Clinical trials: Inherited Bone Marrow Failure Syndromes
Hematologic Malignancies, Nonmalignant Diseases
Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)
Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609814
- Testing
- Clofarabine, Fludarabine Injection
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, San Francisco
Fanconi Anemia
Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy Participants
RATIONALE: Studying samples of bone marrow from patients with Fanconi anemia and from healthy participants in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to Fanconi…
- Trial name
- NCT00896740
- Testing
- microarray analysis, biopsy
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- OHSU Knight Cancer Institute
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
Childhood Langerhans Cell Histiocytosis, Fanconi Anemia
Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases
RATIONALE: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy.
- Conditions
- Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma
- Trial name
- NCT00084695
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Milton S. Hershey Medical Center
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
Graft Versus Host Disease
Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant
This trial will see if extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will prevent acute and chronic graft-versus-host disease (GVHD) in children and adolescents…
- Trial name
- ASCENT (NCT03924401)
- Testing
- Abatacept
- Where
- 9 sites in United States
- Volunteers
- 30 people
- Sponsor
- Emory University
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Haematological Malignancy, Acute Leukemia
A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts
To date, allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes, haemoglobinopathies…
- Conditions
- Haematological Malignancy, Acute Leukemia, Myelodysplastic Syndromes, Hemoglobinopathy in Children
- Trial name
- EVAADE (NCT04331483)
- Testing
- Physical activity program
- Where
- France
- Sponsor
- Centre Leon Berard
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's Anemia
Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Fanconi's Anemia
OBJECTIVES: I. Determine the effectiveness of moderate dose cyclophosphamide and total lymphoid radiotherapy in terms of improving the survival and reducing the morbidity following allogeneic bone marrow transplantation in…
- Trial name
- NCT00005891
- Testing
- cyclophosphamide, Allogeneic Bone Marrow Transplantation
- Where
- United States
- Sponsor
- Fairview University Medical Center
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
Alcohol-Related Carcinoma, Fanconi Anemia
Role of Acetaldehyde in the Development of Oral Cancer
This is a minimal risk intervention study where healthy volunteers and individuals with Fanconi anemia will consume a single dose of alcohol and provide primarily non-invasive biological samples at various time points.
- Conditions
- Alcohol-Related Carcinoma, Fanconi Anemia, Oral Cavity Carcinoma
- Trial name
- NCT05973656
- Testing
- Alcohol, Biospecimen Collection, Breath Test
- Where
- United States
- Volunteers
- 170 people, healthy volunteers welcome
- Sponsor
- Masonic Cancer Center, University of Minnesota
Transfusional Iron Overload, Transfusional Hemosiderosis
Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
This is a clinical research study in patients who have iron overload in the heart due to chronic blood transfusions. The study will have 2 treatment groups and will compare the safety and efficacy of chelation therapy with a…
- Trial name
- CORDELIA (NCT00600938)
- Testing
- Core Study: Deferasirox, Core Study: Deferoxamine, Extension: deferoxamine to deferasirox
- Where
- 21 sites in Canada, China and 9 more
- Volunteers
- 197 people
- Sponsor
- Novartis Pharmaceuticals
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia
Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission
RATIONALE: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Fanconi Anemia, Leukemia
- Trial name
- NCT00305708
- Testing
- anti-thymocyte globulin, busulfan, fludarabine phosphate
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of California, San Francisco
Sickle Cell Disease, Thalassemia
Multi-Center Study of Iron Overload: Pilot Study
The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions…
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- MCSIO (NCT01114776)
- Where
- 3 sites in United States, Germany and 1 more
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Fanconi's Anemia, Pancytopenia
Gene Therapy for the Treatment of Fanconi's Anemia Type C
Fanconi's Anemia is an inherited disorder that can produce bone marrow failure. In addition, some patients with Fanconi's anemia have physical defects usually involving the skeleton and kidneys.
- Trial name
- NCT00001399
- Testing
- Transduced CD34+ Cells
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum
Hereditary Diseases, Acquired Aplastic Anemia
Cord Blood Transplantation in Severe Aplastic Anemia
This is a Prospective Phase II Study to evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse after Immunosuppressive Therapy in the absence of an HLA identical donor;
- Conditions
- Hereditary Diseases, Acquired Aplastic Anemia, Relapse, Absence of an HLA Identical Donor
- Trial name
- APCORD (NCT01343953)
- Testing
- Cord Blood Transplantation
- Where
- France
- Volunteers
- 26 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood…
- Trial name
- NCT06090669
- Testing
- TruSight Oncology, imatinib
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Cancer
Donor Natural Killer Cell Infusion in Preventing Relapse or Graft Failure in Patients Who Have Undergone Donor Bone Marrow Transplant
RATIONALE: Giving donor natural killer cells to patients who have undergone donor bone marrow transplant may make the transplant work better and keep cancer cells from coming back.
- Trial name
- NCT00569283
- Testing
- therapeutic allogeneic lymphocytes
- Where
- 2 sites in South Korea
- Volunteers
- 18 people
- Sponsor
- Asan Medical Center