Clinical trials: Anemia, Aplastic
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
Acute Leukemia, Immune Deficiency Disorder
Umbilical Cord Blood Transplantation From Unrelated Donors
This study is being done to determine how long it takes for the engraftment (recovery of blood cell counts) of umbilical cord stem cells and also how often engraftment of umbilical cord stem cells transplanted from an unrelated…
- Conditions
- Acute Leukemia, Immune Deficiency Disorder, Congenital Hematological Disorder, Metabolism Disorder
- Trial name
- NCT03016806
- Testing
- Total Body Irradiation 1200 cGy, Total Body Irradiation 200 cGy, Cyclophosphamide
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Rochester
Fanconi Anemia
Eltrombopag for Treatment of Fanconi Anemia
An open-label, phase II study to assess the efficacy and safety of eltrombopag for the treatment of children and adolescents with Fanconi anemia.
- Trial name
- NCT06045052
- Testing
- Eltrombopag
- Where
- Spain
- Volunteers
- 8 people
- Sponsor
- Julian Sevilla
Fanconi's Anemia
Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia
OBJECTIVES: I. Determine the probability of engraftment with total body irradiation, cyclophosphamide, fludarabine, and anti-thymocyte globulin followed by HLA nongenotypically identical donor, T-cell depleted hematopoietic cell…
- Trial name
- NCT00005898
- Testing
- anti-thymocyte globulin, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Minnesota
Fanconi's Anemia, Hematologic Disease
Medical Treatment for Diamond Blackfan Anemia
Diamond Blackfan anemia (DBA) is a condition in which the bone marrow is underdeveloped. DBA is considered a congenital disease, meaning patients are born with it.
- Trial name
- NCT00001749
- Testing
- Antithymocyte globulin, Cyclosporine
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Bone Marrow Failure, Osteopetrosis
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
This study proposes the use of a reduced intensity chemotherapy/radiation therapy regimen followed by stem cell transplantation, as compared to standard ablative chemotherapy regimens associated with stem cell transplantation, in…
- Conditions
- Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
- Trial name
- NCT01019876
- Testing
- Fludarabine, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Columbia University
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
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
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia.
- Trial name
- NCT01174108
- Testing
- Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
- Where
- 2 sites in United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
NAC for Hematopoietic Recovery in SAA
This is a prospective single-arm clinical study to evaluate the role of NAC among patients with severe aplastic anemia (SAA) can promote hematopoietic recovery after haploidentical transplantation.
- Trial name
- NCT06518044
- Testing
- N Acetyl L Cysteine
- Volunteers
- 30 people
- Sponsor
- Peking University People's Hospital
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
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
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
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 Syndrome
French National Registry of Bone Marrow Failures
This is a unique clinical and biological database that collects standardized clinical information during the management of all patients with bone marrow failure syndromes (BMF) in France (multicenter registry), from diagnosis and…
- Trial name
- RIME (NCT04781790)
- Testing
- Bone Marrow Failure
- Where
- France
- Volunteers
- 5,000 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA)
Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders
The purpose of this study is to study the efficacy of Traditional Chinese Medicine (TCM) on anaemic and cytopenic haematological disorders including myelodysplastic syndrome (MDS), aplastic anaemia (AA), myelofibrosis (MF) and…
- Conditions
- Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA), Myelofibrosis (MF), Thalassemia Intermedia
- Trial name
- NCT01224496
- Testing
- Chinese herbal concoction twice a day for 6 months
- Where
- Singapore
- Volunteers
- 38 people
- Sponsor
- Singapore General Hospital
Fanconi's Anemia
Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
OBJECTIVES: I. Evaluate the toxicity of amifostine in patients with bone marrow failure related to Fanconi's anemia. II. Determine the efficacy of this treatment regimen in this patient population. III.
- Trial name
- NCT00006127
- Testing
- amifostine
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Dana-Farber Cancer Institute