Clinical trials: Aplasia
Aplastic Anemia, Bone Marrow Failure Syndromes
Nandrolone Decanoate in the Treatment of Telomeropathies
Decrease in blood cell counts due to deficient bone marrow function, called bone marrow failure, as well as some lung diseases, called idiopathic pulmonary fibrosis, can be caused by genetic defects in telomere biology genes…
- Conditions
- Aplastic Anemia, Bone Marrow Failure Syndromes, Idiopathic Pulmonary Fibrosis, Telomere Shortening
- Trial name
- NCT02055456
- Testing
- Nandrolone Decanoate
- Where
- Brazil
- Volunteers
- 20 people
- Sponsor
- University of Sao Paulo
Anemia, Aplastic, Fanconi Anemia
Cord Blood Stem Cell Transplantation Study (COBLT)
To evaluate if HLA-mismatched, unrelated-donor umbilical cord blood stem and progenitor cell units (UCBU) offered a clinically acceptable alternative to matched unrelated-donor allogeneic bone marrow for transplantation with…
- Conditions
- Anemia, Aplastic, Fanconi Anemia, Hematologic Diseases, Leukemia
- Trial name
- NCT00000603
- Testing
- stem cell transplantation
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Severe Aplastic Anemia (SAA)
REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
This study is researching an experimental drug called REGN7257 (called "study drug"). The study is focused on patients who have severe aplastic anemia (SAA), a disease of the bone marrow resulting in an impairment of the…
- Trial name
- NCT04409080
- Testing
- REGN7257
- Where
- 10 sites in United States, France and 2 more
- Volunteers
- 17 people
- Sponsor
- Regeneron Pharmaceuticals
RPS19 Deficient Diamond-Blackfan Anemia
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children.
- Trial name
- NCT07476183
- Testing
- APR-2020
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Apriligen, Inc.
Dyskeratosis Congenita, Fanconi Anemia
Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment
RATIONALE: Studying biopsy, bone marrow, and blood samples from patients with cytopenia that did not respond to treatment may help doctors learn more about the disease and plan the best treatment.
- Conditions
- Dyskeratosis Congenita, Fanconi Anemia, Myelodysplastic Syndromes, Pearson Marrow-pancreas Syndrome
- Trial name
- NCT00499070
- Testing
- polymerase chain reaction, flow cytometry, immunologic technique
- Where
- 10 sites in Austria, Belgium and 8 more
- Volunteers
- 119 people
- Sponsor
- University Hospital Freiburg
Severe Aplastic Anemia, Refractory, Severe Aplastic Anemia, Relapse
Alemtuzumab to Treat Severe Aplastic Anemia
This study will evaluate the safety and usefulness of a new immunosuppressive drug, alemtuzumab (Campath ), in patients with severe aplastic anemia (SAA).
- Trial name
- NCT00195624
- Testing
- Alemtuzumab (Campath ), Cyclosporine
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Treatment-naive Severe Aplastic Anemia
Extension Study of Hetrombopag in Severe Aplastic Anemia
This is a multicenter, placebo-control, phase 3 study of hetrombopag in patients with treatment-naive severe aplastic anemia. All subjects who have completed or withdrawn from the HR-TPO-SAA-III study will voluntarily participate…
- Trial name
- NCT04961710
- Testing
- Hetrombopag Olamine
- Where
- China
- Volunteers
- 157 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Fanconi Anemia, Severe Aplastic Anemia
Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia
The purpose of this study is to discover whether children and adults with Fanconi anemia (FA) can be safely and effectively transplanted with Human Leukocyte Antigen (HLA) mismatched (up to one haplotype), HLA-matched sibling, or…
- Trial name
- Mafia (NCT00590460)
- Testing
- CAMPATH-1H, Anti-CD45, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Baylor College of Medicine
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
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
Telomere Disease
Low-Dose Danazol for the Treatment of Telomere Related Diseases
Background: DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA.
- Trial name
- NCT03312400
- Testing
- Danazol
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplasia Cutis Congenita
Genetic and Functional Analysis of Aplasia Cutis Congenital (ACC)
The goal of this research study is to identify genes and regulatory elements on chromosomes that cause ACC. The investigators also study tissue samples from patients to learn about the processes that lead to this disorder.
- Trial name
- ACC (NCT01630421)
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- UConn Health
Mucopolysaccharidosis I, Mucopolysaccharidosis VI
Human Placental-Derived Stem Cell Transplantation
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease
- Trial name
- HPDSC (NCT01586455)
- Testing
- Human Placental Derived Stem Cell
- Where
- 3 sites in United States
- Volunteers
- 43 people
- Sponsor
- New York Medical College
Leukemia
Alemtuzumab in Myelodysplastic Syndrome (MDS), Aplastic Anemia, and T-Cell Large Granular Lymphocytic Leukemia (T-GL)
The goal of this clinical research study is to determine the effectiveness of alemtuzumab in patients with aplastic anemia, MDS, or T-Cell large granular lymphocytic leukemia. The safety of alemtuzumab will also be studied.
- Trial name
- NCT01191749
- Testing
- Alemtuzumab
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- M.D. Anderson Cancer Center
Holoprosencephaly
Retrospective Study Using Next Generation Sequencing (NGS) on Biological Samples to Improve Genetic Counseling for Patients With Previously Explored Craniofacial Midline Defects.
Holoprosencephaly, or HPE, is the most common congenital cerebral malformation in humans and the most severe of a group of pathologies related to a deficiency of the SHH signalling pathway (Sonic Hedgehog SHH-D).
- Trial name
- EXOMEDIANE (NCT04691414)
- Testing
- NGS
- Where
- France
- Volunteers
- 33 people
- Sponsor
- Rennes University Hospital
Anemia, Aplastic, Red-Cell Aplasia, Pure
Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia
This study will test whether the immune-suppressing drug rituximab can increase blood counts and reduce the need for transfusions in patients with moderate aplastic anemia, pure red cell aplasia, or Diamond Blackfan anemia.
- Conditions
- Anemia, Aplastic, Red-Cell Aplasia, Pure, Anemia, Diamond-Blackfan
- Trial name
- NCT00229619
- Testing
- Rituximab
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Severe Aplastic Anemia, Telomere Biology Disorders
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Background: Bone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the diseases and treatments are lacking.
- Conditions
- Severe Aplastic Anemia, Telomere Biology Disorders, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT05012111
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Bone Marrow Failure Syndromes, Anemia, Aplastic
Identifying Characteristics of Bone Marrow Failure Syndromes
Bone marrow failure syndromes (BMFS) are rare disorders characterized by dysfunctional hematopoietic stem cells, which give rise to all red and white blood cells.
- Conditions
- Bone Marrow Failure Syndromes, Anemia, Aplastic, Myelodysplastic Syndromes, Hemoglobinuria, Paroxysmal
- Trial name
- NCT00315419
- Where
- 4 sites in United States
- Volunteers
- 450 people
- Sponsor
- Office of Rare Diseases (ORD)
Aplastic Anemia
CK0801 for Treatment of Aplastic Anemia
This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care…
- Trial name
- NCT07499102
- Testing
- CK0801
- Volunteers
- 12 people
- Sponsor
- Cellenkos, Inc.
Anemia, Aplastic
Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia
Aplastic anemia is a rare autoimmune disorder in which the bone marrow production of blood cells is greatly decreased or absent.
- Trial name
- NCT00319878
- Testing
- Sirolimus, Cyclosporine
- Where
- 4 sites in United States
- Volunteers
- 52 people
- Sponsor
- Office of Rare Diseases (ORD)