Clinical trials: Bone Marrow Failure Syndromes
Hemoglobin, Stem Cell Transplant
Fertility Preservation in Women Who Will Have Gonadotoxic Therapy or Hematopoetic Stem Cell Transplantation, and in Women With Sickle Cell Disease
Background: Some treatments for cancer or other diseases can lead to infertility in women. These treatments include chemotherapy, some stem cell transplants, and pelvic radiotherapy. They are called gonadotoxic therapies.
- Conditions
- Hemoglobin, Stem Cell Transplant, Aplastic Anemia
- Trial name
- NCT02225145
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Haematological Malignancy, Acute Leukemia
Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation
Allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes haemoglobinopathies, bone…
- Conditions
- Haematological Malignancy, Acute Leukemia, Myelodysplastic Syndromes, Hemoglobinopathy in Children
- Trial name
- EVAADE (NCT05298930)
- Testing
- Physical activity program
- Where
- France
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Centre Leon Berard
Fanconi Anemia
Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
RATIONALE: Giving chemotherapy, such as cyclophosphamide and fludarabine, before a donor stem cell transplant helps to remove the patient's cells to allow for the transplant cells to take and grow.
- Trial name
- NCT00630253
- Testing
- Anti-Thymocyte Globulin, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Anemia, Aplastic
Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Medical Claims Database Study
The main goal of this study was to investigate the effectiveness and safety of eltrombopag (ETB) when compared to other treatments in Japanese aplastic anemia (AA) patients using data from the Medical Data Vision (MDV)…
- Trial name
- NCT06991894
- Where
- United States
- Volunteers
- 2,517 people
- Sponsor
- Novartis Pharmaceuticals
Aplastic Anemia
Mesenchymal Stem Cells Transplantation to Patients With Relapsed/Refractory Aplastic Anemia.
The study is a phase I/II trial designed to establish the safety and efficacy of intravenous administration of bone marrow derived mesenchymal stem cells from related donor to patients with relapsed/refractory aplastic anemia.
- Trial name
- MSC (NCT01305694)
- Testing
- bone marrow derived mesenchymal stem cells
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Guangzhou General Hospital of Guangzhou Military Command
Acute Leukemias, Aplastic Anemia
Safety Study of CD3/CD19 Depleted Haploidentical Stem Cells
Feasibility and toxicity of haploidentical transplantation of CD3/CD19 depleted stem cells in combination with a toxicity reduced conditioning regimen or with standard conditioning regimens according to underlying disease.
- Conditions
- Acute Leukemias, Aplastic Anemia, Childhood Solid Tumor
- Trial name
- NCT01919866
- Testing
- Transplantation of CD3/CD19 depleted stem cells
- Where
- 5 sites in Austria, Germany
- Volunteers
- 120 people
- Sponsor
- University Children's Hospital Tuebingen
Aplastic Anemia
Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia
To assess the tolerability and effectiveness of rabbit antithymocyte globulin (ATG, Thymoglobuline) with ciclosporin in the first line treatment of patients with acquired severe aplastic anaemia, and patients with non-severe…
- Trial name
- RATGAA07 (NCT00471848)
- Testing
- rabbit antithymocyte globulin
- Where
- 14 sites in France, Germany and 4 more
- Volunteers
- 35 people
- Sponsor
- European Society for Blood and Marrow Transplantation
Thrombocytopenia, Metachromatic Leukodystrophy
Stem Cell Transplantation (SCT) for Genetic Diseases
OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II.
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major
- Trial name
- NCT00004378
- Testing
- Stem Cell Transplantation
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Aplastic Anemia
CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia
This is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD7 CAR-T Bridging to allo-HSCT therapy for patients with severe aplastic anemia
- Trial name
- NCT06633328
- Testing
- CD7 CAR-T cells injection, Allogeneic hematopoietic stem cell transplantation
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Zhejiang University
Congenital Amegakaryocytic Thrombocytopenia, Leukemia
Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease
RATIONALE: Giving chemotherapy and total body irradiation before a donor bone marrow transplant or peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
- Trial name
- NCT00295971
- Testing
- anti-thymocyte globulin, therapeutic allogeneic lymphocytes, fludarabine phosphate
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- University of California, San Francisco
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Fungal Infections
Genetic Variation and Variability in Posaconazole Pharmacokinetics in Children
The main goal of this study is to see how the body breaks down an antifungal drug named posaconazole in children with certain cancers, blood disorders, or transplantation of bone marrow or similar blood cells.
- Trial name
- NCT02358499
- Testing
- Posaconazole
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Children's Mercy Hospital Kansas City
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
Aplastic Anemias, Hemoglobinopathies
The HOPE Biobank Resource (BMT CTN 2402 HOPE)
A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood…
- Conditions
- Aplastic Anemias, Hemoglobinopathies, Bone Marrow Failure
- Trial name
- HOPE Resource (NCT07227155)
- Where
- 14 sites in United States
- Volunteers
- 375 people, healthy volunteers welcome
- Sponsor
- Medical College of Wisconsin
Telomere Shortening, Bone Marrow Failure
Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
This is a Phase I/II, open label, single center study to assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders.
- Trial name
- NCT04211714
- Testing
- EXG34217
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Elixirgen Therapeutics, Inc.
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)
Aplastic Anaemia
Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA Receiving sUCBT
To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of Aplastic Anemia (AA)
- Trial name
- NCT07885982
- Testing
- Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®)
- Where
- China
- Volunteers
- 59 people
- Sponsor
- Anhui Provincial Hospital
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
Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA)
Anti-thymocyte globulin (ATG) has been used in severe aplastic anemia as a part of the conditioning regimen. Among the many kinds of ATG preparations, thymoglobulin had been found to be more effective in preventing GVHD and…
- Trial name
- NCT00737685
- Testing
- cyclophosphamide, fludarabine , thymoglobulin
- Where
- South Korea
- Volunteers
- 30 people
- Sponsor
- The Korean Society of Pediatric Hematology Oncology
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