Clinical trials: Severe Anemia
Severe Aplastic Anemia
Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia
BMT CTN 2207 will investigate the use of marrow transplantation for treatment of severe aplastic anemia that has not previously been treated.
- Trial name
- BMT CTN CureAA (NCT06517641)
- Testing
- Haploidentical donor bone marrow transplant, Unrelated donor bone marrow transplant
- Where
- 25 sites in United States
- Volunteers
- 60 people
- Sponsor
- Medical College of Wisconsin
Fanconi Anemia, Severe Aplastic Anemia
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine…
- Conditions
- Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes, T Cell Receptor Alpha/Beta Depletion
- Trial name
- NCT03579875
- Testing
- Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Treatment-naïve Severe Aplastic Anemia
A Study of Romiplostim N01 Plus IST vs. Placebo Plus IST for Treatment-Naive Severe Aplastic Anemia
This is a randomized, double-blind, multicenter trial designed to evaluate treatment with romiplostim N01+ IST compared with placebo + IST in the participants with treatment-naïve severe aplastic anemia.
- Trial name
- NCT07345000
- Testing
- Romiplostim N01, Cyclosporine A (CsA), pALG/ rATG
- Volunteers
- 210 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Severe Aplastic Anemia (SAA)
Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia
This study was designed to evaluate the efficacy and safety of eltrombopag when added to r-ATG and CsA in treatment naive East-Asian adult and pediatric patients with severe aplastic anemia (SAA).
- Trial name
- REACTS (NCT04328727)
- Testing
- eltrombopag, rabbit anti-thymocyte globulin (r-ATG), cyclosporine A (CsA)
- Where
- 12 sites in China, Japan and 2 more
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
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
Recurrent Severe Aplastic Anemia, Refractory Severe Aplastic Anemia
A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia
This phase I trial evaluates the safety and feasibility of using a reduced-intensity regimen of cyclophosphamide, pentostatin, and anti-thymocyte globulin prior to a CD4+ T-cell depleted haploidentical hematopoietic cell…
- Trial name
- NCT05757310
- Testing
- Anti-Thymocyte Globulin, Biospecimen Collection, Bone Marrow Aspirate
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- City of Hope Medical Center
Iron Absorption, Iron Deficiencies
Maximizing the Benefits of Iron in Ready-to-Use Therapeutic Foods for Malnourished Children in Kenya
Reports from the Kenya Red Cross Society (KRCS) in Kwale County, southern Kenya, indicate a limited impact of Ready-to-Use Therapeutic Foods (RUTFs) on malnutrition or anemia.
- Conditions
- Iron Absorption, Iron Deficiencies, Severly Acutely Malnourished Children, Malnutrition in Children
- Trial name
- SAMI (NCT07315295)
- Testing
- Iron absorption from RUTF on Day 0, Treatment with RUTF for 80 days, Iron absorption from RUTF on Day 20
- Where
- 2 sites in Kenya, Switzerland
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- ETH Zurich
Severe Aplastic Anemia, Refractory Aplastic Anemia
Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia
This study is designed as a Phase Ib/II trial. The phase Ib cohort will enroll patients with severe aplastic anemia (SAA) who have failed to respond to intensified immunosuppressive therapy.
- Conditions
- Severe Aplastic Anemia, Refractory Aplastic Anemia, Newly Diagnosed Aplastic Anemia
- Trial name
- NCT07297550
- Testing
- Golidocitinib Capsules
- Volunteers
- 42 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Severe Malaria, Severe Anemia
PDMC Implementation Trial in Kenya
The goal of this implementation trial is to evaluate at least two alternative delivery strategies and adherence support for malaria chemoprevention with dihydroartemisinin-piperaquine in the post-discharge management of children…
- Trial name
- PDMC-SL (NCT06624631)
- Testing
- SMS reminders; adherence support strategy a, Community Health Promoters (CHP) home visits; adherence support strategy b, No reminders; adherence support strategy c
- Where
- Kenya
- Volunteers
- 600 people
- Sponsor
- Liverpool School of Tropical Medicine
Fanconi Anemia, Severe Marrow Failure
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA).
- Conditions
- Fanconi Anemia, Severe Marrow Failure, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
- Trial name
- RAFA (NCT02143830)
- Testing
- Busulfan, Cyclophosphamide, Fludarabine
- Where
- 3 sites in United States
- Volunteers
- 70 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Non Severe Aplastic Anemia, Untreated
Herombopag Added to Cyclosporine in Non Severe Aplastic Anemia
This is a prospective, multicenter, single-arm, phase 2 trial. The aim of this study is to evaluate the efficacy and safety of herombopag combined with cyclosporine for patients with non severe aplastic anemia (NSAA).
- Trial name
- NCT05660785
- Testing
- Herombopag
- Where
- 4 sites in China
- Volunteers
- 54 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Severe Aplastic Anemia, Relapse
CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia
This trial is exploratory research aimed at evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD13-02, a universal CD7 CAR T therapy, in subjects with relapsed/refractory severe aplastic…
- Conditions
- Severe Aplastic Anemia, Relapse, Refractory, CAR T-cell Therapy
- Trial name
- NCT06622694
- Testing
- RD13-02
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Severe Aplastic Anemia
Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia
The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem cell transplant (HSCT) for…
- Trial name
- NCT02845596
- Testing
- cyclosporine, Matched Unrelated Donor Hematopoietic Stem Cell Transplant, horse anti-thymocyte globulin (ATG)
- Where
- 13 sites in United States
- Volunteers
- 40 people
- Sponsor
- Michael Pulsipher
Transfusion-dependent Non-severe Aplastic Anemia
Treatment of Transfusion-dependent Nonsevere Aplastic Anemia With Luspatercept: a Multicenter Prospective Clinical Study
The goal of this clinical trial is to learn whether Luspatercept alone or in combination with Deferasirox can promote hematopoietic function in patients with transfusion-dependent non-severe aplastic anemia, as well as to assess…
- Trial name
- NCT06964971
- Testing
- Luspatercept, Luspatercept combine with Deferasirox
- Where
- China
- Volunteers
- 90 people
- Sponsor
- The First Affiliated Hospital of Zhejiang Chinese Medical University
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT02179359
- Testing
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Moderate to Severe Anemia, Traditional Chinese Medicine (TCM)
Pulse Diagnosis and Anemia: Exploring Integrated Traditional Chinese and Western Medicine Approaches
This observational study evaluates the efficacy and safety of integrated Traditional Chinese Medicine (TCM) and Western medicine in managing moderate to severe anemia.
- Conditions
- Moderate to Severe Anemia, Traditional Chinese Medicine (TCM), Pulse Diagnosis
- Trial name
- NCT06760351
- Testing
- TCM Herbal Therapy, Standard Western Medicine Treatment
- Where
- Taiwan
- Volunteers
- 28 people
- Sponsor
- Chia-Ying Chuang