Clinical trials: Paroxysmal Hemoglobinuria, Nocturnal
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.
- Trial name
- NCT03053102
- Testing
- Danicopan
- Where
- 5 sites in Italy, New Zealand and 2 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
This study was a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this Phase 3 study in PNH patients presenting with residual anemia despite treatment with anti-C5 antibody…
- Trial name
- APPLY-PNH (NCT04558918)
- Testing
- LNP023, Eculizumab, Ravulizumab
- Where
- 39 sites in United States, Brazil and 10 more
- Volunteers
- 97 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy
The primary objective of the study is to evaluate the safety and tolerability of pozelimab and cemdisiran combination therapy in participants with PNH who switch from eculizumab therapy The secondary objectives of the study are…
- Trial name
- NCT04888507
- Testing
- Pozelimab, Cemdisiran
- Where
- United Kingdom
- Volunteers
- 6 people
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria (PNH)
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who were clinically stable after having been treated with eculizumab for at least 6 months.
- Trial name
- NCT03056040
- Testing
- Ravulizumab, Eculizumab
- Where
- 51 sites in United States, Australia and 9 more
- Volunteers
- 202 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy
The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH participants with residual anemia despite treatment with a…
- Trial name
- REDEEM-1 (NCT05116774)
- Testing
- Eculizumab, Ravulizumab, BCX9930
- Where
- 7 sites in France, Hungary and 3 more
- Volunteers
- 12 people
- Sponsor
- BioCryst Pharmaceuticals
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders
RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00544115
- Testing
- busulfan, cyclophosphamide, cyclosporine
- Volunteers
- 260 people
- Sponsor
- City of Hope Medical 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
Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Chronic Myelomonocytic Leukemia
Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders
This phase II trial studies the side effects and best dose of total-body irradiation when given together with fludarabine phosphate followed by a donor peripheral stem cell transplant in treating patients with myelodysplastic…
- Conditions
- Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndrome, Essential Thrombocythemia
- Trial name
- NCT00397813
- Testing
- Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- 3 sites in United States
- Volunteers
- 77 people
- Sponsor
- Fred Hutchinson Cancer Center
Paroxysmal Nocturnal Hemoglobinuria
Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Evaluation of the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT03500549
- Testing
- Pegcetacoplan, Soliris
- Where
- 53 sites in United States, Australia and 9 more
- Volunteers
- 80 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
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
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study
The purpose of this study is to enable continued access to zilucoplan (RA101495) for patients with paroxysmal nocturnal hemoglobinuria (PNH) after they complete a zilucoplan clinical study.
- Trial name
- NCT03225287
- Testing
- Zilucoplan (RA101495)
- Where
- 12 sites in United States, Australia and 6 more
- Volunteers
- 19 people
- Sponsor
- Ra Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary purpose of this study is to evaluate the safety, tolerability, and efficacy of multiple intravenous (IV) doses of ravulizumab administered to complement inhibitor treatment-naïve participants with PNH.
- Trial name
- NCT02605993
- Testing
- Ravulizumab
- Where
- 16 sites in Canada, France and 5 more
- Volunteers
- 26 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study.
- Trial name
- NCT01194804
- Testing
- Eculizumab
- Volunteers
- 27 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-CC5 in healthy adult volunteers and subjects with PNH
- Trial name
- NCT02352493
- Testing
- ALN-CC5, Sterile Normal Saline (0.9% NaCl)
- Where
- 4 sites in Spain, United Kingdom
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria (PNH)
ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who had never been treated with a complement inhibitor (treatment-naïve).
- Trial name
- NCT02946463
- Testing
- Ravulizumab, Eculizumab
- Where
- 120 sites in United States, Argentina and 24 more
- Volunteers
- 272 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
The purpose of the study is to evaluate the safety and efficacy of zilucoplan (RA101495) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
- Trial name
- NCT03078582
- Testing
- Zilucoplan (RA101495)
- Where
- 12 sites in Australia, Canada and 6 more
- Volunteers
- 26 people
- Sponsor
- Ra Pharmaceuticals
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
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
This study will enroll participants aged 12 years or older with a body weight ≥ 40 kilograms (kg) diagnosed with PNH who have not been previously treated with complement inhibitor therapy.
- Trial name
- COMMODORE 3 (NCT04654468)
- Testing
- Crovalimab
- Where
- 5 sites in China
- Volunteers
- 51 people
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms.
- Trial name
- CONSENTII (NCT03427060)
- Testing
- Coversin
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- AKARI Therapeutics
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