Clinical trials: PNH
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
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
Preeclampsia, Severe Preeclampsia
Complement Regulation to Undo Systemic Harm in Preeclampsia
This is a Phase II, single arm, open-label study to determine if treatment with eculizumab prolongs pregnancy compared to historical controls in women with preeclampsia between 23-30 weeks gestation.
- Conditions
- Preeclampsia, Severe Preeclampsia, Eculizumab, HELLP
- Trial name
- CRUSH (NCT04725812)
- Testing
- Eculizumab
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Cedars-Sinai Medical Center
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
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.
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
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)
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
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 Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
The primary objective of the study is to evaluate the safety and tolerability of 2 dosing regimens of pozelimab and cemdisiran combination therapy during the open-label treatment period (OLTP) The secondary objectives of the…
- Trial name
- NCT04811716
- Testing
- Pozelimab, Cemdisiran
- Where
- 13 sites in Hong Kong, Hungary and 4 more
- Volunteers
- 24 people
- Sponsor
- Regeneron 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)
A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH
This study will be the initial exploration of pegcetacoplan in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of pegcetacoplan will guide…
- Trial name
- NCT02264639
- Testing
- Pegcetacoplan
- Where
- 7 sites in United States
- Volunteers
- 9 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
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
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab
To determine the effectiveness of ACH-0144471 (also known as danicopan and ALXN2040) in improving anemia when given with eculizumab for 24 weeks in participants with PNH.
- Trial name
- PNH (NCT03472885)
- Testing
- Danicopan, Eculizumab
- Where
- 5 sites in United States, Italy and 1 more
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
This is a randomized, double-blind, active-controlled phase 3 study of ABP 959 in participants with paroxysmal nocturnal hemoglobinuria.
- Trial name
- DAHLIA (NCT03818607)
- Testing
- ABP 959, Eculizumab
- Where
- 24 sites in United States, Czechia and 12 more
- Volunteers
- 42 people
- Sponsor
- Amgen
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.
PNH
Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This study evaluated the safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of multiple intravenous (IV) doses of ALXN1210 administered to participants with PNH who have not previously been…
- Trial name
- NCT02598583
- Testing
- ALXN1210
- Where
- 8 sites in Australia, South Korea
- Volunteers
- 13 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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 Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
A study designed to evaluate the non-inferiority of crovalimab compared with eculizumab in participants with PNH who have not been previously treated with complement inhibitor therapy.
- Trial name
- COMMODORE 2 (NCT04434092)
- Testing
- Crovalimab, Eculizumab
- Where
- 63 sites in Argentina, Brazil and 21 more
- Volunteers
- 210 people
- Sponsor
- Hoffmann-La Roche
Paroxysmal Hemoglobinuria, Nocturnal
Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.
- Trial name
- NCT00122317
- Testing
- eculizumab
- Where
- 40 sites in United States, Australia and 10 more
- Volunteers
- 187 people
- Sponsor
- Alexion Pharmaceuticals, Inc.