Clinical trials: Thalassemia Intermedia
Thalassemia Majors (Beta-Thalassemia Major)
Gonadal Function in Thalassemic Patient
Patient with transfusion depandant beta thalassemia with accumulation of iron in their body affect pituitary gland and gonades lead to impaired their functions .
- Trial name
- NCT07288762
- Testing
- serum inhibin B serum INSL3 serum AMH
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Sohag University
Beta Thalassemia
Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia
- Trial name
- NCT00790127
- Testing
- HQK-1001
- Where
- 2 sites in Lebanon, Thailand
- Volunteers
- 21 people
- Sponsor
- HemaQuest Pharmaceuticals Inc.
Extremely Premature Infant, Anemia Neonatal
Heterologous Cord Blood-Derived Red Blood Cell for Transfusion in Extremely Preterm Infants
Anemia is a condition in which there are not enough red blood cells to carry oxygen throughout the body. It is very common in extremely preterm infants (born before 28 weeks of pregnancy), and many of these babies require red…
- Conditions
- Extremely Premature Infant, Anemia Neonatal, Blood Transfusion, Umbilical Cord Blood
- Trial name
- NCT07502781
- Testing
- Cord Blood Red Blood Cells, Standard Adult Donor Red Blood Cells
- Where
- Spain
- Volunteers
- 176 people
- Sponsor
- Hospital Clinic of Barcelona
Beta-Thalassemia
Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients
Beta-thalassemias are hereditary blood disorders caused by reduced or absent synthesis of hemoglobin beta chains, with variable outcomes ranging from severe anemia to clinically asymptomatic individuals.
- Trial name
- SIRTHALACLIN (NCT03877809)
- Testing
- Sirolimus 0.5 mg
- Where
- 2 sites in Italy
- Volunteers
- 26 people
- Sponsor
- Rare Partners srl Impresa Sociale
Hematologic Malignancies
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders
To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or…
- Trial name
- HPDSC (NCT00596999)
- Testing
- UCB and HPDSC
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Celgene Corporation
Beta Thalassemia
5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia
This study will evaluate the safety and effectiveness of 5-azacytidine and phenylbutyrate for treating thalassemia major. Patients with this disease have abnormal production of hemoglobin (the oxygen-carrying protein in red blood…
- Trial name
- NCT00005934
- Testing
- 5-Azacytidine
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sickle Cell Anemia, Beta-Thalassemia
Prevalence Of Microalbuminuria Among Children Suffering From Sickle Cell Nephropathy and Sickle Cell/Beta-Thalassemia
Sickle cell nephropathy is a known complication of sickle cell anemia (SCA) manifested by increase in glomerular filtration rate (glomerular hyperfiltration) and results in proteinuria and chronic renal failure.
- Conditions
- Sickle Cell Anemia, Beta-Thalassemia, Microalbuminuria
- Trial name
- NCT01092169
- Testing
- No intervention
- Sponsor
- Hillel Yaffe Medical Center
Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
Determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia and sickle cell disease toward gene therapy to treat their or their child's illness…
- Trial name
- NCT06664541
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Georgetown University
Thalassemia Major
Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion
To evaluate the efficacy and safety of S 303 treated red blood cells (RBCs) in subjects who require chronic transfusion support due to thalassemia major.
- Trial name
- NCT01740531
- Testing
- S-303 Treated Red Blood Cells (RBCs), Conventional, untreated Red Blood Cells
- Where
- 3 sites in Italy, Turkey (Türkiye)
- Volunteers
- 86 people
- Sponsor
- Cerus Corporation
Beta-thalassemia Major Complicated With Diabetes
Zinc Supplementation in Patients With β-Thalassemia Major Complicated With Diabetes Mellitus
Beta-thalassemia represents a group of recessive inherited hemoglobin disorders characterized by reduced synthesis of β-globin chain.
- Trial name
- NCT03851055
- Testing
- Zinc
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Ain Shams University
Beta-Thalassemia
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells.
- Trial name
- NCT06364774
- Testing
- ALS20
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
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
Myeloid Chimerism
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some…
- Trial name
- NCT04008368
- Testing
- CliniMACS CD34 Reagent
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Iron Overload, Thalassemia Major
"Iron Overload and Endocrinological Diseases"
Patients with hemochromatosis or Thalassemia develop progressive tissue and organs damages secondary to iron overload. Iron overload can result both from transfusional hemosiderosis and excess gastrointestinal iron absorption.
- Conditions
- Iron Overload, Thalassemia Major, Thalassemia Intermedia, Hemochromatosis
- Trial name
- NCT06137079
- Where
- Italy
- Volunteers
- 62 people
- Sponsor
- Azienda Ospedaliero-Universitaria di Modena
Thalassemia
The Role of OCTA in Patients Affected by Beta Thalassemia
To investigate, using optical coherence tomography angiography (OCTA), the retinal and choriocapillaris vascular features in patients affected by Beta Thalassemia.
- Trial name
- NCT04582110
- Testing
- Optical coherence tomography angiography
- Where
- Italy
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- Federico II University
Beta-thalassemia, Serum Ferritin
Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)
safety and efficacy of different iron chelation therapy in transfusion dependent beta-thalaasemia patients with low serum ferritin and continued regular transfusion regimen.
- Conditions
- Beta-thalassemia, Serum Ferritin, Iron Chelation Therapy
- Trial name
- NCT01996683
- Testing
- desferal, ferriprox, blood transfusion
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Ain Shams University
Beta-Thalassemia, Thalassemia
Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia
This study will compare two treatments for children with beta-thalassemia who need regular blood transfusions and have not responded well to thalidomide. One group will continue thalidomide alone.
- Trial name
- NCT07884773
- Testing
- Thalidomide, Danazol, Prednisolone
- Where
- Pakistan
- Volunteers
- 172 people
- Sponsor
- Khyber Medical University Peshawar
Hemoglobinopathies
Prevalence of Hemoglobinopathies Associated With Significant Hb Variants in the Chattogram Region of Bangladesh
Hemoglobinopathies represent a collection of genetic conditions that influence the structure or synthesis of haemoglobin, the protein found in red blood cells that facilitates oxygen transport from the lungs throughout the body.
- Trial name
- Hb variants (NCT07207551)
- Where
- Bangladesh
- Volunteers
- 760 people
- Sponsor
- Bangladesh Bioscience Research Group
Transfusion-dependent Beta-Thalassemia
Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem…
- Trial name
- NCT06219239
- Testing
- KL003 cell injection Drug Product
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.
- Trial name
- ENERGIZEKids (NCT07517133)
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 45 people
- Sponsor
- Agios Pharmaceuticals, Inc.