Clinical Trials

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Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 3
  • Intervention Type: Drug
  • Participants: 167
  • Start Date: June 3, 2021
An Open Label, Multicenter Roll-over Extension Program (REP) to Characterize the Long-term Safety and Tolerability of Iptacopan (LNP023) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Completed PNH Phase 2 and Phase3 Studies With Iptacopan
Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 3
  • Intervention Type: Drug
  • Participants: 50
  • Start Date: March 10, 2021
A Phase III, Multicenter, Single Arm Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
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Clinical Trial
  • Status: Recruiting
  • Phase: Phase 3
  • Intervention Type: Drug
  • Participants: 91
  • Start Date: January 25, 2021
A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia, Despite Treatment With an Intravenous Anti-C5 Antibody.
Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 4
  • Intervention Type: Biological
  • Participants: 20
  • Start Date: January 2021
Phase 4, Single-Arm Study of Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
Clinical Trial
  • Status: Enrolling by invitation
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 200
  • Start Date: December 18, 2020
A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 3
  • Intervention Type: Drug
  • Participants: 84
  • Start Date: December 16, 2020
A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 3
  • Intervention Type: Drug
  • Participants: 200
  • Start Date: October 8, 2020
A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors.
Clinical Trial
  • Status: Not yet recruiting
  • Intervention Type: Other
  • Participants: 60
  • Start Date: October 2020
The Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 3
  • Intervention Type: Drug
  • Participants: 250
  • Start Date: September 30, 2020
A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors.
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Drug
  • Participants: 148
  • Start Date: March 3, 2020
A Phase 1 Dose-ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of BCX9930 in Healthy Subjects and in Subjects With Paroxysmal Nocturnal Hemoglobinuria
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 26
  • Start Date: December 16, 2019
A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and With an Approved C5 Inhibitor as Background Therapy
Clinical Trial
  • Status: Enrolling by invitation
  • Phase: Phase 3
  • Intervention Type: Drug
  • Participants: 147
  • Start Date: December 3, 2019
An Open-label Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria
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