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Found 60 clinical trials
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Sickle Cell Disease Research Study - Fulcrum Therapeutics  

The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.

hemoglobin f
thalassemia
  • 12956 views
  • 31 Jan, 2023
  • 5 locations
Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia

The purpose of this study is to evaluate a novel transplant strategy for the long-term benefit of patients with transfusion dependent high-risk thalassemia.

  • 0 views
  • 15 Jul, 2022
  • 1 location
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

beta thalassemia
sildenafil
hypertension
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

thymoglobulin
mycophenolate mofetil
fludarabine
cell transplantation
busulfan
  • 17 views
  • 11 Feb, 2022
  • 1 location
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
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 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
hysterectomy
growth factor
luspatercept
cancer
  • 168 views
  • 28 Oct, 2022
  • 132 locations
Open Label, Multi-center, Phase IV Study of Ruxolitinib or Ruxolitinib and Panobinostat Combination, for Patients Who Have Completed Prior Global Novartis or Incyte Sponsored Studies

This is a long term safety study for patients that have been treated with either ruxolitinib or a combination of ruxolitinib with panobinostat, on a Novartis or Incyte sponsored study, who have been judged by the study Investigator to benefit from ongoing treatment.

panobinostat capsules
progressive disease
panobinostat
ruxolitinib tablets
ruxolitinib
  • 148 views
  • 15 Oct, 2022
  • 28 locations
The BENeFiTS Trial in Beta Thalassemia Intermedia (PB04-001)

Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers

thalassemia
  • 12 views
  • 03 May, 2022
  • 4 locations