Clinical trials: Hemoglobinopathy
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Patients with medical conditions requiring allogeneic hematopoietic cell transplantation (allo-HCT) are at risk of developing a condition called graft versus host disease (GvHD) which carries a high morbidity and mortality.
- Conditions
- Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia
- Trial name
- HAPLOTAB (NCT05236764)
- Testing
- CliniMACS
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Non Malignant Diseases, Immunodeficiencies
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
This Phase II pilot study evaluates the safety and effectiveness of reduced-intensity conditioning (RIC) regimens prior to allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric and young adult patients with…
- Conditions
- Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- RIC (NCT01050855)
- Testing
- Reduced-Intensity Conditioning
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Children's Hospital of Philadelphia
Metabolic Disorders, Hematologic, Immune, or Bone Marrow Disorders
Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases
The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells.
- Conditions
- Metabolic Disorders, Hematologic, Immune, or Bone Marrow Disorders, Hemoglobinopathies, Non-malignant Disorders
- Trial name
- NCT00920972
- Testing
- Treatment Plan 1: Stratum 1, Treatment Plan 2: Strata 2, 3, or 4, GVHD Regimen A: UCB Recipients
- Where
- 28 sites in United States, Canada
- Volunteers
- 220 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hemoglobin Disorder
Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Cell Transplant or Gene Therapy
This is a prospective, longitudinal, non-therapeutic study which includes routine assessment for long-term effects, as per FDA guidelines after receipt of an allogeneic HCT or autologous genetically modified cellular products for…
- Trial name
- NCT06646640
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- St. Jude Children's Research Hospital
Aplastic Anemias, Hemoglobinopathies
The HOPE Biobank Resource (BMT CTN 2402 HOPE)
A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood…
- Conditions
- Aplastic Anemias, Hemoglobinopathies, Bone Marrow Failure
- Trial name
- HOPE Resource (NCT07227155)
- Where
- 14 sites in United States
- Volunteers
- 375 people, healthy volunteers welcome
- Sponsor
- Medical College of Wisconsin
Hemoglobinopathies
A Clinical Exploratory Study of YOLT-204 in the Treatment of Hemoglobinopathies
This is a single-arm, open-label, dose-escalation study designed to enroll approximately 5-30 patients with TDT or SCD. The objectives are to evaluate the safety and tolerability in patients following intravenous administration…
- Trial name
- NCT07774351
- Testing
- YOLT-204
- Volunteers
- 30 people
- Sponsor
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Sickle Cell Disease, Sickle Cell Anaemia
Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype
SIL-8301 (senicapoc) is being developed for the chronic treatment of patients with sickle cell disease in both adults and children.
- Conditions
- Sickle Cell Disease, Sickle Cell Anaemia, Sickle Cell Anemia
- Trial name
- RESCUE (NCT07282210)
- Testing
- Senicapoc
- Where
- 4 sites in Canada
- Volunteers
- 105 people
- Sponsor
- Biossil Inc.
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 Disorders, Hemoglobinopathies
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) in patients with non-malignant or malignant diseases.
- Conditions
- Hematologic Disorders, Hemoglobinopathies, Immunodeficiencies
- Trial name
- NCT01666080
- Testing
- Busulfan, Fludarabine, Total body irradiation
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Hemoglobinopathy (Disorder), Severe Aplastic Anemia
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition.
- Conditions
- Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
- Trial name
- NCT04356469
- Testing
- Haploidentical Hematopoietic Cell Transplantation
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Johns Hopkins All Children's Hospital
Sickle Cell Disease, Hydroxyurea Failure
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human…
- Conditions
- Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies
- Trial name
- NCT05329649
- Testing
- CTX001
- Where
- 7 sites in United States, Germany and 2 more
- Volunteers
- 13 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Beta-Thalassemia, Thalassemia
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT05356195
- Testing
- CTX001
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 16 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Bone Marrow Failure, Hemoglobinopathies
Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases
This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant diseases requiring hematopoietic cell transplantation.
- Conditions
- Bone Marrow Failure, Hemoglobinopathies, Primary Immune Deficiency (PID), Autoimmune Cytopenia
- Trial name
- NCT07660783
- Testing
- ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
- Where
- 2 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
Graft Failure, Sickle Cell Disease
Allo HSCT for High Risk Hemoglobinopathies
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation…
- Conditions
- Graft Failure, Sickle Cell Disease, Hemoglobinopathies
- Trial name
- NCT06872333
- Testing
- Alemtuzumab, Total Body Irradiation, Cell Infusion
- Where
- United States
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Masonic Cancer Center, University of Minnesota
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
- Conditions
- Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders
- Trial name
- FAM BMT (NCT03128996)
- Testing
- RIC regimen, GVHD prophylaxis regimen
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Washington University School of Medicine
Hemoglobinopathies
Discarded Bone Marrow for Hematology Research
The primary objective of this study is to establish a mechanism to obtain discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-hemoglobinopathy individuals.
- Trial name
- NCT04671212
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- St. Jude Children's Research Hospital
Leukemia, High Risk Acute Lymphoblastic Leukemia
Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
The major morbidities of allogeneic hematopoietic stem cell transplant (HSCT) using donors that are not human leukocyte antigen (HLA) matched siblings are graft vs host disease (GVHD) and life- threatening infections.
- Conditions
- Leukemia, High Risk Acute Lymphoblastic Leukemia, High Risk Acute Myeloid Leukemia, Relapse Leukemia
- Trial name
- NCT06839456
- Testing
- Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Children's Hospital of Philadelphia
Sickle Cell Disease
A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of…
- Trial name
- HIBISCUS (NCT04624659)
- Testing
- Etavopivat Tablets Low dose, Etavopivat Tablets High dose, Etavopivat Tablets
- Where
- 142 sites in United States, Canada and 15 more
- Volunteers
- 450 people
- Sponsor
- Forma Therapeutics, Inc.
Beta-Thalassemia, Thalassemia
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD).
- Conditions
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn
- Trial name
- NCT05477563
- Testing
- CTX001
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 26 people
- Sponsor
- Vertex Pharmaceuticals Incorporated