Clinical trials: Aplastic Anaemia
Chronic Kidney Disease, End-Stage Renal Failure
Cohort Study to Determine the Long-Term Safety and Efficacy of Biogeneric Epoetin Treatment for Renal Anemia
The purpose of this study is to establish the long-term safety and efficacy of Biogeneric Epoetin, the attainability of therapeutic target for anaemia management, and the impact of Epoetin treatment on long-term health outcome…
- Conditions
- Chronic Kidney Disease, End-Stage Renal Failure, Anaemia
- Trial name
- NCT00238043
- Testing
- Biogeneric Epoetin
- Where
- 14 sites in Malaysia
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Ministry of Health, Malaysia
Fanconi Anemia
FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
This is a Phase I/II clinical trial of gene therapy for treating Fanconi anemia using a self-inactivating lentiviral vector to functionally correct the defective gene.
- Trial name
- NCT03351868
- Testing
- Gene-modified autologous stem cells
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Fanconi Anemia, Head and Neck Squamous Cell Carcinoma
Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC)
This research study is a phase Ib/II, single-arm, non-randomized, non-blind, multicenter study designed to determine whether Afatinib is effective and safe in patients with locoregionally unresectable and / or metastatic HNSCC…
- Trial name
- AFAN (NCT06648096)
- Testing
- Afatinib
- Where
- 2 sites in Germany, Spain
- Volunteers
- 25 people
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Lymphoma, Leukemia
Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation
RATIONALE: Following stem cell transplantation, a major risk is graft-versus-host disease (GVHD). This occurs when donor immune cells that have been infused recognise the host's cells as 'foreign' and attack these cells.
- Conditions
- Lymphoma, Leukemia, Myeloma, Myelodysplastic Syndromes
- Trial name
- ToTem (NCT03836690)
- Testing
- CD62L- Tem
- Where
- United Kingdom
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University College, London
Bone Marrow Failure Syndromes, Aplastic Anemias
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse
- Trial name
- BMF (NCT07712562)
- Testing
- Dibotatug (DR-01)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Dren Bio
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
Aplastic Anemia
Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia
Primary Objectives: 1. To determine the feasibility and toxicity of employing purine-analog based conditioning for allogeneic donor stem cell transplantation in patients with severe aplastic anemia (AA). 2.
- Trial name
- NCT00427336
- Testing
- Fludarabine, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- M.D. Anderson Cancer Center
Bone Marrow Transplant (BMT)
HPC Offered for PRESERVE Expansion
Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons.
- Trial name
- HOPE (NCT07145411)
- Testing
- Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.
Anemia, Aplastic Anemia
Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study
This pilot study evaluates safety of administration of red blood cell transfusions requested by patients based on their symptoms instead of levels of hemoglobin for the treatment of chronic anemia in patients with blood disorders.
- Conditions
- Anemia, Aplastic Anemia, Bone Marrow Failure, Hematopoietic and Lymphoid Cell Neoplasm
- Trial name
- NCT03086252
- Testing
- Red Blood Cell Transfusions (RBCT)
- Where
- United States
- Sponsor
- Emory University
Aplastic Anemia
Romiplostim N01 in Chemoradiotherapy-induced Aplastic Anemia
This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of chemoradiotherapy-induced aplastic anemia (AA)
- Trial name
- NCT07572084
- Testing
- Romiplostim N01
- Volunteers
- 43 people
- Sponsor
- Peking Union Medical College Hospital
Aplastic Anemia, Myelodysplastic Syndromes
Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients
To investigate the efficacy of posaconazole as prophylaxis antifungal agent in aplastic anemia / hypoplastic myelodysplastic syndrome (AA/hMDS) patients undergoing antithymocyte globulin (ATG) treatment
- Conditions
- Aplastic Anemia, Myelodysplastic Syndromes, Fungal Infection
- Trial name
- NCT03318159
- Testing
- Posaconazole
- Where
- South Korea
- Volunteers
- 20 people
- Sponsor
- Seoul National University Hospital
Bone Marrow Failure Disorders, Aplastic Anemia
TPO-Mimetic Use in Children for Hematopoietic Failure
This is an open label, prospective Pilot interventional study will investigate the safety and efficacy of Romiplostim, thrombopoietin (TPO) mimetic, in children (ages: 0 to 21 years) with broad scope of bone marrow failure…
- Conditions
- Bone Marrow Failure Disorders, Aplastic Anemia, Thrombocytopenia, Refractory Cytopenia of Childhood
- Trial name
- NCT04478227
- Testing
- Romiplostim
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Anjali Sharathkumar
Aplastic Anemia
ATGAM General Investigation
The objective of this study is to confirm the safety of ATGAM in patients with moderate to severe aplastic anemia under the actual use in Japan.
- Trial name
- NCT06039020
- Testing
- Anti-human thymocyte immunoglobulin, equine
- Where
- Japan
- Volunteers
- 1 people
- Sponsor
- Pfizer
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Anemia, Aplastic, Blood Disease
Etiology of Blood Dyscrasias: Analysis of the International Agranulocytosis and Aplastic Anemia Study Data
To quantify the role of drugs and other factors in the etiology of agranulocytosis and aplastic anemia.
- Conditions
- Anemia, Aplastic, Blood Disease, Agranulocytosis
- Trial name
- NCT00005307
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Fanconi Anemia
Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia
Prospective interventional open-label non-randomized controlled trial to assess safety and efficacy of metformin in treating cytopenia in children and adolescents with Fanconi Anemia.
- Trial name
- NCT06519786
- Testing
- Metformin
- Where
- 2 sites in Egypt
- Volunteers
- 30 people
- Sponsor
- Ain Shams University
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies
This phase II trial studies how well reduced intensity donor stem cell transplant works in treating patients with hematologic malignancies.
- Conditions
- Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT01384513
- Testing
- Fludarabine, Busulfan, Total Body Irradiation (TBI)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Bone Marrow Failure Syndromes, Erythrocyte Disorder
Investigation of the Genetics of Hematologic Diseases
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases.
- Conditions
- Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis
- Trial name
- NCT02720679
- Where
- United States
- Volunteers
- 1,716 people
- Sponsor
- St. Jude Children's Research Hospital
Anemia, Chronic Kidney Disease
Efficacy and Safety of Peginesatide (AF37702) in the Treatment of Anemia in Participants With Chronic Kidney Disease
The purpose of this study is to evaluate the ability of peginesatide (AF37702) to increase and maintain increased hemoglobin levels in participants with chronic kidney disease (CKD) (either not on dialysis, receiving regular…
- Conditions
- Anemia, Chronic Kidney Disease, Chronic Renal Failure, Pure Red Cell Aplasia
- Trial name
- NCT00314795
- Testing
- Peginesatide
- Where
- 4 sites in France, Germany and 1 more
- Volunteers
- 22 people
- Sponsor
- Takeda
Diamond Blackfan Anemia
Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia
This study will provide information needed to develop more effective treatments for patients with Diamond-Blackfan anemia (DBA). Current treatments include steroids, such as prednisone, and blood transfusions.
- Trial name
- NCT00011505
- Testing
- G-CSF, Leukapheresis
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)