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Found 34 clinical trials
L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension

This study compares L-arginine Versus Sildenafil as treatment for pulmonary hypertension in Children with Beta Thalassemia

hypertension
beta thalassemia
sildenafil
arginine
  • 27 views
  • 23 Feb, 2022
  • 1 location
Clinical Study to Evaluate the Possible Efficacy and Safety of L- Carnitine and Sildenafil in Children Having Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity

This study aims to investigate the possible efficacy and safety of L_Carnitine and Sildenafil on patient with Beta thalassemia complicated with increased Tricuspid Regurgitant Jet Velocity

  • 0 views
  • 28 Oct, 2022
  • 1 location
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study

The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective

ejection fraction
cyclophosphamide
ruxolitinib
cyclosporine
mycophenolate mofetil
  • 17 views
  • 11 Feb, 2022
  • 1 location
A Phase 3b, Open-label, Single-arm, Rollover Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants: Participants receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the …

ace-536
growth factor
cancer
hysterectomy
luspatercept
  • 168 views
  • 28 Oct, 2022
  • 132 locations
A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T) (ENERGIZE-T)

The primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-dependent alpha- or beta-thalassemia (TDT

  • 4 views
  • 13 Oct, 2022
  • 45 locations
A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome

This study will investigate the safety and tolerability of SLN124 in patients with Thalassaemia or patients with Very Low- and Low-risk Myelodysplastic Syndrome after single ascending s.c. doses and multiple doses in healthy male and female subjects. Up to 7 cohorts of 56 patients with Thalassaemia and up to 7 …

  • 0 views
  • 18 Apr, 2022
  • 12 locations
A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE) (ENERGIZE)

The primary purpose of this study is to compare the effect of mitapivat versus placebo on anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).

  • 0 views
  • 17 Oct, 2022
  • 42 locations
Addition of JSP191 (C-kit Antibody) to Non-myeloablative Hematopoietic Cell Transplantation For Sickle Cell Disease and Beta-Thalassemia

Background Sickle cell disease (SCD) is an inherited disorder of the blood. It can damage a person s organs and cause serious illness and death. A blood stem cell transplant is the only potential cure for SCD. Treatments that improve survival rates are needed. Objective To find out if a …

hydroxyurea
thalassemia
sirolimus
Accepts healthy volunteers
alemtuzumab
  • 0 views
  • 27 Oct, 2022
  • 1 location
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM).

splenic sequestration
hydroxyurea
thalassemia
hemoglobin c disease
analgesia
  • 0 views
  • 25 Mar, 2022
  • 1 location
  • 0 views
  • 21 Oct, 2022
  • 2 locations