, such as busulfan and etoposide, and intensity-modulated radiation therapy before a donor stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT00540995
- Testing
- busulfan, etoposide, intensity-modulated radiation therapy
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- City of Hope Medical Center
Anemia, Sickle Cell
Nitric Oxide Inhalation to Treat Sickle Cell Pain Crises
This study will examine whether nitric oxide (NO) gas can reduce the time it takes for pain to go away in patients who are in sickle cell crisis. NO is important in regulating blood vessel dilation, and consequently, blood flow.
- Trial name
- NCT00094887
- Testing
- Nitric Oxide
- Where
- 11 sites in United States
- Volunteers
- 150 people
- Sponsor
- Mallinckrodt
Sickle Cell Disease & Thalassemia
Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
- Trial name
- NCT01590628
- Testing
- NiCord
- Where
- 3 sites in United States
- Volunteers
- 16 people
- Sponsor
- Gamida Cell ltd
Transfusional Iron Overload, Transfusional Hemosiderosis
Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
This is a clinical research study in patients who have iron overload in the heart due to chronic blood transfusions. The study will have 2 treatment groups and will compare the safety and efficacy of chelation therapy with a…
- Trial name
- CORDELIA (NCT00600938)
- Testing
- Core Study: Deferasirox, Core Study: Deferoxamine, Extension: deferoxamine to deferasirox
- Where
- 21 sites in Canada, China and 9 more
- Volunteers
- 197 people
- Sponsor
- Novartis Pharmaceuticals
Autoimmune Pancytopenia, Autoimmune Lymphoproliferative Syndrome (ALPS)
Sirolimus for Autoimmune Disease of Blood Cells
Treatment for patients with autoimmune destruction of blood cells is poor. The part of the body that fights infections is called the immune system and white blood cells (WBCs) are part of the immune system.
- Conditions
- Autoimmune Pancytopenia, Autoimmune Lymphoproliferative Syndrome (ALPS), Evans Syndrome, Idiopathic Thrombocytopenic Purpura
- Trial name
- NCT00392951
- Testing
- sirolimus
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Children's Hospital of Philadelphia
Sickle Cell Disease
Families Taking Control (FTC): Family-based Problem-solving Intervention for Children With Sickle Cell Disease
This study aims to develop an effective, brief, family-based intervention targeting quality of life and school functioning for youth with sickle cell disease.
- Trial name
- FTC (NCT02273310)
- Testing
- Problem-Solving Skills Training for Disease Management
- Volunteers
- 83 people
- Sponsor
- University of Pennsylvania
Malaria, Vivax
A Pharmacokinetics, Safety and Efficacy Study of Tafenoquine (TQ) in Pediatric Subjects With Plasmodium Vivax (P. Vivax) Malaria
This is a prospective, open-label, multicenter, non-comparative, single arm study of pediatric subjects with Plasmodium vivax (P. vivax) malaria, aged 6 months to <16 years of age. A total of 60 subjects will be enrolled.
- Trial name
- NCT02563496
- Testing
- Tafenoquine, Chloroquine
- Where
- 4 sites in Colombia, Vietnam
- Volunteers
- 60 people
- Sponsor
- GlaxoSmithKline
Sickle Cell Anemia
Sparing Conversion to Abnormal TCD (Transcranial Doppler) Elevation (SCATE)
The primary goal of the Phase III SCATE trial is to compare 30 months of alternative therapy (hydroxyurea) to standard care (observation) in children with sickle cell anemia and conditional (170 - 199cm/sec) Transcranial Doppler…
- Trial name
- SCATE (NCT01531387)
- Testing
- Hydroxyurea
- Where
- 3 sites in United States, Brazil and 1 more
- Volunteers
- 38 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Cyanotic Congenital Heart Disease, Anemia
Erythropoietin to Prevent Unnecessary Transfusions In Patients With Cyanotic CHD - A Prospective Control Trial
Cyanotic congenital cardiac patients require higher hemoglobin concentrations (red blood cell levels) for optimal oxygen delivery to the body.
- Conditions
- Cyanotic Congenital Heart Disease, Anemia, Cyanosis, Congenital Heart Disease
- Trial name
- NCT02564796
- Testing
- Epoetin Alfa and Iron, Iron
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of California, San Diego
Sickle Cell Anemia
Haploidentical PBMC Transplant for Severe Congenital Anemias
Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias.
- Trial name
- NCT00977691
- Testing
- PBSC Transplant, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Diamond Blackfan Anemia, Blackfan Diamond Syndrome
Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions.
- Conditions
- Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia
- Trial name
- LeucineDBA (NCT01362595)
- Testing
- leucine
- Where
- 11 sites in United States
- Volunteers
- 55 people
- Sponsor
- Northwell Health
Sickle Cell Disease
Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
Many youth with chronic disease have difficulty taking medication every day and therefore do not receive full benefit from treatment.
- Trial name
- NCT03462511
- Testing
- HABIT Intervention, Standard of Care, Education materials
- Where
- 4 sites in United States
- Volunteers
- 50 people
- Sponsor
- Columbia University
Sickle Cell Disease
A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS)…
- Trial name
- NCT04983264
- Testing
- GBT021601
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Pfizer
G6PD Deficiency
Evaluation of a Diagnostic to Identify Glucose-6-phosphate Dehydrogenase (G6PD) Deficiency in Brazil
Th objectives of this study are: To determine the performance of G6PD tests in detecting G6PD activity and hemoglobin (Hb) compared to a reference assay To assess the comprehension of the G6PD test packaging and labelling among…
- Trial name
- NCT04033640
- Testing
- SD Biosensor STANDARD G6PD Test, Pointe Scientific Test Kit, HemoCue System
- Where
- 2 sites in Brazil
- Volunteers
- 1,754 people, healthy volunteers welcome
- Sponsor
- PATH
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated