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

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ClinicalTrials.gov Identifier: NCT03453619
Recruitment Status : Recruiting
First Posted : March 5, 2018
Last Update Posted : April 20, 2018
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 48 participants
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
Estimated Primary Completion Date : July 22, 2019
Estimated Study Completion Date : October 22, 2019


Arm Intervention/treatment
Experimental: APL-2
Open Label, Study Drug, APL-2
Drug: APL-2
APL-2 is a C3 complement mediator administered as daily subcutaneous infusions




Primary Outcome Measures :
  1. Proteinuria [ Time Frame: 16 weeks ]
    Change From Baseline in Proteinuria at 16 weeks



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Ages Eligible for Study:   18 Years to 69 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Prior to screening, patients with at least 6 months of sufficient medical history will begin with a 4 week screening period, all other patients will enter the 12 week screening period.

Medical History Inclusion Criteria:

Documented at a minimum of 2 separate occasions over a duration no less than 6 months:

  • Stable proteinuria >1500 mg/day
  • Average uACR > 600 mg albumin/g creatinine
  • Blood pressure <140/90mm/Hg If applicable; stable dose of ACE/ARB for at least 6 months prior to screening.

    1. Patients of at least 18 and less than 70 years of age at screening, 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)
    2. Patients must have a diagnosis of IgAN, LN, Primary MN, or C3G confirmed by renal biopsy and required measurements performed prior to study participation

      1. IgAN: Prior biopsy results for C3 and C4d staining should be made available
      2. LN: Diagnostic biopsy showing proliferative focal or diffuse lesions (Class III or IV) by renal biopsy with or without Class V (membranous) changes. Subject should have either a repeat biopsy in the last 6 months, or evidence of disease activity (nephritic changes on urinalysis or nephrotic changes)
      3. Primary MN: PLA2R high titer plus nephrotic range proteinuria ( >3.5 g proteinuria/day)
      4. C3G: Low serum C3 level and presence of C3 nephritic factor
    3. Have 24 hour proteinuria 1500 mg/day (determined by 24 hour urine collection) and an average urine albumin/creatinine ratio > 600 mg albumin/g creatinine determined by spot urine samples collected consecutively during each visit during the screening period (Visits 1/3a, 2, 3b) prior to 24 hour urine collection.
    4. If on immunosuppressive treatment (e.g. cyclophosphamide, mycophenolate mofetil, calcineurin inhibitors, steroids), have been on a stable dose for at least 2 months prior to Screening Visit 1/3a with no expected change in the dose for the study duration
    5. eGFR ≥ 30 mL/min/1.73 m2 calculated by CKD-EPI creatinine equation at screening visit 1/3a
    6. Have a systolic blood pressure of < 140 mmHg and a diastolic blood pressure of < 90 mmHg at rest and if on physician-directed, stable, optimized treatment with angiotensin converting enzyme inhibitors (ACEI) and/or angiotensin receptor blockers (ARB) have received treatment for at least 3 or 6 months prior to the Screening Visit 1 or 3a, respectively.
    7. Willing to receive vaccinations against Neisseria meningitides 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, OR able to provide documentary evidence of Neisseria meningitides types A, C, W, Y and B (administered as two separate vaccinations), Pneumococcal conjugate vaccine or Pneumococcal polysaccharide vaccine 23 (PCV13 or PPSV23, respectively) and Haemophilus influenza Type B (Hib) vaccination within 2 years prior to Day -14.
    8. Women of child-bearing potential (WOCBP) must have a negative blood pregnancy test at screening and must agree to use protocol defined methods of contraception from screening through 3 months after last dose of APL-2
    9. Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm from screening through 3 months after receiving last dose of APL-2
    10. Willing and able to give informed consent
    11. Willing and able to self-administer APL-2 (administration by caregiver will be allowed)

Exclusion Criteria:

  1. Hemoglobin < 9.0 g/dL at screening Visits 1/3a and 3b
  2. Platelet count < 100,000/mm3 at screening Visits 1/3a and 3b
  3. Absolute neutrophil count < 500 cells/mm3 at screening Visits 1/3a and 3b
  4. ALT or AST > 3.0 x the upper limit of normal at screening Visits 1/3a and 3b
  5. Use of belimumab, eculizumab, or rituximab within 6 months prior to Screening Visit 1/3a
  6. Previous treatment with APL-2
  7. History of renal transplant
  8. Diagnosis of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection, or positive serology at screening Visits 1/3a and 3b
  9. Any active infection requiring antibiotic treatment during the screening or treatment periods
  10. Malignancy except for cured basal or squamous cell skin cancer, curatively treated in situ disease or have been disease-free for ≥5 years or more from cancer
  11. Secondary IgA nephropathy: e.g. due to lupus, liver cirrhosis, IgA Vasculitis (Henoch-Schonlein purpura)
  12. Membranous Nephropathy secondary to another known disease
  13. Malignant/uncontrolled hypertension (>160mm systolic or 110mmHg diastolic)
  14. Current unstable kidney function for other reasons, e.g. toxin induced acute kidney injury
  15. Presence or suspicion of active bacterial or viral infection or severe recurrent bacterial infections
  16. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period
  17. Pregnant, breast-feeding, or intending to conceive during the course of the study
  18. Inability to cooperate or any condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study
  19. Unwillingness to receive or intolerant of SC injections of study medication or known allergy to ingredients in APL-2.
  20. Positive results for drug abuse (upon urinary drug screen) or alcohol dependence at screening (Visit 1/3a)

Information from the National Library of Medicine

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


Contacts
Contact: Federico Grossi, MD, PhD 617-977-5701 clinicaltrials@apellis.com

Locations
United States, California
Apellis Investigational Site Not yet recruiting
Stanford, California, United States, 94305
United States, District of Columbia
Apellis Investigational Site Recruiting
Washington, District of Columbia, United States, 20037
United States, Florida
Apellis Investigational Site Recruiting
Coral Gables, Florida, United States, 33134
United States, Maryland
Apellis Investigational Site Recruiting
Takoma Park, Maryland, United States, 20912
United States, Missouri
Apellis Investigational Site Recruiting
Kansas City, Missouri, United States, 64111
United States, New York
Apellis Investigational Site Recruiting
Bronx, New York, United States, 10461
United States, North Carolina
Apellis Investigational Site Recruiting
Wilmington, North Carolina, United States, 28401
United States, Virginia
Apellis Investigational Site Recruiting
Alexandria, Virginia, United States, 22304
Apellis Investigational Site Recruiting
Chesapeake, Virginia, United States, 23320
United States, Wisconsin
Apellis Investigational Site Recruiting
Wauwatosa, Wisconsin, United States, 53226
Sponsors and Collaborators
Apellis Pharmaceuticals, Inc.

Responsible Party: Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03453619     History of Changes
Other Study ID Numbers: APL2-201
First Posted: March 5, 2018    Key Record Dates
Last Update Posted: April 20, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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