Clinical trials: Newborn; Anemia
Sickle Cell Disease
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with…
- Trial name
- NCT07224360
- Testing
- Anumigilimab
- Where
- 38 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- CSL Behring
Anemia, Sickle Cell
A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male Subjects
This study will provide information regarding the metabolic pathway of GBT440, the need for evaluation of potential drug-drug interactions, and the need for studies in special populations.
- Trial name
- NCT02497924
- Testing
- GBT440
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics
Beta-Thalassemia
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting.
- Trial name
- NCT06271512
- Testing
- No Intervention
- Where
- 6 sites in United States
- Volunteers
- 150 people
- Sponsor
- Genetix Biotherapeutics Inc.
Beta-Thalassemia
A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia
This proof-of-mechanism study is being performed to investigate the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of multiple oral doses of bitopertin in adults with NTD beta-thalassemia.
- Trial name
- NCT03271541
- Testing
- Bitopertin
- Where
- 4 sites in Italy, Lebanon and 1 more
- Volunteers
- 12 people
- Sponsor
- Hoffmann-La Roche
Chronic Pain
Effectiveness, Safety, and Tolerability Study of Oxymorphone Immediate Release (IR) Oral Liquid in Opioid Tolerant Pediatric Subjects
The purpose of this study is to evaluate the effectiveness, tolerability, and safety of oxymorphone immediate release (IR) oral liquid as an analgesic in pediatric subjects having severe to moderate chronic pain.
- Trial name
- NCT01206907
- Testing
- oxymorphone IR
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Sickle Cell Disease, Vaso-occlusive Crisis
Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis
GMI-1070 is a new drug that may reduce the stickiness of cells in the blood. The purpose of this study is to evaluate whether GMI-1070 can reduce the time it takes for pain to go away in patients with vaso-occlusive crisis (also…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Pain Crisis
- Trial name
- NCT01119833
- Testing
- GMI-1070
- Where
- 22 sites in United States, Canada
- Volunteers
- 81 people
- Sponsor
- GlycoMimetics Incorporated
Sickle Cell Disease
Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease
The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.
- Trial name
- NCT00842088
- Testing
- HQK-1001
- Where
- 9 sites in United States, Jamaica
- Volunteers
- 24 people
- Sponsor
- HemaQuest Pharmaceuticals Inc.
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
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Post Hematopoietic Stem Cell Transplantation
This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant…
- Trial name
- NCT01610297
- Testing
- ICL670
- Where
- 6 sites in Turkey (Türkiye)
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Pulmonary Hypertension, Lung Disease
Hospital-Based Program for Treatment of Severe Cardiopulmonary Disease With Inhaled Nitric Oxide
The purpose of this program is to evaluate the logistic issues and patient requirements for chronic pulsed INOmax delivery in ambulatory, home-care patients.
- Conditions
- Pulmonary Hypertension, Lung Disease, Sickle Cell Disease, Cardiac Transplant
- Trial name
- NCT00041574
- Testing
- Inhaled Nitric Oxide
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Mallinckrodt
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Anemia, Sickle ß0-Thalassemia
A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for Sickle Cell Anemia or Sickle ß0 Thalassemia as evaluated by the number of occurrences of sickle cell crises.
- Trial name
- NCT01179217
- Testing
- L-glutamine
- Where
- 31 sites in United States
- Volunteers
- 230 people
- Sponsor
- Emmaus Medical, Inc.
Thalassemia
A Study of SNH-119014 in Adult Participants With Non-transfusion-dependent Thalassemia (NTDT)
This study is a multicenter study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of treatment with SNH-119014 in adult participants with non-transfusion-dependent thalassemia.
- Trial name
- NCT07579949
- Testing
- SNH-119014 dose 1, SNH-119014 dose 2
- Where
- 8 sites in China
- Volunteers
- 30 people
- Sponsor
- ScinnoHub Pharmaceutical Co., Ltd.
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT01419704
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Sponsor
- Talaris Therapeutics Inc.
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
Thalassemia Major, Hemosiderosis
Efficacy Study in Removing Excess Iron From the Heart
The purpose of this study is to determine whether deferiprone has superior efficacy in removing excess iron from the heart when compared with deferoxamine.
- Trial name
- NCT00105495
- Testing
- Ferriprox (deferiprone), Desferal (deferoxamine)
- Where
- 4 sites in Greece, Italy
- Volunteers
- 60 people
- Sponsor
- ApoPharma
Sickle Cell Disease
Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)
This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle…
- Trial name
- NCT04443907
- Testing
- OTQ923
- Where
- 3 sites in United States
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
Transfusional Iron Overload
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional…
- Trial name
- NCT01044186
- Testing
- ICL670
- Where
- 18 sites in United States, Greece and 1 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Healthy Volunteers Sickle Cell Disease, Thalassemia
A Research Study of the Effect of Food on Etavopivat in Healthy Participants
The purpose of this study is to evaluate the effect of food on the amount of etavopivat in the bloodstream of healthy participants. Participants will take a single oral dose of etavopivat following a high-fat meal (i.e.
- Trial name
- NCT06433661
- Testing
- Etavopivat
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S