Clinical trials: Pancytopenia
Fanconi Anemia
Eltrombopag for Fanconi Anemia
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly.
- Trial name
- NCT03206086
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Pancytopenia
Clinical, Hematological and Pathological Profile of Pancytopenia
Describe clinical characteristic, peripheral blood parameters and bone marrow examination in pancytopenia patients and Analyze etiology of pancytopenia and study the outcomes regarding duration of hospital stay.
- Trial name
- NCT07785726
- Volunteers
- 140 people
- Sponsor
- Assiut University
Bone Marrow Failure Syndromes, Aplastic Anemias
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse
- Trial name
- BMF (NCT07712562)
- Testing
- Dibotatug (DR-01)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Dren Bio
Severe Aplastic Anemia
Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
Background: Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells.
- Trial name
- NCT01623167
- Testing
- Cohort 1: hATG, CsA, EPAG Day 14 to Month 6, Cohort 2: hATG, CsA, EPAG Day 14 to Month 3, Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6
- Where
- United States
- Volunteers
- 207 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Immune Thrombocytopenia, Autoimmune Hemolytic Anemia
Severe Immune Cytopenia Registry Www.Sic-reg.Org
Prospective registry study for children and young adults with severe immune cytopenias (persisting/chronic immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome) to improve the management, facilitate the…
- Conditions
- Immune Thrombocytopenia, Autoimmune Hemolytic Anemia, Evans Syndrome, Primary Immunodeficiency
- Trial name
- sic-reg (NCT03576742)
- Testing
- potential biomarkers
- Where
- Austria
- Volunteers
- 80 people
- Sponsor
- Medical University of Graz
Shwachman-Diamond Syndrome, SDS
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in the world - to share their experience living with SDS via a safe…
- Conditions
- Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia
- Trial name
- SDS-GPS (NCT06999954)
- Where
- United States
- Volunteers
- 8,000 people
- Sponsor
- Shwachman-Diamond Syndrome Alliance Inc
Inherited BMF Syndrome, Inherited Platelet Disorder
Diagnosis and Phenotype Characterisation Using Genomics in Patients With Inherited Bone Marrow Failure (IBMDx Study)
This project seeks to perform whole genome sequence (WGS) and whole transcriptome sequence (WTS) analysis on 350 patients with suspected inherited bone marrow failure syndromes and related disorder (IBMFS-RD) in order to increase…
- Conditions
- Inherited BMF Syndrome, Inherited Platelet Disorder, Hematologic Diseases
- Trial name
- IBMDx (NCT05196789)
- Testing
- whole genome and transcriptome sequencing
- Where
- Australia
- Volunteers
- 350 people
- Sponsor
- Peter MacCallum Cancer Centre, Australia
Pancytopenia
Spectrum of Hematological Disorders in Pediatrics
To determine the spectrum of pancytopenia with its frequency and etiology in children presenting to Assiut University Children Hospital during one year
- Trial name
- NCT05473650
- Testing
- CBC.with with differential Peripheral smear
- Volunteers
- 100 people
- Sponsor
- Assiut University
Aplastic Anemia, Neutropenia
A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia
Background: Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions.
- Conditions
- Aplastic Anemia, Neutropenia, Pancytopenia, Severe Aplastic Anemia
- Trial name
- NCT01187017
- Testing
- Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant
This protocol provides expanded access to bone marrow transplants for children who lack a histocompatible (tissue matched) stem cell or bone marrow donor when an alternative donor (unrelated donor or half-matched related donor)…
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT01200017
- Testing
- CD34+ enriched, T Cell Depleted donor stem cell product
- Where
- United States
- Sponsor
- Christopher Dvorak
Pancytopenia
Collection of Blood From Patients With Pancytopenia
Aplastic anemia is a condition in which the cells normally found in blood are greatly decreased. The normal levels of white blood cells, red blood cells, and platelets are much lower in patients with aplastic anemia.
- Trial name
- NCT00001214
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Autoimmune Pancytopenia, Autoimmune Lymphoproliferative Syndrome (ALPS)
Sirolimus for Autoimmune Disease of Blood Cells
Treatment for patients with autoimmune destruction of blood cells is poor. The part of the body that fights infections is called the immune system and white blood cells (WBCs) are part of the immune system.
- Conditions
- Autoimmune Pancytopenia, Autoimmune Lymphoproliferative Syndrome (ALPS), Evans Syndrome, Idiopathic Thrombocytopenic Purpura
- Trial name
- NCT00392951
- Testing
- sirolimus
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Children's Hospital of Philadelphia
Aplastic Anemia
Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects
This was an open label, non-randomized, phase II study of eltrombopag in combination with rabbit ATG/CsA in subjects with moderate or more severe AA who did not received prior ATG/ALG-based immunosuppressive therapy.
- Trial name
- NCT02404025
- Testing
- Eltrombopag, Rabbit ATG, CsA
- Where
- 11 sites in Japan
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
Cytopaenia
A Study to Assess the Safety and Efficacy of Eltrombopag in Japanese Subjects With Refractory, Moderate or More Severe Aplastic Anemia
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Japanese moderate or more severe aplastic anemia (AA) subjects with a platelet count <30,000/microliter who were refractory…
- Trial name
- NCT02148133
- Testing
- Eltrombopag 12.5 mg, Eltrombopag 25 mg
- Where
- 12 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Pancytopenia
Management of Childhood Pancytopenia
Pancytopenia is an important hematologic Problem.it is a decrease in all three cellular elements of peripheral blood leading to aneamia, leucopenia and thrombocytopenia.
- Trial name
- NCT03521947
- Testing
- Complete blood picture
- Volunteers
- 25 people
- Sponsor
- Assiut University
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)
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)
Aplastic Anemia, Neutropenia
Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia
Background: Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions.
- Conditions
- Aplastic Anemia, Neutropenia, Pancytopenia, Severe Aplastic Anemia
- Trial name
- NCT01193283
- Testing
- Cyclophosphamide, Cyclosporine
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
Comparison of Two Different Doses of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia
Acquired Aplastic anemia is one of the most frequent reason of bone marrow failure in East (Pakistan). The first treatment option is Allogenic Bone Marrow transplantation which is an expansive treatment option and also require a…
- Trial name
- ATG (NCT02028416)
- Testing
- ATG-fresenius
- Where
- Pakistan
- Volunteers
- 60 people
- Sponsor
- National Institute of Blood Disease Center, Pakistan
Pancytopenia Due to Chemotherapy
Effect of rhIL-11 in Patients With Thrombocytopenia for Childhood ALL
The goal of this clinical research study is to find out if rhIL-11（Interleukin 11） may increase the platelet count in Childhood patients with acute lymphocytic leukemia (ALL) who develop low platelet counts while receiving…
- Trial name
- NCT02314273
- Testing
- rhIL-11
- Where
- 5 sites in China
- Volunteers
- 120 people
- Sponsor
- Shanghai Children's Medical Center