Clinical trials: Aplasia
Anemia, Diamond-Blackfan
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Background: Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work.
- Trial name
- NCT04269889
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission
Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This clinical trial studies the use of reduced intensity chemotherapy and radiation therapy before donor stem cell transplant in treating patients with hematologic malignancies.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, Aplastic Anemia, Chronic Myelomonocytic Leukemia
- Trial name
- NCT02566304
- Testing
- Fludarabine, Total-Body Irradiation, T Cell-Depleted Donor Lymphocyte Infusion
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Thrombocytopenia, Hematologic Diseases
Thrombosomes® in Bleeding Thrombocytopenic Patients
The study evaluates the safety and potential early signals of efficacy of allogeneic Thrombosomes in bleeding thrombocytopenic patients
- Conditions
- Thrombocytopenia, Hematologic Diseases, Bone Marrow Aplasia
- Trial name
- NCT03394755
- Testing
- Thrombosomes
- Where
- 7 sites in United States, Norway
- Volunteers
- 24 people
- Sponsor
- Cellphire Therapeutics, Inc.
Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated and very rare diseases. Therefore, little data about clinical characteristics, especially the variety of symptoms in the course of the respective…
- Trial name
- ePRO-AA-PNH (NCT04128943)
- Testing
- symptom questionnaire, recording of vital signs, European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire
- Where
- Switzerland
- Volunteers
- 9 people
- Sponsor
- University Hospital, Basel, Switzerland
Congenital Heart Disease
Genetic Analysis of Left-Right Axis Formations
The objective of these studies is to identify genetic factors that contribute to the pathogenesis of complex congenital heart disease and other more rare conditions resulting from disturbances in organ positioning.
- Trial name
- NCT00341133
- Where
- United States
- Volunteers
- 900 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Nonmyeloablative Allogeneic Transplant
Allogeneic transplant from a matched sibling for the treatment of a variety of illnesses including bone marrow failure states, leukemias, myelodysplastic or myeloproliferative syndromes, lymphoma, or myeloma using a…
- Conditions
- Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
- Trial name
- Mini-allo (NCT01272817)
- Testing
- Nonmyeloablative Allogeneic Transplant
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Scripps Health
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
Aplastic Anemia
Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA)
Severe aplastic anemia is a fatal disease and patients without HLA matched siblings need alternative treatment option. Cord blood transplantation (CBT) has become an alternative treatment means in various diseases, but it has not…
- Trial name
- NCT00881933
- Testing
- Cyclophosphosphamide, TBI
- Where
- South Korea
- Volunteers
- 5 people
- Sponsor
- The Korean Society of Pediatric Hematology Oncology
Severe Aplastic Anemia
RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial
After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study
- Trial name
- RACE2 (NCT05049668)
- Testing
- ATGAM plus CsA with or without Eltrombopag
- Where
- France
- Volunteers
- 197 people
- Sponsor
- European Society for Blood and Marrow Transplantation
Severe Aplastic Anemia
Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy
Background: Severe aplastic anemia (SAA) is a form of bone marrow failure. It usually results from a cytotoxic T cell attack on the marrow stem cell. Two treatments can be used for most people with SAA.
- Trial name
- NCT04436380
- Where
- United States
- Volunteers
- 173 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Septo-Optic Dysplasia, Agenesis of Corpus Callosum
Sleep Profile of Patients With Septo-optic Dysplasia
The aim of this study is to evaluate sleep of patients with septo-optic dysplasia compared to patients with an isolated disorder of peripheral visual system and patients with corpus callosum agenesis since both visus defict and…
- Conditions
- Septo-Optic Dysplasia, Agenesis of Corpus Callosum, Blindness
- Trial name
- NCT06262152
- Testing
- Actigraphy, blood and salivary sample, and sleep evaluation
- Where
- Italy
- Volunteers
- 45 people
- Sponsor
- IRCCS National Neurological Institute "C. Mondino" Foundation
Aplastic Anemia
Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia
RATIONALE: Immunosuppressive therapies, such as anti-thymocyte globulin and cyclosporine, may improve bone marrow function and increase blood cell counts.
- Trial name
- NCT01231841
- Testing
- cyclosporine, anti-thymocyte globulin
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- The Cleveland Clinic
Intellectual Deficiency, Cerebral Abnormality
Corpus Callosum Agenesis and Intellectual Disability
Corpus callosum agenesis or dysgenesis (CCA) is a major brain malformation (˜1/4000 births) presently diagnosed by prenatal ultrasonography.
- Trial name
- ACCREM (NCT02167568)
- Testing
- Genetic
- Where
- France
- Volunteers
- 360 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hematologic Neoplasms, Anemia, Aplastic
Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors
The purpose of this study is to transplant haploidentical related peripheral blood stem cells (PBSCs) that come from a relative such as a parent, sibling, a child or other relative who has a half-matched tissue type with the…
- Conditions
- Hematologic Neoplasms, Anemia, Aplastic, Hemoglobinuria, Paroxysmal, Multiple Myeloma
- Trial name
- NCT00618969
- Testing
- haploidentical allogeneic PBSC transp
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- University of Arizona
Aplastic Anemia
UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia
This Phase II open-label interventional clinical trial aims to evaluate the efficacy of romiplostim, in patients with severe aplastic anemia (SAA), both treatment naïve and relapsed/refractory, in inducing trilineage…
- Trial name
- NCT07001254
- Testing
- Romiplostim, Immunosuppressive therapy (IST)
- Where
- United States
- Sponsor
- Anjali Sharathkumar
Fanconi Anemia
Unrelated HSCT in Patients With Fanconi Anemia
The protocol is designed for the compassionate treatment of patients with Fanconi Anemia who do not have an HLA-matched sibling donor.
- Trial name
- NCT02127905
- Testing
- CD34+ selected cells
- Where
- United States
- Sponsor
- Neena Kapoor, M.D.
Fanconi Anemia
A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia
This is a multi-center, Phase 1/2 study to determine the Optimal Biologic Dose (OBD) and to evaluate the safety, tolerability, PK, and preliminary activity of FP 045 when administered orally in young adult/adolescent and…
- Trial name
- NCT04522375
- Testing
- FP-045
- Sponsor
- Foresee Pharmaceuticals Co., Ltd.
Stem Cell Transplantation, Bone Marrow Transplantation
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
Many genetic diseases of lymphohematopoietic cells (such as sickle cell anemia, thalassemia, Diamond-Blackfan anemia, Combined Immune Deficiency (CID), Wiskott-Aldrich syndrome, chronic granulomatous disease, X-linked…
- Conditions
- Stem Cell Transplantation, Bone Marrow Transplantation, Peripheral Blood Stem Cell Transplantation, Allogeneic Transplantation
- Trial name
- NCT02512679
- Testing
- Cyclophosphamide Dose Level 1, Cyclophosphamide Dose Level 2, Cyclophosphamide Dose Level 3
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Los Angeles
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