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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03459443




Registration number
NCT03459443
Ethics application status
Date submitted
26/02/2018
Date registered
9/03/2018

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
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
Secondary ID [1] 0 0
2017-002674-39
Secondary ID [2] 0 0
ACH471-205
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
C3 Glomerulonephritis 0 0
C3 Glomerulopathy 0 0
Immune Complex Membranoproliferative Glomerulonephritis 0 0
IC-MPGN 0 0
Dense Deposit Disease 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Kidney disease
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline In Composite Biopsy Score At End Of Initial 12-Month Treatment Period
Timepoint [1] 0 0
Baseline, end of initial 12-Month Treatment Period
Primary outcome [2] 0 0
Participants With Reduction In Proteinuria At End Of Initial 12-Month Treatment Period
Timepoint [2] 0 0
Baseline, end of initial 12-Month Treatment Period
Secondary outcome [1] 0 0
Change From Baseline In Proteinuria At End Of Initial 12-Month Treatment Period
Timepoint [1] 0 0
Baseline, end of initial 12-Month Treatment Period
Secondary outcome [2] 0 0
Percent Change From Baseline In Proteinuria At End Of Initial 12-Month Treatment Period
Timepoint [2] 0 0
Baseline, end of initial 12-Month Treatment Period
Secondary outcome [3] 0 0
Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To End Of Initial 12-Month Treatment Period
Timepoint [3] 0 0
End of initial 12-Month Treatment Period
Secondary outcome [4] 0 0
Change From Baseline In eGFR At End Of Initial 12-Month Treatment Period
Timepoint [4] 0 0
Baseline, end of initial 12-Month Treatment Period
Secondary outcome [5] 0 0
Participants With Significant Improvement In eGFR Relative To Baseline At End Of Initial 12-Month Treatment Period
Timepoint [5] 0 0
Baseline, end of initial 12-Month Treatment Period
Secondary outcome [6] 0 0
Change From Baseline in eGFR Over 12 Months of Treatment For Participants Meeting eGFR Inclusion Criteria
Timepoint [6] 0 0
End of initial 12-Month Treatment Period
Secondary outcome [7] 0 0
Change From Baseline In Measured GFR At The End Of The Initial 12-Month Treatment Period
Timepoint [7] 0 0
End of initial 12-Month Treatment Period

Eligibility
Key inclusion criteria
Key

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

Key
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/06/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
29/03/2021
Sample size
Target
Accrual to date
Final
22
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Clinical Study Site - Sydney
Recruitment hospital [2] 0 0
Clinical Study Site - Brisbane
Recruitment hospital [3] 0 0
Clinical Study Site - Melbourne
Recruitment postcode(s) [1] 0 0
- Sydney
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
Belgium
State/province [6] 0 0
Antwerpen
Country [7] 0 0
Italy
State/province [7] 0 0
Ranica
Country [8] 0 0
Netherlands
State/province [8] 0 0
Leiden
Country [9] 0 0
Netherlands
State/province [9] 0 0
Nijmegen

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Supporting document/s available: Statistical analysis plan (SAP), Clinical study report (CSR)
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03459443