A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN
Study Identifier:
ACH471-201
ClinicalTrials.gov Identifier:
EudraCT Identifier:
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete
Available Documents
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: Danicopan
Date
Aug 2017 - Dec 2018
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 16 - 65 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Must have had clinical diagnosis of C3G (C3 glomerulonephritis or dense deposit disease, the 2 types of C3G) or idiopathic IC-MPGN by renal biopsy for at least 3 months prior to dosing, with the pathologic diagnosis verified by a review of the renal biopsy by the study central pathologist
- C3 must have been \<50% of the lower limit of normal
- C4 complement protein (C4) must have been \>90% of the lower limit of normal
- Must have been willing to comply with study-specific vaccination requirements for Haemophilus influenzae, Streptococcus pneumoniae, and Neisseria meningitidis strains A, C, W, and Y
- Negative pregnancy test for females prior to dosing and throughout the study
Exclusion Criteria
- History of a major organ transplant (for example, heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant. Individuals receiving renal replacement therapy were also excluded
- Evidence of monoclonal gammopathy of unclear significance, infections, malignancy, autoimmune diseases, or other conditions to which C3G or IC-MPGN may have been secondary
- Estimated glomerular filtration rate (using Modification of Diet in Renal Disease equation) \<45 milliliters/minute/1.73 square meters at the time of Screening or at any time over the preceding 4 weeks
- Receipt of eculizumab at any dose or interval within the past 75 days prior to dosing
- Use of tacrolimus or cyclosporine within 2 weeks of the first dose of danicopan
- History of febrile illness, a body temperature \>38°Celsius, or other evidence of a clinically significant active infection, within 14 days prior to study drug administration
- History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection
- Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who was pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration
Sex
Female & Male
Age
16 - 65 Years
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: Danicopan
Date
Aug 2017 - Dec 2018
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 16 - 65 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Must have had clinical diagnosis of C3G (C3 glomerulonephritis or dense deposit disease, the 2 types of C3G) or idiopathic IC-MPGN by renal biopsy for at least 3 months prior to dosing, with the pathologic diagnosis verified by a review of the renal biopsy by the study central pathologist
- C3 must have been \<50% of the lower limit of normal
- C4 complement protein (C4) must have been \>90% of the lower limit of normal
- Must have been willing to comply with study-specific vaccination requirements for Haemophilus influenzae, Streptococcus pneumoniae, and Neisseria meningitidis strains A, C, W, and Y
- Negative pregnancy test for females prior to dosing and throughout the study
Exclusion Criteria
- History of a major organ transplant (for example, heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant. Individuals receiving renal replacement therapy were also excluded
- Evidence of monoclonal gammopathy of unclear significance, infections, malignancy, autoimmune diseases, or other conditions to which C3G or IC-MPGN may have been secondary
- Estimated glomerular filtration rate (using Modification of Diet in Renal Disease equation) \<45 milliliters/minute/1.73 square meters at the time of Screening or at any time over the preceding 4 weeks
- Receipt of eculizumab at any dose or interval within the past 75 days prior to dosing
- Use of tacrolimus or cyclosporine within 2 weeks of the first dose of danicopan
- History of febrile illness, a body temperature \>38°Celsius, or other evidence of a clinically significant active infection, within 14 days prior to study drug administration
- History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection
- Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who was pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration
Protocol Summary
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.
Trial Locations
Location
Status
Location
Clinical Trial Site
Melbourne, Australia
Status
N/A
Location
Clinical Trial Site
Antwerp, Belgium
Status
N/A
Location
Clinical Trial Site
Leiden, Netherlands
Status
N/A