Clinical trials: Hemoglobinuria, Paroxysmal
Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated and very rare diseases. Therefore, little data about clinical characteristics, especially the variety of symptoms in the course of the respective…
- Trial name
- ePRO-AA-PNH (NCT04128943)
- Testing
- symptom questionnaire, recording of vital signs, European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire
- Where
- Switzerland
- Volunteers
- 9 people
- Sponsor
- University Hospital, Basel, Switzerland
Paroxysmal Nocturnal Hemoglobinuria
Specified Drug-use Surveillance of Fabhalta Capsules
This is a multicenter, single-arm, non-interventional study (NIS) with a central registration system and an all-case surveillance system. The observation period is 48 weeks after the start of treatment with Fabhalta.
- Trial name
- NCT06606314
- Where
- 91 sites in Japan
- Volunteers
- 132 people
- Sponsor
- Novartis Pharmaceuticals
Hemoglobinuria, Paroxysmal
Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of this study was to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) parameter estimates of eculizumab to confirm the dose regimens for pediatric and adolescent participants with PNH.
- Trial name
- NCT00867932
- Testing
- Eculizumab
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acute Lymphoblastic Leukemias, Acute Myelocytic Leukemia
Stem Cell Transplantation for Patients With Hematologic Malignancies
Childhood leukemias which cannot be cured by chemotherapy alone may be effectively treated by allogeneic bone marrow transplantation.
- Conditions
- Acute Lymphoblastic Leukemias, Acute Myelocytic Leukemia, Chronic Myeloid Leukemia, Juvenile Myelomonocytic Leukemia
- Trial name
- NCT00152139
- Testing
- Allogeneic Stem Cell Transplantation, Chemotherapy and antibodies
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- St. Jude Children's Research Hospital
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patients with active PNH who are treatment-naive to complement inhibitor therapy or have not…
- Trial name
- NCT03946748
- Testing
- REGN3918
- Where
- 12 sites in Hong Kong, Hungary and 3 more
- Volunteers
- 24 people
- Sponsor
- Regeneron Pharmaceuticals
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
This clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00856388
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Roswell Park Cancer Institute
Allogeneic Stem Cell Transplant, Leukemia
Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders
This is a phase II, single-center study to evaluate the efficacy of a novel cytoreductive regimen followed by CD34+E- selected T cell depleted allogeneic stem cell (or soybean agglutinated and E-rosetted BM) transplant as…
- Conditions
- Allogeneic Stem Cell Transplant, Leukemia, Non-Hodgkins, Lymphoblastic Lymphoma
- Trial name
- NCT00587054
- Testing
- cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplant
- Where
- United States
- Volunteers
- 129 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Paroxysmal Nocturnal Hemoglobinuria
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH.
- Trial name
- COMPLETE (NCT05776472)
- Testing
- Pegcetacoplan
- Where
- 87 sites in Australia, Belgium and 13 more
- Volunteers
- 165 people
- Sponsor
- Swedish Orphan Biovitrum
Paroxysmal Nocturnal Hemoglobinuria
Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
This study will collect clinical response data on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as a specific therapeutic strategy as per ordinary clinical…
- Trial name
- NCT05274633
- Testing
- Ravulizumab
- Where
- 6 sites in Italy
- Volunteers
- 120 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
PNH - Paroxysmal Nocturnal Hemoglobinuria
Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in PNH Patients.
This is a multicenter, randomized, open-label, Phase II clinical study to evaluate the efficacy, safety, and pharmacokinetics of LP-005 injection in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT07212426
- Testing
- LP-005 Injection
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Longbio Pharma
Paroxysmal Nocturnal Hemoglobinuria
To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in healthy volunteers.
- Trial name
- NCT05490017
- Testing
- KP104
- Where
- Australia
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Kira Pharmacenticals (US), LLC.
Paroxysmal Nocturnal Hemoglobinuria
Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
The primary purpose of this study is to assess the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab in participants who are prescribed and are receiving a higher than approved dose of eculizumab to treat…
- Trial name
- NCT04320602
- Testing
- Eculizumab, Ravulizumab
- Where
- 4 sites in United Kingdom
- Volunteers
- 18 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
NTQ5082 capsules are a small molecule CFB inhibitor. This study is a multicenter, randomized, open-label, active-controlled Phase III clinical trial designed to evaluate the efficacy and safety of NTQ5082 capsules in the…
- Trial name
- NCT07177859
- Testing
- NTQ5082 capsules 200 mg, Eculizumab Injection
- Where
- China
- Volunteers
- 78 people
- Sponsor
- Nanjing Chia-tai Tianqing Pharmaceutical
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute