Clinical trials: Bone Marrow Failure Syndrome
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.
Myelodysplastic Syndromes
Trial of Volasertib With or Without Azacitidine in Patients With Myelodysplastic Syndromes
The objectives of this trial are to evaluate the safety, tolerability, maximum tolerated dose (MTD), pharmacokinetics and preliminary efficacy of volasertib in two dosing schedules of intravenous volasertib as monotherapy or in…
- Trial name
- NCT02721875
- Testing
- Volasertib, Azacitidine
- Where
- Japan
- Volunteers
- 1 people
- Sponsor
- Boehringer Ingelheim
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
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
Aplastic Anemia, Drug Effect
A Prospective, Randomized, Controlled Study of rhTPO in Combination With Herombopag + CsA vs Herombopag + CsA for the Treatment of Primary TD-NSAA
Aplastic anemia (AA) is a group of clinical syndromes. Treatment options are very limited. The results of a previous clinical study showed good efficacy and a high safety profile of herombopag in improving thrombocytopenia, but…
- Trial name
- NCT06004791
- Testing
- Herombopag + CsA, rhTPO combined with Herombopag + CsA
- Where
- China
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Peking Union Medical College Hospital
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
Immunosuppresion, Thrombocytopenia
Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia
Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and a hypocellular bone marrow.
- Conditions
- Immunosuppresion, Thrombocytopenia, Pancytopenia, Neutropenia
- Trial name
- NCT00260689
- Testing
- Anti-thymocyte globulin (rabbit), Anti-thymocyte globulin (horse), Cyclosporine
- Where
- United States
- Volunteers
- 136 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Severe Aplastic Anemia
A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)
Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant.
- Trial name
- TransIT (NCT05600426)
- Testing
- cyclosporine, Matched Unrelated Donor Hematopoetic Stem Cell Transplant, horse anti-thymocyte globulin (ATG)
- Where
- 52 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- Boston Children's Hospital
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
Efficacy and Safety of Avatrombopag Combined With IST for the Treatment of HAAA and SAA With Abnormal Liver Function
This is a multicenter, single-arm clinical study. The objective was to evaluate the efficacy and safety of Avatrombopag combined with IST in very/sever aplastic anemia patients with abnormal liver function or HAAA patients…
- Trial name
- NCT05571332
- Testing
- Avatrombopag 20 MG Oral Tablet
- Where
- China
- Volunteers
- 39 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
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
Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure Syndromes
Examine red and white blood cells of PNH patients with bone marrow failure syndromes.
- Trial name
- EXPLORE (NCT01192425)
- Volunteers
- 5,580 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acute Myelogenous Leukemia, Lymphoid Leukemia
T-Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Hematologic Malignancies
Objectives: 1. To evaluate disease free survival after Campath 1H-based in vivo T-cell depletion and non-myelo-ablative ablative stem cell transplantation in patients with hematologic malignancies. 2.
- Conditions
- Acute Myelogenous Leukemia, Lymphoid Leukemia, Chronic Myelogenous Leukemia, Malignant Lymphoma
- Trial name
- NCT00683046
- Testing
- Fludarabine, Melphalan, Stem cells
- Where
- United States
- Volunteers
- 204 people
- Sponsor
- University of Chicago
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
Leukemia, Aplastic Anemia
Methadone as an Alternative Treatment for Children Underdoing HSCT
Mucositis is a normal side effect of stem cell transplant which happens as a result of chemotherapy being given prior to a new donor cell infusion (bone marrow transplant).
- Conditions
- Leukemia, Aplastic Anemia, Bone Marrow Failure Syndromes, SCID
- Trial name
- MATCH (NCT06940570)
- Testing
- PCA plus methadone (Experimental), PCA Alone (SOC)
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University of Texas Southwestern Medical Center
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