Clinical trials: Hemoglobinuria, Paroxysmal
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)
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of…
- Trial name
- NCT06934967
- Testing
- LNP023
- Where
- 15 sites in United States, Brazil and 4 more
- Volunteers
- 12 people
- Sponsor
- Novartis Pharmaceuticals
Leukemia
Eculizumab in Treating Patients With Paroxysmal Nocturnal Hemoglobinuria
RATIONALE: Chemoprevention is the use of certain drugs to keep cancer from forming, growing, or coming back. The use of eculizumab may prevent leukemia and stop the destruction of red blood cells in patients with paroxysmal…
- Trial name
- NCT00112983
- Testing
- eculizumab
- Where
- United States
- Sponsor
- Jonsson Comprehensive Cancer Center
Paroxysmal Nocturnal Hemoglobinuria
Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, open-label, intra-subject, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and therapeutic potential of BCX10013 in participants with PNH.
- Trial name
- NCT06100900
- Testing
- BCX10013
- Where
- 4 sites in Malaysia, South Africa
- Volunteers
- 8 people
- Sponsor
- BioCryst Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- ACCESS-1 (NCT05133531)
- Testing
- Ravulizumab, Pozelimab, Cemdisiran
- Where
- 68 sites in United States, Brazil and 22 more
- Volunteers
- 202 people
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria, PNH
Efficacy and Safety of Lanoracopan in the Treatment of PNH
This study aimed to evaluate the efficacy and safety of Lanoracopan Hydrochloride in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Trial name
- NCT07788105
- Testing
- Lanoracopan Hydrochloride
- Volunteers
- 90 people
- Sponsor
- Peking Union Medical College Hospital
Paroxysmal Nocturnal Hemoglobinuria
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
- Trial name
- NCT05389449
- Testing
- Danicopan
- Where
- 45 sites in United States, Brazil and 13 more
- Volunteers
- 80 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Study of the Safety and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
- Trial name
- NCT05889299
- Testing
- OMS906
- Where
- Ukraine
- Volunteers
- 15 people
- Sponsor
- Omeros Corporation
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
First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH
This is a 3-part Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single (Part 1) and multiple (Part 2) ascending doses of BCX9930 in healthy subjects and in subjects with…
- Trial name
- NCT04330534
- Testing
- BCX9930
- Where
- 4 sites in Austria, South Africa and 1 more
- Volunteers
- 168 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
The primary objective of the study is: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch…
- Trial name
- ACCESS 2 (NCT05131204)
- Testing
- Cemdisiran, Eculizumab, Pozelimab
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Study of Single-dose MY008211A in Healthy Adults
The trial is the first human trial. The safety, tolerability, PK and PD of MY008211A Tablets will be evaluated in healthy subjects.
- Trial name
- NCT05642585
- Testing
- MY008211A tablets
- Where
- China
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
Paroxysmal Hemoglobinuria, Nocturnal
Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria
This is a Phase I/II, first-in-human study consisting of four sequential parts and an open-label extension (OLE). The safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single doses of crovalimab will be…
- Trial name
- COMPOSER (NCT03157635)
- Testing
- Crovalimab
- Where
- 15 sites in France, Germany and 5 more
- Volunteers
- 59 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria
Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
This program is designed to provide access to pozelimab and cemdisiran and document the long-term safety of pozelimab and cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT06028594
- Testing
- Pozelimab, Cemdisiran
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria, Hemoglobinuria
Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)
Paroxysmal nocturnal hemoglobinuria is an acquired chronic hemolytic anemia,this study is designed to evaluate the safety and efficacy of Levamisole combined with cyclosporine A in patients with Subclinical Paroxysmal Nocturnal…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, Hemoglobinuria, Hemoglobinuria, Paroxysmal, Proteinuria
- Trial name
- NCT01760096
- Testing
- Levamisole+cyclosporin A+Glucocorticoids, cyclosporin A+Glucocorticoids, Glucocorticoids
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
The study will evaluate the efficacy and safety of the oral Factor D (FD) inhibitor ALXN2050 (ACH-0145228) monotherapy in patients with PNH that are treatment naïve, or patients currently treated with eculizumab who still…
- Trial name
- NCT04170023
- Testing
- ALXN2050
- Where
- 11 sites in Canada, Italy and 5 more
- Volunteers
- 29 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults
Primary objective The tolerability and safety of SAR443809 Secondary The PK parameters of SAR443809 The PD activity of SAR443809 The immunogenicity of SAR443809
- Trial name
- NCT06326814
- Testing
- Humanized anti-Factor Bb monoclonal antibody
- Where
- 2 sites in United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- Sanofi
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
Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
This was a Phase II randomized, open-label, multicenter, efficacy, safety, pharmacokinetic and pharmacodynamic study assessing four iptacopan doses in adult Paroxysmal nocturnal hemoglobinuria (PNH) patients with active hemolysis…
- Trial name
- NCT03896152
- Testing
- LNP023
- Where
- 5 sites in Malaysia, Singapore and 2 more
- Volunteers
- 13 people
- Sponsor
- Novartis Pharmaceuticals
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