Clinical trials: Anemia
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
Sevuparin Infusion for the Management of Acute VOC in Subjects With SCD
A Multi-Centre, Phase II, Randomized, Double-Blind, Placebo-Controlled Study to investigate Efficacy and Safety of Sevuparin Infusion for the Management of Acute Vaso-Occlusive Crisis (VOC) in Subjects with Sickle-Cell Disease…
- Trial name
- NCT02515838
- Testing
- Sevuparin
- Where
- 20 sites in Bahrain, Jamaica and 5 more
- Volunteers
- 147 people
- Sponsor
- Modus Therapeutics AB
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.
Sickle Cell Disease, Iron Overload
Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions.
- Conditions
- Sickle Cell Disease, Iron Overload, Hemolytic Anemia
- Trial name
- NCT00110617
- Testing
- Deferasirox (ICL670), Deferoxamine (DFO)
- Where
- 59 sites in United States, Canada
- Volunteers
- 212 people
- Sponsor
- Novartis Pharmaceuticals
Anemia
A Study to Evaluate the Safety of Epoetin Alfa and Its Effectiveness in Facilitating the Presurgical Collection of Blood From Anemic Patients for Possible Self-transfusion During and After Scheduled Joint Surgery.
The purpose of this study is to evaluate the safety of epoetin alfa and its effectiveness in facilitating the presurgical collection of blood from anemic patients for possible self-transfusion during and after scheduled joint…
- Trial name
- NCT00270062
- Testing
- epoetin alfa
- Volunteers
- 77 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
Anemia, Aplastic
Safety and Efficacy Study of Ex Vivo Immunotherapy for Treatment of Aplastic Anemia
Our hypothesis is that ex vivo activated immune cells would produce multiple known and unknown potent hematopoietic cytokines, working in concert, these cytokines help stem cell growth and differentiation.
- Trial name
- NCT00399971
- Testing
- Ex Vivo Immunotherapy
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Shenzhen Zhongxing Yangfan Biotech Co. Ltd.
Atypical Hemolytic Uremic Syndrome(aHUS)
A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients
This is a China, non-interventional, observational study and will follow the Good Phar-macoepidemiology Practices guidelines. This study will enrol paediatric and adult patients diagnosed with aHUS who will be treated according…
- Trial name
- NCT06099236
- Where
- 26 sites in China
- Volunteers
- 367 people
- Sponsor
- AstraZeneca
Beta-Thalassemia, Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161…
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
- Trial name
- NCT04208529
- Testing
- CTX001
- Where
- 20 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hematologic Malignancy, Acute Leukemia
Study to Characterize Mismatched to Fully HLA-Matched Ossium HPC, Marrow and Living Donor Transplantation in Patients With Hematologic Malignancies
Prospective, multi-center open label study of HLA-partially to fully matched allogeneic cryopreserved deceased donor bone marrow transplantation and living donor transplantation for patients with hematologic malignancies.
- Conditions
- Hematologic Malignancy, Acute Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT07710781
- Testing
- Ossium HPC, Marrow, Standard of care living donor - Haplo Related, Standard of care living donor - Mismatched Unrelated
- Volunteers
- 300 people
- Sponsor
- Ossium Health, Inc.
Anemia
A Study of Patients Treated With Erythropoietin Receptor Agonists for Anemia Who Developed Anti-erythropoietin Antibodies
The purpose of this study is to assess the presence of anti erythropoietin antibodies (anti EPO Ab) in participants responding to any erythropoietin receptor agonist (ERA) therapy to treat or prevent anemia without loss of…
- Trial name
- NCT00210977
- Testing
- No intervention
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
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
G6PD, G6PD Deficiency
Comparative Evaluation of the FINDER Instrument and FINDER G6PD Cartridge in Adults and Neonates
A prospective clinical study to compare the clinical performance of the FINDER™ Instrument and the G6PD assay to an FDA cleared instrument/reagent combination in adult and neonate populations.
- Trial name
- NCT04146246
- Testing
- G6PD assay
- Where
- 3 sites in United States
- Volunteers
- 119 people, healthy volunteers welcome
- Sponsor
- Baebies, Inc.
Β-thalassemia
Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia.
- Trial name
- NCT06655662
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- 3 sites in China
- Volunteers
- 8 people
- Sponsor
- Shenzhen Hemogen
Anemia
Iron Sucrose in Non-Dialysis Dependent (NDD-CKD) Pediatric Patients
The primary objective of this study is to assess the pharmacokinetics of Venofer (Iron Sucrose Injection) in NDD-CKD pediatric patients.
- Trial name
- NCT00721188
- Testing
- Venofer
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- American Regent, Inc.
Sickle Cell Disease, Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- Trial name
- FLORAL (NCT06609226)
- Testing
- Etavopivat A, Etavopivat B, Etavopivat C
- Where
- 107 sites in United States, Canada and 15 more
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S
Anemia
Measurement of Total Hemoglobin by Pulse Oximetry in Neonatal Population
Assess ability of Masimo SpHb sensor to demonstrate robust performance on various sensor application sites on neonates/infants
- Trial name
- NCT03120702
- Testing
- Investigational Rainbow SpHb sensor
- Where
- United States
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- Masimo Corporation
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
Pulmonary Hypertension
Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients With Pulmonary Arterial Hypertension (PAH)
The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in sickle cell disease (SCD) patients diagnosed with pulmonary arterial hypertension.
- Trial name
- NCT00310830
- Testing
- Bosentan
- Volunteers
- 14 people
- Sponsor
- Actelion