Clinical trials: PNH
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
Danicopan PMS in Korea
As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients who are treated with Danicopan as an add-on to ravulizumab or eculizumab in normal clinical…
- Trial name
- NCT07457151
- Where
- 4 sites in South Korea
- Volunteers
- 27 people
- Sponsor
- AstraZeneca
Bone Marrow Failure Disorders, VEXAS Syndrome
Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
STUDY DESCRIPTION: Bone marrow failure can result from immune mediated attack on stem cells or from inherited genetic defects such as telomere biology disorders or Fanconi anemia.
- Conditions
- Bone Marrow Failure Disorders, VEXAS Syndrome, Hemoglobinurea, Paroxysmal, Myelodysplastic Syndromes
- Trial name
- NCT07102849
- Where
- United States
- Volunteers
- 1,400 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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 (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
PNH
A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH
This is a Phase IIa, open-label, multiple dose, study in patients with PNH who have not received eculizumab (Soliris ®) in the past. A single cohort of subjects is planned for evaluation.
- Trial name
- NCT03593200
- Testing
- Pegcetacoplan
- Where
- 3 sites in Bulgaria, Serbia
- Volunteers
- 4 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
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
Global PNH Patient Registry
The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history study that will result in a more comprehensive understanding of the…
- Trial name
- NCT05755867
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Aplastic Anemia and MDS International Foundation
Paroxysmal Nocturnal Hemoglobinuria (PNH)
SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
The goal of this clinical trial is to learn if drug SLN12140 works to treat Complement Inhibitor-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria in adults.
- Trial name
- NCT07387302
- Testing
- SLN12140
- Volunteers
- 10 people
- Sponsor
- Linno Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, Natural History Study
Natural History Study of Adult Patients With Paroxysmal Nocturnal Hemoglobinuria of High-risk Hemolysis in China
This is a single-center observational study conducted in adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis.
- Trial name
- NCT05125341
- Where
- China
- Volunteers
- 67 people
- Sponsor
- CARE Pharma Shanghai Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study was to determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- NCT02534909
- Testing
- LFG316, LNP023
- Where
- 7 sites in Czechia, Japan and 1 more
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
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
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 (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 (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
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.
Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML)
Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies
Blood and marrow stem cell transplant has improved the outcome for patients with high-risk hematologic malignancies. However, most patients do not have an appropriate HLA (immune type) matched sibling donor available and/or are…
- Conditions
- Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML), Leukemia, Myeloid, Chronic(CML), Juvenile Myelomonocytic Leukemia(JMML)
- Trial name
- NCT00186823
- Testing
- Miltenyi Biotec CliniMACS, Stem Cell Transplantation, TBI, systemic chemotherapy and antibodies as follows:
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- St. Jude Children's Research Hospital
Aplastic Anemia, Paroxysmal Hemoglobinuria, Nocturnal
Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
OBJECTIVES: I. Confirm the efficacy demonstrated in a pilot study using high dose cyclophosphamide in patients with severe aplastic anemia. II.
- Trial name
- NCT00004464
- Testing
- cyclophosphamide, filgrastim
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
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
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.