A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G)

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-06-12

Study Completion Date

2020-12-18

Brief Summary

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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 proteinuria attributable to C3G.

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Detailed Description

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Conditions

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C3 Glomerulopathy C3 Glomerulonephritis Dense Deposit Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Investigator and participant blinded; Sponsor open. Participants were randomized 1:1 to receive either danicopan or placebo for a period of 6 months, followed by an open-label extension.

Study Groups

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Danicopan (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period)

Danicopan was administered at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period.

All participants who completed the double-blind treatment period were enrolled in the open-label extension period and were to receive danicopan 200 mg TID.

Group Type ACTIVE_COMPARATOR

Danicopan

Intervention Type DRUG

Danicopan was administered as an oral tablet.

Placebo (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period)

Placebo was administered TID during the 6-month treatment period.

All participants who completed the double-blind treatment period were enrolled in the open-label extension period and were to receive danicopan 200 mg TID.

Group Type PLACEBO_COMPARATOR

Danicopan

Intervention Type DRUG

Danicopan was administered as an oral tablet.

Placebo

Intervention Type DRUG

Matching placebo was administered as an oral tablet.

Interventions

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Danicopan

Danicopan was administered as an oral tablet.

Intervention Type DRUG

Placebo

Matching placebo was administered as an oral tablet.

Intervention Type DRUG

Other Intervention Names

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ACH-4471 ACH4471 4471 ACH-0144471 (formerly) ALXN2040

Eligibility Criteria

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Inclusion Criteria

* Had biopsy-confirmed primary C3G
* Had clinical evidence of ongoing disease based on significant proteinuria, attributable to C3G disease in the opinion of the Principal Investigator (PI), and present prior to study entry and confirmed during Screening
* Was willing to comply with vaccination requirements.

Exclusion Criteria

* Had a history or presence of any clinically relevant co-morbidities that would make the participant inappropriate for the study
* Had ever received danicopan
* Had more than 50% fibrosis or more than 50% of glomeruli with cellular crescents on the pre-treatment renal biopsy
* Had an estimated glomerular filtration rate \<30 milliliters/minute/1.73 meters squared at the time of screening or at any time over the preceding 4 weeks
* Was a renal transplant recipient or receiving renal replacement therapy
* Had a history of a major organ transplant or hematopoietic stem cell/marrow transplant
* Had evidence of monoclonal gammopathy of unclear significance, infections, malignancy, autoimmune diseases, or other conditions to which C3G is secondary
* Had other renal diseases that would interfere with interpretation of the study
* Had been diagnosed with or showed evidence of hepatobiliary cholestasis
* Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration
* Had a 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
* Had evidence of human immunodeficiency virus, hepatitis B infection, or active hepatitis C infection at Screening
* Had laboratory abnormalities at screening that, in the opinion of the PI, would make the participant inappropriate for the study

Minimum Eligible Age

17 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No