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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03459443
Registration number
NCT03459443
Ethics application status
Date submitted
26/02/2018
Date registered
9/03/2018
Date last updated
21/08/2023
Titles & IDs
Public title
A Proof of Concept Study for a 12 Month Treatment in Patients With C3G or IC-MPGN Treated With ACH-0144471
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Scientific title
An Open-Label Phase 2 Proof-of-Concept Study in Patients With C3 Glomerulopathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN) Treated With ACH-0144471
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Secondary ID [1]
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2017-002674-39
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Secondary ID [2]
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ACH471-205
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
C3 Glomerulonephritis
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C3 Glomerulopathy
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Immune Complex Membranoproliferative Glomerulonephritis
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IC-MPGN
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Dense Deposit Disease
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Condition category
Condition code
Renal and Urogenital
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Kidney disease
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Inflammatory and Immune System
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Danicopan
Experimental: Danicopan - Danicopan was to be administered to participants with C3G or IC-MPGN at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then the dosage was to be increased to 200 mg TID for the remainder of the study.
Treatment: Drugs: Danicopan
Danicopan was to be administered as an oral tablet.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline In Composite Biopsy Score At End Of Initial 12-Month Treatment Period
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Assessment method [1]
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The composite biopsy score was based on a score incorporating changes in the activity index, glomerular C3c staining, and glomerular macrophage infiltration at the end of the initial 12 months of treatment. The composite renal biopsy index scoring system ranged from 0 to 21, with higher scores indicating worse outcomes.
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Timepoint [1]
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Baseline, end of initial 12-Month Treatment Period
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Primary outcome [2]
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Participants With Reduction In Proteinuria At End Of Initial 12-Month Treatment Period
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Assessment method [2]
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Proteinuria reduction was defined as =30% decrease from baseline based on 24-hour urine protein (mg/day).
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Timepoint [2]
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Baseline, end of initial 12-Month Treatment Period
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Secondary outcome [1]
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Change From Baseline In Proteinuria At End Of Initial 12-Month Treatment Period
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Assessment method [1]
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Proteinuria was assessed based on 24-hour urine collections at baseline and end of the initial 12-month Treatment Period.
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Timepoint [1]
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Baseline, end of initial 12-Month Treatment Period
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Secondary outcome [2]
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Percent Change From Baseline In Proteinuria At End Of Initial 12-Month Treatment Period
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Assessment method [2]
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Proteinuria was assessed based on 24-hour urine collections at baseline and end of initial 12-month Treatment Period.
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Timepoint [2]
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Baseline, end of initial 12-Month Treatment Period
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Secondary outcome [3]
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Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To End Of Initial 12-Month Treatment Period
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Assessment method [3]
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Slope of eGFR was estimated using a simple linear regression for each participant, including all data values from baseline until the end of the Initial 12-Month Treatment Period, with eGFR as the dependent variable and time as the independent variable.
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Timepoint [3]
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End of initial 12-Month Treatment Period
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Secondary outcome [4]
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Change From Baseline In eGFR At End Of Initial 12-Month Treatment Period
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Assessment method [4]
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Change from baseline in eGFR at end of initial 12-Month Treatment Period is presented.
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Timepoint [4]
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Baseline, end of initial 12-Month Treatment Period
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Secondary outcome [5]
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Participants With Significant Improvement In eGFR Relative To Baseline At End Of Initial 12-Month Treatment Period
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Assessment method [5]
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Significant improvement relative to baseline was defined as a = 25% increase from baseline in eGFR.
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Timepoint [5]
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Baseline, end of initial 12-Month Treatment Period
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Secondary outcome [6]
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Change From Baseline in eGFR Over 12 Months of Treatment For Participants Meeting eGFR Inclusion Criteria
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Assessment method [6]
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Participants were eligible for enrollment if inclusion criteria were met including having an eGFR >=30 milliliters (mL)/minute (min)/1.73 square meter (m^2) at the time of screening or at any time over the preceding 4 weeks. This Outcome Measure was registered in case there were participants who were enrolled and ended up not meeting the Eligibility Criteria and was intended to report data for change from baseline in eGFR for only the participants who met the eligibility criteria (that is, participants who did not meet the eligibility criteria would have been excluded from analysis for this Outcome Measure). Since all enrolled participants met the Eligibility Criteria, none of the participants were excluded from this analysis. Therefore, this data is the same data that is presented in Outcome Measure #6 "Change From Baseline In eGFR At End Of Initial 12-Month Treatment Period". Change from baseline in eGFR at end of initial 12-Month Treatment Period is presented.
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Timepoint [6]
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End of initial 12-Month Treatment Period
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Secondary outcome [7]
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Change From Baseline In Measured GFR At The End Of The Initial 12-Month Treatment Period
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Assessment method [7]
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Data for this Outcome Measure was to be collected where available. None of the sites collected data for this Outcome Measure.
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Timepoint [7]
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End of initial 12-Month Treatment Period
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Eligibility
Key inclusion criteria
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1. At least 12 years of age
2. Completion of the ACH471-201 clinical study OR diagnosed with biopsy-confirmed primary
C3G or IC-MPGN
3. If a pre-treatment biopsy is obtained, or if a historical biopsy is available for
review, it must have no more than 50% global fibrosis and no more than 50% of
glomeruli with cellular crescents
4. Clinical evidence of ongoing disease based on significant proteinuria (defined as =500
mg/day of protein in a 24-hour urine) attributable to C3G disease or IC-MPGN in the
opinion of the principal investigator (PI), and present prior to study entry and
confirmed during Screening
5. If on corticosteroids, anti-hypertensive medications, anti-proteinuric medications
(for example, angiotensin-converting enzyme inhibitors or angiotensin receptor
blockers), or mycophenolate mofetil, must be on a stable dose for at least 2 weeks
prior to screening
6. Female participants must use an acceptable method birth control to prevent pregnancy
during the clinical study and for 30 days after the last dose of study medication
7. Male participants must use highly effective birth control with a female partner to
prevent pregnancy during the clinical study and for 90 days after the last dose of
study medication
8. Must be up-to-date on routine vaccinations, or willing to be brought up-to-date, based
on local guidelines
9. Must have access to emergency medical care
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Minimum age
12
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
Exclusion Criteria
1. Have a history of a major organ transplant (for example, heart, lung, kidney, or
liver) or hematopoietic stem cell/marrow transplant
2. Have a history or presence of any clinically relevant co-morbidities that would make
the participant inappropriate for the study (for example, a comorbidity that is likely
to result in deterioration of the participant's condition, affect the participant's
safety during the study, or confound the results of the study), in the opinion of the
PI
3. Have an eGFR <30 milliliter/minute/1.73 m^2 at the time of screening or at any time
over the preceding 4 weeks
4. Is a renal transplant recipient or receiving renal replacement therapy
5. Have other renal diseases that would interfere with the interpretation of the study
6. Have evidence of monoclonal gammopathy of unclear significance, infections,
malignancy, autoimmune diseases, or other conditions to which C3G or IC-MPGN is
secondary
7. Have been diagnosed with or show evidence of hepatobiliary cholestasis
8. Females who are pregnant, nursing, or planning to become pregnant during the study or
within 90 days of ACH-0144471 administration or participants with a female partner who
is pregnant, nursing, or planning to become pregnant during the study or within 90
days of ACH-0144471 administration
9. Have a history of febrile illness, a body temperature >38°Celsius, or other evidence
of a clinically significant active infection, within 14 days prior to danicopan
administration
10. Have evidence of human immunodeficiency virus, hepatitis B infection, or active
hepatitis C infection at Screening
11. Have a history of meningococcal infection within the prior year
12. Have a history of hypersensitivity reactions to commonly used antibacterial agents,
including beta-lactams, penicillin, aminopenicillins, fluoroquinolones,
cephalosporins, and carbapenems, which, in the opinion of the investigator and/or an
appropriately qualified immunology or infectious disease expert, would make it
difficult to properly provide either empiric antibiotic therapy or treat an active
infection.
13. Have participated in a clinical study in which an investigational drug was given
within 30 days, or within 5 half-lives of the investigational drug, whichever is
longer, prior to the first dose of ACH-0144471
14. Have received eculizumab at any dose or interval within the past 50 days prior to the
first dose of ACH-0144471
15. Have received tacrolimus or cyclosporine within 2 weeks of the first dose of
ACH-0144471
16. Have a 12-lead electrocardiogram (ECG) with a QT interval Fridericia correction
formula >450 millisecond (msec) for males or >470 msec for females, or have ECG
findings which, in the opinion of the PI, could put the participant at undue risk
17. Have received any drug known to prolong the corrected QT interval within 2 weeks of
the first dose of ACH-0144471 and which, in the opinion of the PI, could put the
participant at undue risk
18. Have any of the following laboratory abnormalities at screening:
- Alanine transaminase > upper limit of normal (ULN)
- Aspartate aminotransferase > ULN
- Absolute neutrophil counts <1,000/microliter
- Total bilirubin >1.5* ULN
- Indirect bilirubin > ULN
- Any laboratory abnormality that, in the opinion of the PI, would make the
participant inappropriate for the study
19. Unwilling or unable to comply with the study protocol for any reason
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
20/06/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
29/03/2021
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Sample size
Target
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Accrual to date
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Final
22
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
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Recruitment hospital [1]
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Clinical Study Site - Sydney
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Recruitment hospital [2]
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Clinical Study Site - Brisbane
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Recruitment hospital [3]
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Clinical Study Site - Melbourne
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Recruitment postcode(s) [1]
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- Sydney
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Recruitment postcode(s) [2]
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- Brisbane
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Recruitment postcode(s) [3]
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- Melbourne
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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Country [2]
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United States of America
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State/province [2]
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California
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Country [3]
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United States of America
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State/province [3]
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Connecticut
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Country [4]
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United States of America
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State/province [4]
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Ohio
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Country [5]
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United States of America
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State/province [5]
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Pennsylvania
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Country [6]
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Belgium
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State/province [6]
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Antwerpen
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Country [7]
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Italy
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State/province [7]
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Ranica
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Country [8]
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Netherlands
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State/province [8]
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Leiden
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Country [9]
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Netherlands
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State/province [9]
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Nijmegen
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alexion Pharmaceuticals, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The primary purpose of this study was to evaluate the efficacy of 12 months of oral
ACH-0144471 (also known as danicopan and ALXN2040) in participants with C3G or IC-MPGN based
on histologic scoring and proteinuria.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03459443
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03459443
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