Clinical trials: PNH
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)
An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical Study No. BCD-148-2/NOCTURN
To study the long-term safety and efficacy of BCD-148 in patients with paroxysmal nocturnal hemoglobinuria (PNH), previously treated in clinical study (CS) BCD-148-2/NOCTURN
- Trial name
- NCT06987864
- Testing
- eculizumab
- Where
- 11 sites in Russia
- Volunteers
- 24 people
- Sponsor
- Biocad
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
Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of LP-005 in healthy volunteers.
- Trial name
- NCT06294301
- Testing
- LP-005 Dose 1 (Single), LP-005 Dose 2 (Single), LP-005 Dose 3 (Single)
- Where
- China
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- Longbio Pharma
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this research study is to understand the safety, pharmacokinetics and pharmacodynamics of a single dose of TT30 (ALXN1102 and ALXN1103 formulations) when given IV (through a vein) or SC (under the skin) to patients…
- Trial name
- NCT01335165
- Testing
- ALXN1102, ALXN1103
- Where
- 5 sites in United States, Czechia and 3 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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.
Paroxysmal Nocturnal Hemoglobinuria
Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, single-arm, open-label study. Patients with Paroxysmal Nocturnal Hemoglobinuria who had previously received and completed the HRS-5965 study well included.
- Trial name
- NCT06238544
- Testing
- HRS-5965 tablets, HRS-5965 Capsules
- Where
- 2 sites in China
- Volunteers
- 132 people
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.
- Trial name
- NCT06134414
- Testing
- MY008211A tablets
- Volunteers
- 40 people
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
Paroxysmal Nocturnal Hemoglobinuria, Hemoglobinuria
Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria
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 classic paroxysmal nocturnal…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, Hemoglobinuria, Hemoglobinuria, Paroxysmal, Proteinuria
- Trial name
- PNH-2012 (NCT01642979)
- Testing
- Levamisole+cyclosporin A+Glucocorticoids, cyclosporin A+Glucocorticoids, Glucocorticoids
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study on Mass Balance of [14C]HSK39297 in Healthy Chinese Adult Male Subjects
To evaluate the absorption, metabolism and excretion in healthy Chinese male subjects after a single oral dose of [14C]HSK39297
- Trial name
- NCT06852092
- Testing
- Carbon-14 labeled HSK39297
- Where
- China
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Haisco Pharmaceutical Group Co., Ltd.
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
Rheologic Disease, Sickle Cell Disease
Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease
The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of…
- Conditions
- Rheologic Disease, Sickle Cell Disease, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT01294891
- Testing
- Imaging
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University
Paroxysmal Nocturnal Hemoglobinuria, PNH, Complement-mediated Kidney Disease
A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants
This study is a first-in-human (FIH) trial of CMS-D017 conducted in healthy Chinese adult participants, consisting of two parts: Part 1-a single ascending dose (SAD) study (referred to as Part 1 SAD), and Part 2-a multiple…
- Trial name
- NCT07462780
- Testing
- CMS-D017 Capsule
- Where
- China
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Shenzhen Kangzhe Biotechnology Co., Ltd.
PNH
Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of a Single Ascending Dose (SAD) of CAN106 Administered Intravenously (IV) in Healthy Subjects
This is a single site, single dose escalation study in healthy subject with CAN106. The study is to assess the safety and tolerability of single escalating doses of CAN106; to characterize the PK and PD profile of CAN106; and to…
- Trial name
- NCT05095168
- Testing
- CAN106
- Where
- Singapore
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- CARE Pharma Shanghai Ltd.
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
Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is an extension study for patients who have completed a prior P10-LP005-02 clinical study. The aim of this study is to evaluate the long-term safety, efficacy, and pharmacokinetics of LP-005 injection in adult patients with…
- Trial name
- NCT07266155
- Testing
- LP-005 Injection
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Longbio Pharma
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab.
- Trial name
- NCT03030183
- Testing
- Zilucoplan (RA101495)
- Where
- 6 sites in United States
- Volunteers
- 3 people
- Sponsor
- Ra Pharmaceuticals
Paroxysmal Nocturnal Haemoglobinuria
Real-life Treatment Outcomes of Ravulizumab in PNH
The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.
- Trial name
- PNH-RECORD (NCT06633536)
- Testing
- Ravulizumab
- Where
- 11 sites in Poland
- Volunteers
- 64 people
- Sponsor
- AstraZeneca
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
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.