Clinical trials: Hemoglobinuria, Paroxysmal
Paroxysmal Nocturnal Hemoglobinuria
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .
- Trial name
- NCT04085601
- Testing
- APL-2
- Where
- 30 sites in Colombia, Hong Kong and 8 more
- Volunteers
- 53 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.
The objectives of the study are to assess the safety, tolerability, preliminary efficacy and PK of multiple subcutaneous (SC) doses of pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who have not received…
- Trial name
- PADDOCK (NCT02588833)
- Testing
- Pegcetacoplan
- Where
- 7 sites in Hong Kong, Malaysia and 2 more
- Volunteers
- 23 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
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
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
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, PNH
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.
- Trial name
- NCT06578949
- Testing
- Ravulizumab
- Where
- 8 sites in China
- Volunteers
- 18 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study.
- Trial name
- NCT01194804
- Testing
- Eculizumab
- Volunteers
- 27 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.
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
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 (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
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
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 (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.
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, 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