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Phase II Study Assessing Safety and Efficacy of APL-2 in Glomerulopathies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03453619
Recruitment Status : Active, not recruiting
First Posted : March 5, 2018
Last Update Posted : February 7, 2020
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE February 27, 2018
First Posted Date  ICMJE March 5, 2018
Last Update Posted Date February 7, 2020
Actual Study Start Date  ICMJE January 22, 2018
Estimated Primary Completion Date April 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 5, 2019)
Proteinuria [ Time Frame: 48 weeks ]
Proteinuria reduction by 50%, calculated by the change in uPCR from baseline to Week 48.
Original Primary Outcome Measures  ICMJE
 (submitted: February 27, 2018)
Changes in Proteinuria [ Time Frame: 16 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 5, 2019)
  • Changes of Disease Specific Biomarkers [ Time Frame: Week 48 ]
  • Complete clinical remission defined as normalization of proteinuria as defined by <200 mg/g uPCR at Week 48 [ Time Frame: Week 48 ]
  • Stabilization or improvement in estimated Glomerular Filtration Rate (eGFR) from baseline to Week 48 [ Time Frame: Week 48 ]
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase II Study Assessing Safety and Efficacy of APL-2 in Glomerulopathies
Official Title  ICMJE A Phase 2 Study to Evaluate the Safety and Biologic Activity of APL- 2 in Patients With IgA Nephropathy, Lupus Nephritis, Primary Membranous Nephropathy, or C3 Glomerulopathy (C3 Glomerulonephritis and Dense Deposit Disease)
Brief Summary This is a Phase II trial assessing the safety and preliminary efficacy of daily APL-2 subcutaneous infusion administered for 16 weeks with a 6 month safety follow up, in patients with glomerulopathies
Detailed Description Not Provided
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
  • IgA Nephropathy
  • Lupus Nephritis
  • Membranous Nephropathy
  • C3 Glomerulonephritis
  • Dense Deposit Disease
Intervention  ICMJE Drug: APL-2
APL-2 administered as a daily subcutaneous infusion for 48 weeks
Study Arms  ICMJE Experimental: APL-2
Open Label, Study Drug, APL-2
Intervention: Drug: APL-2
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 Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: December 5, 2019)
21
Original Estimated Enrollment  ICMJE
 (submitted: February 27, 2018)
36
Estimated Study Completion Date  ICMJE April 2020
Estimated Primary Completion Date April 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Patients of at least 18 years of age at screening (16 years of age for C3G), able to provide written informed consent, and able to understand and comply with all scheduled procedures and other requirements of the study by the opinion of Principal Investigator (PI)
  • Patients must have a diagnosis of IgAN, LN, Primary MN, or C3G confirmed by renal biopsy and required measurements performed prior to study participation

    • IgAN: Prior biopsy results for C3 and C4d staining should be made available
    • LN: Diagnostic biopsy showing proliferative focal, diffuse, or membranous lesions (Class III, IV or V, respectively) by renal biopsy. Subject should have either a biopsy in the last 6 months, or evidence of disease activity (nephritic changes on urinalysis or nephrotic changes)
    • Primary MN: PLA2R positive titer plus nephrotic range proteinuria (defined as uPCR >2350 mg/g)
    • C3G plus one of the following: Low serum C3 level or historical renal biopsy within the last 3 years
  • Have proteinuria >750 mg/g (calculated by uPCR on 24 hour urine collection) collected during the first screening visit (Visit 3a).
  • eGFR≥30mL/min/1.73 m2 calculated by CKD-EPI creatinine equation at screening visit 3a and currently not on dialysis
  • Must have stable or worsening renal disease, on stable and optimized treatment, in the opinion of the PI, for at least 2 months prior to the first dose of APL-2 (Visit 4); treatments may include, but are not limited to, immunosuppressive agents, anti-hypertensives and/or anti-proteinurics.
  • Willing to receive vaccinations against Neisseria meningitidis at least 2 weeks prior to dosing on Day 1 with a booster on Day 56 (for both vaccinations) and Pneumococcal and Hib vaccines at least 2 weeks prior to dosing on Day 1.

Exclusion Criteria:

  • Absolute neutrophil count <1000 cells/mm3 at screening Visits 3a and 3b
  • ALT or AST >3.0 x the upper limit of normal at screening Visits 3a and 3b
  • Previous treatment with APL-2
  • History of solid organ transplant
  • Diagnosis of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection, or positive serology at screening Visits 3a and 3b (previous HBV or HCV diagnosis cleared by treatment is allowed)
  • Renal disease secondary to another condition (e.g. infection, malignancy, monoclonal gammopathy, or a medication)
  • Presence or suspicion of active bacterial or viral infection or severe recurrent bacterial infections
  • Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period
  • Unwillingness to receive or intolerant of SC infusions of study medication or known allergy to ingredients in APL-2.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03453619
Other Study ID Numbers  ICMJE APL2-201
Has Data Monitoring Committee Yes
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 Not Provided
Responsible Party Apellis Pharmaceuticals, Inc.
Study Sponsor  ICMJE Apellis Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Apellis Pharmaceuticals, Inc.
Verification Date February 2020

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