Clinical trials: CFD
Paroxysmal Nocturnal Hemoglobinuria
Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria
This study was designed to provide continued access to BCX9930 for participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) who had benefited from treatment with BCX9930 in another BioCryst-sponsored study for PNH who, in the…
- Trial name
- NCT05741346
- Testing
- BCX9930
- Where
- 11 sites in France, Hungary and 5 more
- Volunteers
- 28 people
- Sponsor
- BioCryst Pharmaceuticals
C3 Glomerulonephritis, Dense Deposit Disease
A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN
The primary objective of this study was to determine whether ACH-0144471 (also known as danicopan and ALXN2040) increases blood C3 complement protein (C3) levels in participants with low C3 levels due to either C3G or IC-MPGN.
- Conditions
- C3 Glomerulonephritis, Dense Deposit Disease, C3 Glomerulopathy, Immune Complex Mediated Membranoproliferative Glomerulonephritis
- Trial name
- NCT03124368
- Testing
- Danicopan
- Where
- 3 sites in Australia, Belgium and 1 more
- Volunteers
- 6 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
Hepatic Impairment
Study of Danicopan in Participants With Hepatic Impairment
The purpose of this study was to evaluate the safety, tolerability, and pharmacokinetics (PK) of ACH-0144471 (danicopan) in participants with hepatic impairment (HI) as compared to healthy matched participants.
- Trial name
- NCT03555539
- Testing
- Danicopan
- Where
- 2 sites in United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Healthy
Study of Radiolabeled ALXN2050 in Healthy Adult Males
This is an open-label study to assess the absorption, metabolism, excretion, and mass balance of a single oral dose of carbon-14 ([14C])-ALXN2050 in healthy adult males.
- Trial name
- NCT04609670
- Testing
- [14C]-ALXN2050
- Where
- United States
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Healthy
Study of Multiple Doses of Danicopan in Healthy Participants
This was a multiple ascending dose, randomized, double-blind study assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of danicopan in healthy participants.
- Trial name
- NCT04889690
- Testing
- Danicopan
- Where
- New Zealand
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Healthy
A Study of Modified Release Formulations of Danicopan in Healthy Adult Participants
The purpose of this study was to evaluate and compare the plasma pharmacokinetic profiles of ACH-0144471 (danicopan) in healthy participants after administration of single oral doses of modified release prototype formulations.
- Trial name
- NCT03384186
- Testing
- Danicopan Modified Release Prototype 1, Danicopan Modified Release Prototype 2, Danicopan Modified Release Prototype 3
- Where
- United Kingdom
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
C3 Glomerulopathy, C3 Glomerulonephritis
A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G)
The primary purpose of this proof-of-concept clinical study was to evaluate the efficacy and safety of the study drug, ACH-0144471 (also known as danicopan and ALXN2040), in participants with C3G who also had significant…
- Conditions
- C3 Glomerulopathy, C3 Glomerulonephritis, Dense Deposit Disease
- Trial name
- NCT03369236
- Testing
- Danicopan
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 13 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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.
Healthy
A Study of the Cardiac Effects of ALXN2050 in Healthy Adults
This is a randomized, double-blind, double-dummy, placebo- and positive-controlled parallel study to evaluate the effect of ALXN2050 on the QT interval in healthy adult participants.
- Trial name
- NCT04660890
- Testing
- ALXN2050, Moxifloxacin
- Where
- United States
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Healthy
A Drug Interaction Study Between Danicopan and Midazolam, Fexofenadine, and Mycophenolate Mofetil in Healthy Participants
The purpose of this study was to determine the potential drug interaction between danicopan (ACH-0144471) and midazolam, fexofenadine, and mycophenolate mofetil.
- Trial name
- NCT03108274
- Testing
- Danicopan, Midazolam, Fexofenadine
- Where
- New Zealand
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Healthy
A Study of Multiple Doses of ALXN2050 in Healthy Adults
This was a Phase 1, placebo-controlled, randomized, double-blind (participant and investigator blind, sponsor open), multiple-ascending dose study conducted in healthy participants to demonstrate the safety and tolerability and…
- Trial name
- NCT05047484
- Testing
- ALXN2050
- Where
- New Zealand
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Impaired Hepatic Function, Healthy
Study of ALXN2050 in Participants With Hepatic Impairment
This study will investigate the impact of impaired hepatic function (IHF) on the plasma pharmacokinetics of ALXN2050 in order to provide dosing recommendations for future indications in individuals with varying degrees of IHF.
- Trial name
- NCT05259085
- Testing
- ALXN2050
- Where
- 2 sites in United States
- Volunteers
- 26 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Healthy
A Drug Interaction Study of ACH-0145228
This was a 2-part study (Part 1 and Part 2), with each part being an open-label, fixed sequence, 2-period study in healthy adult participants.
- Trial name
- NCT04709081
- Testing
- ACH-0145228, Midazolam, Digoxin
- Where
- United States
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
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.
Paroxysmal Nocturnal Hemoglobinuria
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
- Trial name
- NCT05389449
- Testing
- Danicopan
- Where
- 45 sites in United States, Brazil and 13 more
- Volunteers
- 80 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy
Study to Evaluate the Safety, Tolerability of BCX9930 in Participants With Either Complement 3 Glomerulopathy (C3G), Immunoglobulin A Nephropathy (IgAN), or Primary Membranous Nephropathy (PMN)
The objective of this study was to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN.
- Conditions
- Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, Membranous Nephropathy
- Trial name
- RENEW (NCT05162066)
- Testing
- BCX9930
- Where
- 11 sites in France, Italy and 2 more
- Volunteers
- 2 people
- Sponsor
- BioCryst Pharmaceuticals
Healthy
Study of the Metabolism of Danicopan in Healthy Adults
This is a 2-part open-label, randomized, single-dose, 3-sequence, 3-period crossover, relative bioavailability, and food-effect study comparing different formulations of danicopan in healthy adult participants.
- Trial name
- NCT04609696
- Testing
- Danicopan: Powder-In-Capsule 1, Danicopan: Powder-In-Capsule 2, Danicopan: Tablet
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Renal Impairment, Healthy
Study of ALXN2050 in Participants With Renal Impairment
This study will investigate the impact of impaired renal function on the plasma pharmacokinetics of ALXN2050 in order to provide dosing recommendations for future indications in individuals with impaired renal function.
- Trial name
- NCT04623710
- Testing
- ALXN2050
- Where
- 2 sites in United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH
This is a 3-part Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single (Part 1) and multiple (Part 2) ascending doses of BCX9930 in healthy subjects and in subjects with…
- Trial name
- NCT04330534
- Testing
- BCX9930
- Where
- 4 sites in Austria, South Africa and 1 more
- Volunteers
- 168 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals