Clinical trials: Paroxysmal Hemoglobinuria, Nocturnal
Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML)
Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies
Blood and marrow stem cell transplant has improved the outcome for patients with high-risk hematologic malignancies. However, most patients do not have an appropriate HLA (immune type) matched sibling donor available and/or are…
- Conditions
- Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML), Leukemia, Myeloid, Chronic(CML), Juvenile Myelomonocytic Leukemia(JMML)
- Trial name
- NCT00186823
- Testing
- Miltenyi Biotec CliniMACS, Stem Cell Transplantation, TBI, systemic chemotherapy and antibodies as follows:
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- St. Jude Children's Research Hospital
Extrahepatic Portal Vein Obstruction (EHPVO), Thrombophilia
Hematological Disorders in EHPVO Patients
This study focuses on patients who have a condition called extrahepatic portal vein obstruction (EHPVO), where a blood clot blocks the portal vein outside the liver.
- Conditions
- Extrahepatic Portal Vein Obstruction (EHPVO), Thrombophilia, Myeloproliferative Neoplasms (MPN), Antiphospholipid Syndrome (APS)
- Trial name
- EHPVO-HEM (NCT07108023)
- Testing
- No intervention (Observational Study)
- Volunteers
- 115 people
- Sponsor
- Rahab Nady
Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis
Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This was a Phase 2, open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of iptacopan when administered in addition to Standard of care (SoC) in patients with paroxysmal…
- Trial name
- NCT03439839
- Testing
- iptacopan, Standard of Care
- Where
- 3 sites in France, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria, PNH
To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.
Phase I Study Evaluating Safety, Tolerability, PK and PD of Single and Multiple Doses of TUL321 Capsule in Healthy Participants and Food Effect on PK
- Trial name
- NCT07760467
- Testing
- Part1: TUL321, Part2: TUL321
- Where
- China
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Zhuhai United Laboratories Co., Ltd.
Paroxysmal Nocturnal Haemoglobinuria
Study of Global Coagulation Tests in Patients With Paroxysmal Nocturnal Haemoglobinuria
Paroxysmal nocturnal haemaoglobinuria (PNH) is a rare disease which results in breakdown of the red blood cells and bone marrow failure. It is associated with an increased risk of blood clots.
- Trial name
- NCT01020188
- Where
- United Kingdom
- Volunteers
- 60 people
- Sponsor
- King's College Hospital NHS Trust
Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Basiliximab #2: In-Vivo Activated T-Cell Depletion to Prevent Graft-Versus_Host Disease (GVHD) After Nonmyeloablative Allotransplantation for the Treatment of Blood Cancer
The purpose of this study is to compare the effects (good and bad) of the medication basiliximab in combination with cyclosporine (investigational therapy) for the prevention of a complication of bone marrow transplantation known…
- Conditions
- Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Chronic Lymphocytic Leukemia
- Trial name
- NCT00975975
- Testing
- Basiliximab
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Indiana University
Hemoglobinuria, Paroxysmal
The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment Protocol
The primary objective is to provide access to eculizumab for PNH patient pending commercial availability.
- Trial name
- EMBRACE (NCT00438789)
- Testing
- eculizumab
- Where
- United States
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, Embolic Stroke of Undetermined Source
Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal hematological disorder, which can cause arterial or venous thrombosis.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, Embolic Stroke of Undetermined Source, Transient Ischemic Attack, Cerebral Vein Thrombosis
- Trial name
- NCT03329365
- Where
- Canada
- Volunteers
- 200 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Paroxysmal Nocturnal Hemoglobinuria
Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 administered intravenously to adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
- Trial name
- NCT05646524
- Testing
- NM8074
- Volunteers
- 12 people
- Sponsor
- NovelMed Therapeutics
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT02179359
- Testing
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
PNH
Pegcetacoplan Long Term Safety and Efficacy Extension Study
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
- Trial name
- NCT03531255
- Testing
- Pegcetacoplan
- Where
- 62 sites in United States, Australia and 19 more
- Volunteers
- 137 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.
An open label study to evaluate the efficacy and safety of FXS6837 capsules compared to eculizumab in naive PNH patients. About 90 PNH patients who are naive to complement inhibitor therapies will be enrolled to take FXS6837…
- Trial name
- NCT07747090
- Testing
- FXS6837 Capsule, Eculizumab infusion
- Where
- China
- Volunteers
- 90 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
- Trial name
- NCT01374360
- Where
- United States
- Volunteers
- 6,061 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with…
- Conditions
- Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia
- Trial name
- NCT04965597
- Testing
- Treosulfan, Fludarabine Phosphate, Tacrolimus
- Where
- 24 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
Paroxysmal Nocturnal Hemoglobinuria, PNH
A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This study was designed to evaluate the long-term safety of daily oral treatment with BCX9930 in participants who had participated in a previous BCX9930 trial for PNH and showed a benefit of treatment as determined by the…
- Trial name
- NCT04702568
- Testing
- BCX9930, Eculizumab, Ravulizumab
- Where
- 5 sites in Austria, South Africa and 1 more
- Volunteers
- 19 people
- Sponsor
- BioCryst Pharmaceuticals
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid Cancer
Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT00012376
- Testing
- bryostatin 1, sargramostim, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Cancer Institute (NCI)
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems.
- Conditions
- Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Trial name
- NCT03520647
- Testing
- Cyclophosphamide, Peripheral Blood Stem Cells
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Paroxysmal Nocturnal Hemoglobinuria
Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.
- Trial name
- NCT01192399
- Testing
- Eculizumab
- Volunteers
- 29 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
PNH - Paroxysmal Nocturnal Hemoglobinuria
Study of SGB-9768 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, randomized, open-Label Phase II study to evaluate the efficacy and safety of multiple doses of SGB-9768 in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT07796256
- Testing
- SGB-9768
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Suzhou Sanegene Bio Inc.
Leukemia, Myelodysplastic Syndromes
Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome
RATIONALE: Giving low doses of chemotherapy and antithymocyte globulin before a donor stem cell transplant helps stop the growth of abnormal cells.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Nonmalignant Neoplasm, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT00731328
- Testing
- nonmyeloablative allogeneic hematopoietic stem cell transplantation
- Where
- South Korea
- Volunteers
- 15 people
- Sponsor
- Asan Medical Center