Clinical trials: Hemoglobinopathies
Sickle Cell Disease
Preventing Acute Chest Syndrome by Transfusion Feasibility Study
Acute chest syndrome (ACS) is similar to severe pneumonia and is a common cause of hospitalizations for people with sickle cell disease (SCD). Blood transfusions are one treatment option for ACS.
- Trial name
- PROACTIVE (NCT00951808)
- Testing
- Single blood transfusion, Standard care
- Where
- 24 sites in United States
- Volunteers
- 237 people
- Sponsor
- Carelon Research
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype
This is a single-arm, multi-site, single-dose, Phase 3 study in 23 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0…
- Trial name
- NCT02906202
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 24 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease
Voxelotor Neurocognitive Function Study
This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition…
- Trial name
- NCT05228834
- Testing
- Voxelotor Only Product in Oral Dose Form
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Pfizer
Sickle Cell Disease
Comparison of Patient Centered Outcomes for People With Sickle Cell Disease in the Acute Care Setting
The Emergency Department has been the standard location where patients with Sickle Cell Disease (SCD) go to seek care for the treatment of acute painful events.
- Trial name
- ESCAPED (NCT02411396)
- Where
- 3 sites in United States
- Volunteers
- 483 people
- Sponsor
- Johns Hopkins University
Sickle Cell Disease
Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD).
- Trial name
- NCT03263559
- Testing
- Haploidentical Bone Marrow Transplantation, Hydroxyurea, Rabbit-ATG
- Where
- 32 sites in United States
- Volunteers
- 95 people
- Sponsor
- Medical College of Wisconsin
Sickle Cell Disease
Aspirin Prophylaxis in Sickle Cell Disease
Neurologic complications secondary to cerebrovascular damage are prevalent in children with sickle cell disease. These patients experience both clinically overt cerebrovascular accidents and "silent infarctions" demonstrated by…
- Trial name
- START (NCT00178464)
- Testing
- aspirin
- Volunteers
- 11 people
- Sponsor
- University of Rochester
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
Sickle Cell Anemia, Sickle Cell Disease
Novel Use Of Hydroxyurea in an African Region With Malaria
Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in…
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Malaria
- Trial name
- NOHARM (NCT01976416)
- Testing
- Hydroxyurea
- Where
- Uganda
- Volunteers
- 208 people
- Sponsor
- Indiana University
Sickle Cell Disease (SCD)
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis…
- Trial name
- STAND (NCT03814746)
- Testing
- Crizanlizumab (SEG101)
- Where
- 58 sites in United States, Belgium and 19 more
- Volunteers
- 253 people
- Sponsor
- Novartis Pharmaceuticals
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Patients with medical conditions requiring allogeneic hematopoietic cell transplantation (allo-HCT) are at risk of developing a condition called graft versus host disease (GvHD) which carries a high morbidity and mortality.
- Conditions
- Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia
- Trial name
- HAPLOTAB (NCT05236764)
- Testing
- CliniMACS
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia
In this feasibility trial, the investigators will compare participants treated with montelukast and hydroxyurea to those treated with placebo and hydroxyurea for a total of 8 weeks.
- Trial name
- NCT01960413
- Testing
- Montelukast added to Hydroxyurea
- Where
- 2 sites in United States
- Volunteers
- 46 people
- Sponsor
- Vanderbilt University Medical Center
Sickle Cell Disease, Thalassemia Major
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD)…
- Trial name
- NCT04523376
- Testing
- CliniMACS
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Timothy Olson
Sickle Cell Anemia
Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia
The study assesses the efficacy, safety and tolerability of ACZ885 (canakinumab) in pediatric and young adult patients with sickle cell anemia (SCA).
- Trial name
- NCT02961218
- Testing
- ACZ885
- Where
- 15 sites in United States, Canada and 5 more
- Volunteers
- 49 people
- Sponsor
- Novartis Pharmaceuticals
Thalassemia, Non-transfusional-dependent Thalassemia (NTDT), Myeloplastic Dysplasia (MDS), Other Anemia
An Epidemiological Study to Assess Iron Overload Using MRI in Patients With Transfusional Siderosis (TIMES Study)
Iron, one of the most common elements in nature and the most abundant transition metal in the body, is readily capable of accepting and donating electrons.
- Trial name
- NCT01736540
- Testing
- MRI scan
- Where
- 13 sites in Australia
- Volunteers
- 243 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease
This research study is designed to investigate a new potential medicine for mobilizing stem cells and apheresis collection in patients with Sickle Cell Disease. MGTA-145, the new potential medicine, will be given with plerixafor.
- Trial name
- NCT05445128
- Testing
- MGTA-145, Plerixafor
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- Ensoma
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.
Sickle Cell Disease
Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients
The constitution of blood relies upon hematopoietic stem cells (HSCs), which stay in the bone marrow and differentiate to all lineages of peripheral blood cells.
- Trial name
- NCT03226691
- Testing
- Plerixafor
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Chronic Pain, Depression, Anxiety
Cognitive Behavioral Therapy and Real-Time Pain Management Intervention for Sickle Cell Via Mobile Applications
The investigators are conducting a comparative effectiveness trial among adult patients with sickle cell disease (SCD) who report chronic pain (N = 350), randomized to receive either mobile phone-delivered computerized cognitive…
- Conditions
- Chronic Pain, Depression, Anxiety, Opioid Use
- Trial name
- CaRISMA (NCT04419168)
- Testing
- cCBT, m-Education
- Where
- 7 sites in United States
- Volunteers
- 359 people
- Sponsor
- University of Pittsburgh
Thalassemia, Hypertension, Pulmonary
Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension
Thalassemia is an inherited blood disorder that can result in mild to severe anemia. Many people with thalassemia also have pulmonary hypertension, which is high blood pressure in the arteries in the lungs.
- Trial name
- NCT00872170
- Testing
- Sildenafil
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Carelon Research
Sickle Cell Disease
Biotin-RBC Transfusion in SCD
This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion…
- Trial name
- NCT04426591
- Testing
- Biotin Labeled Red Blood Cells, Pathogen-reduced Biotin Labeled Red Blood Cells
- Where
- 3 sites in United States
- Volunteers
- 22 people
- Sponsor
- Marianne Yee