Clinical trials: Hemoglobinuria, Paroxysmal
Paroxysmal Nocturnal Hemoglobinuria (PNH), Persistent Anemia
A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor ("C5-inhibitor") and…
- Trial name
- NCT07187401
- Testing
- ALN-CFB
- Where
- 8 sites in Canada, South Korea and 1 more
- Volunteers
- 24 people
- Sponsor
- Regeneron Pharmaceuticals
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 (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
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
PNH - Paroxysmal Nocturnal Hemoglobinuria
Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The proposed study, NM8074-PNH-101, is a phase II, open-label, multi-dose, unicenter trial to evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects.
- Trial name
- NCT05731050
- Testing
- NM8074
- Volunteers
- 6 people
- Sponsor
- NovelMed Therapeutics
Paroxysmal Nocturnal Hemoglobinuria, PNH
Danicopan Early Access Program
This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
- Trial name
- NCT05982938
- Testing
- Danicopan
- Where
- 15 sites in Italy
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104
The purpose of this study is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in complement inhibitor-naïve participants with PNH.
- Trial name
- NCT05476887
- Testing
- KP104
- Where
- 4 sites in China
- Volunteers
- 35 people
- Sponsor
- Kira Pharmacenticals (US), LLC.
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
Ultomiris-exposed Pregnant/ Postpartum, Pregnancy
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of…
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
- Trial name
- NCT06312644
- Testing
- Ultomiris
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- ACCESS-EXT (NCT05744921)
- Testing
- Pozelimab, Cemdisiran
- Where
- 48 sites in Brazil, Canada and 18 more
- Volunteers
- 202 people
- Sponsor
- Regeneron 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.
Paroxysmal Nocturnal Hemoglobinuria
Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study is to assess the long-term safety and tolerability of repeat-dose OMS906 5 mg/kg IV administration at 8-week intervals in patients with PNH.
- Trial name
- NCT06298955
- Testing
- OMS906 study drug
- Where
- 5 sites in Germany, Switzerland and 2 more
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Omeros Corporation
PNH - Paroxysmal Nocturnal Hemoglobinuria
A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, randomized, single-blind Phase II trial to evaluate the efficacy and safety of XH-S003 capsules in PNH patients.
- Trial name
- XH-S003-II-101 (NCT06978699)
- Testing
- XH-S003 Capsule
- Where
- China
- Volunteers
- 24 people
- Sponsor
- S-INFINITY Pharmaceuticals Co., Ltd
PNH
A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean PNH Patients
This study is a retrospective non-interventional study, and the main objective of this study is to identify the impact of eculizumab treatment and the improvement of complications before and after eculizumab treatment, and to…
- Trial name
- NCT05337683
- Where
- South Korea
- Volunteers
- 80 people
- Sponsor
- Handok Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
The first-in-human Phase 1/Phase 2a study described herein will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in both healthy participants (HP) and in patients with paroxysmal…
- Trial name
- PNH (NCT05876312)
- Testing
- ADX-038
- Where
- 4 sites in Australia, United Kingdom
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- ADARx Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with paraxysmal noturnal…
- Trial name
- Soliris (NCT05886244)
- Testing
- Eculizumab
- Where
- 7 sites in China
- Volunteers
- 25 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients.
- Trial name
- PNH-NIS (NCT06931691)
- Testing
- LNP023
- Where
- 14 sites in China
- Volunteers
- 80 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.
The purpose of the study was to find out if iptacopan is effective and safe in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switched from their current standard of care treatment (eculizumab or ravulizumab)…
- Trial name
- APPULSE (NCT05630001)
- Testing
- Iptacopan
- Where
- 23 sites in United States, France and 6 more
- Volunteers
- 52 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.
- Trial name
- NCT04469465
- Testing
- Danicopan, C5 Inhibitor
- Where
- 62 sites in United States, Brazil and 16 more
- Volunteers
- 86 people
- Sponsor
- Alexion Pharmaceuticals, Inc.