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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 : September 2, 2020
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Study Description
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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

Condition or disease Intervention/treatment Phase
IgA Nephropathy Lupus Nephritis Membranous Nephropathy C3 Glomerulonephritis Dense Deposit Disease Drug: APL-2 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : January 22, 2018
Actual Primary Completion Date : April 16, 2020
Estimated Study Completion Date : December 2022

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Arms and Interventions
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Arm Intervention/treatment
Experimental: APL-2
Open Label, Study Drug, APL-2
 Drug: APL-2
APL-2 administered as a daily subcutaneous infusion for 48 weeks


Outcome Measures
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Primary Outcome Measures :
  1. Proteinuria [ Time Frame: 48 weeks ]
    Proteinuria reduction by 50%, calculated by the change in uPCR from baseline to Week 48.


Secondary Outcome Measures :
  1. Changes of Disease Specific Biomarkers [ Time Frame: Week 48 ]
  2. Complete clinical remission defined as normalization of proteinuria as defined by <200 mg/g uPCR at Week 48 [ Time Frame: Week 48 ]
  3. Stabilization or improvement in estimated Glomerular Filtration Rate (eGFR) from baseline to Week 48 [ Time Frame: Week 48 ]

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

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.
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03453619


Locations
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Sponsors and Collaborators
Apellis Pharmaceuticals, Inc.
More Information
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Responsible Party: Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03453619    
Other Study ID Numbers: APL2-201
First Posted: March 5, 2018    Key Record Dates
Last Update Posted: September 2, 2020
Last Verified: August 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Kidney Diseases
Nephritis
Lupus Nephritis
Glomerulonephritis, IGA
Glomerulonephritis
Glomerulonephritis, Membranous
 Glomerulonephritis, Membranoproliferative
Urologic Diseases
Lupus Erythematosus, Systemic
Connective Tissue Diseases
Autoimmune Diseases
Immune System Diseases