Clinical trials: Neonatal Anemia
Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies
RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions. PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic…
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01273766
- Testing
- deferasirox, laboratory biomarker analysis, enzyme-linked immunosorbent assay
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Wake Forest University Health Sciences
Sickle Cell Disease
Self-Management for Youth and Families Living With SCD - SMYLS
The purpose of this proposal is to integrate family-centered self-management strategies with mobile health (mHealth) technology to improve reach, self-management behaviors, and child and caregiver physical and psychosocial…
- Trial name
- NCT03585543
- Testing
- Voice Crisis Alert V2
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Medical University of South Carolina
Sickle Cell Disease
Improving Self-Management in Adolescents With Sickle Cell Disease
The objective of this study is to determine the feasibility and acceptability of SCThrive, a an innovative, technology-enhanced, group self-management intervention that uses a mixed in-person and online format and supported by a…
- Trial name
- SCThrive (NCT02851615)
- Testing
- SCThrive Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Adenosine 2A Agonist Lexiscan in Children and Adults With Sickle Cell Disease
Sickle cell disease (SCD) is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape.
- Trial name
- NCT01085201
- Testing
- Lexiscan
- Where
- 7 sites in United States
- Volunteers
- 39 people
- Sponsor
- Dana-Farber Cancer Institute
Sickle Cell Disease
High Dose Vitamin D for Sickle Cell Disease
Vitamin D deficiency (VDD) is very common among African American adolescents and adults in the US, ten times higher than is seen in Caucasians. VDD is also quite common in sickle cell disease (SCD).
- Trial name
- SCD-VitD (NCT01331148)
- Testing
- Vitamin D
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Wake Forest University Health Sciences
Sickle Cell Disease
A Study of SHP655 (rADAMTS13) in Sickle Cell Disease
TAK-755 (previously known as SHP655) is a medicine used to treat sickle cell disease (SCD). The main aim of the study is to measure the safety and tolerability of TAK-755 in SCD participants.
- Trial name
- RAISE (NCT03997760)
- Testing
- TAK-755
- Where
- 14 sites in United States
- Volunteers
- 19 people
- Sponsor
- Shire
Sickle Cell Anemia
Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol
The BABY HUG Treatment Study was designed to see if treatment with the drug hydroxyurea (also called HU) in children with sickle cell disease could prevent organ damage, especially in the spleen and kidneys.
- Trial name
- BABY HUG (NCT01783990)
- Testing
- Hydroxyurea
- Where
- 14 sites in United States
- Volunteers
- 150 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Anemia, Diamond-Blackfan
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Background: Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work.
- Trial name
- NCT04269889
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Leg Ulcers
Resolution of Sickle Cell Leg Ulcers With Voxelotor
This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease.
- Trial name
- RESOLVE (NCT05561140)
- Testing
- Voxelotor Oral Tablet
- Where
- 23 sites in Brazil, Kenya and 1 more
- Volunteers
- 88 people
- Sponsor
- Pfizer
Sickle Cell Disease
Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease
This is a randomized, double-blind, placebo-controlled study in sickle cell disease participants with a history of Vaso-occlusive Crises (VOCs).
- Trial name
- NCT05098028
- Testing
- Low Dose Rifaximin ER, Low Dose Rifaximin DER, High Dose Rifaximin ER
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 44 people
- Sponsor
- Bausch Health Americas, Inc.
Sickle Cell Crisis, Acute Pain
Low-dose Ketamine vs Morphine for Vaso-occlusive Crisis in Sicklers
This clinical trial will inform of the role of Low dose ketamine in the acute treatment of severe painful sickle cell crisis in children in a day-case sickle cell centre.
- Trial name
- KEM-VOC (NCT02434939)
- Testing
- Low dose ketamine, Morphine
- Where
- Uganda
- Volunteers
- 240 people
- Sponsor
- Makerere University
Thalassemia
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
Study AG348-C-010 is a multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of treatment with AG-348 in adult participants with non-transfusion-dependent thalassemia (NTDT).
- Trial name
- NCT03692052
- Testing
- AG-348
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 20 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Beta Thalassemia Intermedia
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.
- Trial name
- NCT04059406
- Testing
- sapablursen
- Where
- 19 sites in Australia, Greece and 3 more
- Volunteers
- 29 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Severe Thalassemia
Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks…
- Trial name
- URTH (NCT01005576)
- Testing
- Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
- Where
- 18 sites in United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease
Intravenous Magnesium for Sickle Cell Vasoocclusive Crisis
The purpose of this study is to determine the safety and efficacy of intravenous magnesium in shortening the duration of a pain crisis and to determine the health-related quality of life and short term outcomes of children…
- Trial name
- MAGiC (NCT01197417)
- Testing
- Intravenous Magnesium Sulfate
- Where
- 8 sites in United States
- Volunteers
- 208 people
- Sponsor
- Medical College of Wisconsin
Non-transfusion Dependent Thalassemia
Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study
CICL670A2209: This study will evaluate the safety and efficacy of deferasirox in non-transfusion dependent thalassemia patients with iron overload.
- Trial name
- THALASSA (NCT00873041)
- Testing
- deferasirox
- Where
- 21 sites in United States, Greece and 7 more
- Volunteers
- 166 people
- Sponsor
- Novartis Pharmaceuticals
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
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
Sickle Cell Disease, Vaso-occlusive Pain Episode
Arginine Therapy for Sickle Cell Disease Pain
The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores…
- Trial name
- NCT02536170
- Testing
- L-arginine, L-arginine Loading Dose
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Emory University
Sickle Cell Disease
Simvastatin (Zocor) Therapy in Sickle Cell Disease
Recent clinical and experimental data indicate that statins have effects beyond cholesterol lowering that may be beneficial in sickle cell disease by protecting the vascular endothelium.
- Trial name
- NCT00508027
- Testing
- Simvastatin
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland