Clinical trials: Thalassaemia, active
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)
Beta-Thalassemia
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and…
- Trial name
- NCT06772766
- Testing
- 9MW3011
- Where
- 2 sites in China
- Volunteers
- 40 people
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
Sickle Cell Disease, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
- Conditions
- Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia
- Trial name
- NCT05777993
- Testing
- Mitapivat
- Where
- 23 sites in Belgium, Brazil and 6 more
- Volunteers
- 55 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Transfusion-dependent Beta-Thalassemia
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
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
- NCT05860595
- Testing
- KL003 cell injection Drug Product
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Fetal Hemoglobin, Thalassemia Major
Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing…
- Conditions
- Fetal Hemoglobin, Thalassemia Major, Genetic Disease
- Trial name
- BTM (NCT06490627)
- Testing
- Thalidomide
- Where
- Pakistan
- Volunteers
- 54 people
- Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Alpha-Thalassemia, Alpha Thalassemia Major
International Registry of Patients With Alpha Thalassemia
This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of…
- Conditions
- Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor
- Trial name
- ATM Registry (NCT04872179)
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- University of California, San Francisco
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
Alpha Thalassemia
Screening for Alpha Thalassemia in Healthy Volunteers
Background: Alpha thalassemia is a blood disorder. It is caused by genetic deletions. Part of the DNA is missing from a group of genes called alpha globin. Alpha thalassemias are some of the most common genetic deletions.
- Trial name
- NCT02692872
- Where
- United States
- Volunteers
- 367 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Beta-Thalassemia
A Long-term Follow-up Study in Participants Who Received CS-101
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-06 (NCT06024876),No investigational drug product will be administered in the study.
- Trial name
- NCT06685536
- Testing
- Safety and efficacy assessments
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Transfusion-dependent Beta-Thalassemia
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent…
- Trial name
- NCT02633943
- Testing
- Safety and efficacy assessments
- Where
- 15 sites in United States, Australia and 6 more
- Volunteers
- 66 people
- Sponsor
- Genetix Biotherapeutics Inc.
B Thalassemia Major
Oral Health Status in Children With β-Thalassemia Major
To evaluate the effectiveness of a structured oral health educational program on oral hygiene status and oral health-related quality of life (OHRQoL) among children diagnosed with β-thalassemia.
- Trial name
- NCT07614217
- Testing
- oral health educational program for Immediate Intervention Group, routine oral hygiene instructions
- Volunteers
- 46 people
- Sponsor
- Cairo University
Haemoglobin Barts Hydrops
In-utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Bart's Hydrops Fetalis Syndrome
This is a prospective observational pilot study on pregnant women who are diagnosed to have Bart's hydrops fetalis syndrome (BHFS) affected fetuses and opt for continuation of pregnancy will be invited to consider undergoing…
- Trial name
- NCT05797272
- Testing
- in-utero HSCT
- Where
- Hong Kong
- Volunteers
- 10 people
- Sponsor
- Chinese University of Hong Kong
Red Cell Alloantibodies, Blood Transfusion-Dependent
Evaluation of Micro RNA-155 Expression in Relation to Alloantibody Formation in Transfusion-Dependent Patients
Transfusion-dependent patients, particularly those with β-thalassemia major, require lifelong regular red blood cell (RBC) transfusions to maintain adequate hemoglobin levels and prevent severe anemia.
- Conditions
- Red Cell Alloantibodies, Blood Transfusion-Dependent, Beta Thalassemia
- Trial name
- NCT07480980
- Volunteers
- 100 people
- Sponsor
- Assiut University
Sickle Cell Anemia (HbSS), Sickle-β0-thalassemia (HbSβ0)
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has been studied for many years and has been shown to provide benefits for…
- Trial name
- ENHANCE (NCT07177300)
- Testing
- PK-optimized oral hydroxyurea at MTD until 15 years of age.
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical…
- Trial name
- NYMC-571 (NCT02675959)
- Testing
- Defibrotide
- Where
- 4 sites in United States
- Volunteers
- 40 people
- Sponsor
- New York Medical College
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Transfusion Dependent Thalassemia
Evaluation of Recombinant Humanized Anti-CD25 Monoclonal Antibody for Preventing Graft-versus-host Disease After Haploidentical/matched Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Transfusion-dependent Thalassemia
Graft-versus-host disease (GVHD) is a major factor affecting the efficacy and quality of life of alternative donor transplantation in thalassemia major (TM), severely limiting the clinical application of alternative donor…
- Trial name
- NCT06657391
- Testing
- recombinant humanized anti-CD25 monoclonal antibody
- Where
- 5 sites in China
- Volunteers
- 396 people
- Sponsor
- Rongrong Liu
Beta-Thalassemia
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s).
- Trial name
- NCT03351829
- Testing
- Gene-modified autologous hematopoeitic stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute