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出境医 / 临床实验 / A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy

A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy

Study Description
Brief Summary:
The purpose of this study is to evaluate the effectiveness and safety of IONIS-FB-LRx, an antisense inhibitor of complement factor B messenger ribonucleic acid (CFB mRNA), and to evaluate the effect of IONIS-FB-LRx on plasma factor B (FB) levels and serum AH50, CH50 activity in participants with primary immunoglobulin A (IgA) nephropathy.

Condition or disease Intervention/treatment Phase
Primary IgA Nephropathy Drug: IONIS-FB-LRx Phase 2

Detailed Description:
This is a Phase 2, single arm open-label clinical study in up to 10 participants that will consist of screening period (which may include a titration of maximum dose/maximally tolerated dose of angiotensin converting enzyme (ACE) and/or angiotensin II receptor blocker (ARB)), a 24-week treatment period and a 12-week post-treatment follow-up evaluation period.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Phase 2a Clinical Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Subjects With Primary IgA Nephropathy
Actual Study Start Date : December 4, 2019
Estimated Primary Completion Date : January 2021
Estimated Study Completion Date : January 2021
Arms and Interventions
Arm Intervention/treatment
Experimental: IONIS-FB-LRx Drug: IONIS-FB-LRx
Participants will receive IONIS-FB-LRx, by subcutaneous injection (SC) at Weeks 1, 3, 5, and every 4 weeks through Week 25.
Outcome Measures
Primary Outcome Measures :
  1. Percent Reduction in 24-hour Urine Protein Excretion [ Time Frame: Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured) ]

Secondary Outcome Measures :
  1. Absolute Reduction in 24-hour Urine Protein Excretion [ Time Frame: Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured) ]
  2. Absolute Reduction in Albuminuria (UACr Ratio) [ Time Frame: Baseline to Week 29 ]
  3. Absolute Reduction in Proteinuria (UPCr Ratio) [ Time Frame: Baseline to Week 29 ]
  4. Percent Change from Baseline in Plasma Factor B (FB) [ Time Frame: Up to Week 29 ]
  5. Percent Change from Baseline in Plasma AH50 [ Time Frame: Up to Week 29 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  • Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal OR use a highly effective method of birth control
  • Biopsy-proven primary immunoglobulin A (IgA) nephropathy
  • Hematuria
  • Proteinuria

Exclusion Criteria

  • Clinically significant abnormalities in medical history (e.g., dementia, stroke, acute coronary syndrome, thrombocytopenia, or major surgery within 6 months of Screening)
  • Diagnosis of primary or secondary immunodeficiencies of B-lymphocyte function, splenectomy, or history of recurrent meningococcal disease
  • Active infection 30 days prior to study
  • Estimated glomerular filtration rate (eGFR) ≤ 40 milliliters per minute per 1.73 square meters (mL/min/1.73m^2) using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)
  • Presence of another renal disease including, but not limited to, diabetes and/or diabetic nephropathy, thin basement membrane disease, Alport's disease, IgA Nephritis (Henoch-Schonlein purpura), lupus nephritis, Minimal Change Disease, post-infectious glomerulonephritis or any other cause of proteinuria or secondary IgA nephropathy (including, but not limited to Celiac disease, Crohn's disease, human immunodeficiency virus (HIV), liver cirrhosis)
  • History of renal transplant or another organ transplant
  • Treatment with another investigational drug, biological agent, or device within 6 months of screening investigational agent, whichever is longer
  • Administration of immunosuppressive/immunomodulatory medication 12 months prior to study drug administration, except for short-term treatments.
  • Other protocol-specified inclusion/exclusion criteria may apply
Contacts and Locations

Contacts
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Contact: Ionis Pharmaceuticals 800-679-4747 patients@ionisph.com

Locations
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Australia, New South Wales
IONIS Investigative Site Recruiting
Liverpool, New South Wales, Australia, 2170
Contact    (02) 8738 3710    Michael.suranyi@health.nsw.gov.au   
IONIS Investigative Site Recruiting
St Leonards, New South Wales, Australia, 2065
Contact    (04) 5756 7527    muhgeot.wong@sydney.edu.au   
Australia, Victoria
IONIS Investigative Site Recruiting
Parkville, Victoria, Australia, 3050
Contact    (03) 9342 8530    research@mh.org.au   
Canada, British Columbia
IONIS Investigative Site Recruiting
Vancouver, British Columbia, Canada, V6Z 1Y6
Contact    604-806-9460    kvela@providencehealth.bc.ca   
Canada, Ontario
IONIS Investigative Site Recruiting
Toronto, Ontario, Canada, M4G 3E8
Contact    416-480-6100 x 3247    anny.gonzalez@sunnybrook.ca   
Contact    416-480-6100 x 83705    tatjana.sukovic@sunnybrook.ca   
New Zealand
IONIS Investigative Site Recruiting
Christchurch, New Zealand, 8011
Contact    +64 (0) 3 3729 477    research@ccst.co.nz   
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Tracking Information
First Submitted Date  ICMJE July 8, 2019
First Posted Date  ICMJE July 10, 2019
Last Update Posted Date March 3, 2020
Actual Study Start Date  ICMJE December 4, 2019
Estimated Primary Completion Date January 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 28, 2020) 		Percent Reduction in 24-hour Urine Protein Excretion [ Time Frame: Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured) ]
Original Primary Outcome Measures  ICMJE
 (submitted: July 8, 2019)
  • Percent Reduction in 24-hour Urine Protein Excretion [ Time Frame: Baseline to Week 29 ]
  • 24-hour Urine Protein Excretion Four Weeks after Last Dose of Study Drug [ Time Frame: Up to Week 29 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 28, 2020)
  • Absolute Reduction in 24-hour Urine Protein Excretion [ Time Frame: Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured) ]
  • Absolute Reduction in Albuminuria (UACr Ratio) [ Time Frame: Baseline to Week 29 ]
  • Absolute Reduction in Proteinuria (UPCr Ratio) [ Time Frame: Baseline to Week 29 ]
  • Percent Change from Baseline in Plasma Factor B (FB) [ Time Frame: Up to Week 29 ]
  • Percent Change from Baseline in Plasma AH50 [ Time Frame: Up to Week 29 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: July 8, 2019)
  • Absolute Reduction in 24-hour Urine Protein Excretion [ Time Frame: Baseline to Week 29 ]
  • 24-hour Urine Protein Excretion Four Weeks after Last Dose of Study Drug (if Terminates Prior to Week 25) [ Time Frame: Baseline to Week 29 ]
  • Absolute Reduction in Albuminuria (UACr Ratio) [ Time Frame: Baseline to Week 29 ]
  • Absolute Reduction in Proteinuria (UPCr Ratio) [ Time Frame: Baseline to Week 29 ]
  • Percent Change from Baseline in Plasma Factor B (FB) [ Time Frame: Up to Week 29 ]
  • Percent Change from Baseline in Plasma AH50 [ Time Frame: Up to Week 29 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy
Official Title  ICMJE An Open-Label Phase 2a Clinical Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Subjects With Primary IgA Nephropathy
Brief Summary The purpose of this study is to evaluate the effectiveness and safety of IONIS-FB-LRx, an antisense inhibitor of complement factor B messenger ribonucleic acid (CFB mRNA), and to evaluate the effect of IONIS-FB-LRx on plasma factor B (FB) levels and serum AH50, CH50 activity in participants with primary immunoglobulin A (IgA) nephropathy.
Detailed Description This is a Phase 2, single arm open-label clinical study in up to 10 participants that will consist of screening period (which may include a titration of maximum dose/maximally tolerated dose of angiotensin converting enzyme (ACE) and/or angiotensin II receptor blocker (ARB)), a 24-week treatment period and a 12-week post-treatment follow-up evaluation period.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Primary IgA Nephropathy
Intervention  ICMJE Drug: IONIS-FB-LRx
Participants will receive IONIS-FB-LRx, by subcutaneous injection (SC) at Weeks 1, 3, 5, and every 4 weeks through Week 25.
Study Arms  ICMJE Experimental: IONIS-FB-LRx
Intervention: Drug: IONIS-FB-LRx
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 8, 2019) 		10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 2021
Estimated Primary Completion Date January 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria

  • Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal OR use a highly effective method of birth control
  • Biopsy-proven primary immunoglobulin A (IgA) nephropathy
  • Hematuria
  • Proteinuria

Exclusion Criteria

  • Clinically significant abnormalities in medical history (e.g., dementia, stroke, acute coronary syndrome, thrombocytopenia, or major surgery within 6 months of Screening)
  • Diagnosis of primary or secondary immunodeficiencies of B-lymphocyte function, splenectomy, or history of recurrent meningococcal disease
  • Active infection 30 days prior to study
  • Estimated glomerular filtration rate (eGFR) ≤ 40 milliliters per minute per 1.73 square meters (mL/min/1.73m^2) using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)
  • Presence of another renal disease including, but not limited to, diabetes and/or diabetic nephropathy, thin basement membrane disease, Alport's disease, IgA Nephritis (Henoch-Schonlein purpura), lupus nephritis, Minimal Change Disease, post-infectious glomerulonephritis or any other cause of proteinuria or secondary IgA nephropathy (including, but not limited to Celiac disease, Crohn's disease, human immunodeficiency virus (HIV), liver cirrhosis)
  • History of renal transplant or another organ transplant
  • Treatment with another investigational drug, biological agent, or device within 6 months of screening investigational agent, whichever is longer
  • Administration of immunosuppressive/immunomodulatory medication 12 months prior to study drug administration, except for short-term treatments.
  • Other protocol-specified inclusion/exclusion criteria may apply
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Ionis Pharmaceuticals 800-679-4747 patients@ionisph.com
Listed Location Countries  ICMJE Australia,   Canada,   New Zealand
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04014335
Other Study ID Numbers  ICMJE ISIS 696844-CS4
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Ionis Pharmaceuticals, Inc.
Study Sponsor  ICMJE Ionis Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Ionis Pharmaceuticals, Inc.
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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