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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 : August 14, 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 and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients of at least 18 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
  3. IgAN: Prior biopsy results for C3 and C4d staining should be made available
  4. 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)
  5. Primary MN: PLA2R high titer plus nephrotic range proteinuria (defined as uPCR >2350mg/g)
  6. C3G: Low serum C3 level and presence of C3 nephritic factor
  7. Have proteinuria >750mg/g (calculated by spot uPCR on 24 hour urine collection) collected during the first screening visit (Visits 1/3a).
  8. If on immunosuppressive treatment (e.g. cyclophosphamide, mycophenolate mofetil, calcineurin inhibitors, steroids), have been on a stable dose for at least 2 months (6 months for 4-week screening period) prior to Screening Visit 1/3a with no expected change in the dose for the study duration
  9. eGFR ≥ 30 mL/min/1.73 m2 calculated by CKD-EPI creatinine equation at screening visit 1/3a
  10. Have an average systolic blood pressure of < 140 mmHg and a diastolic blood pressure of < 90 mmHg at rest during the screening period 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 months (6 months for 4-week screening period) prior to the Screening Visit 1/3a.
  11. 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.
  12. 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
  13. 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
  14. Willing and able to give informed consent
  15. 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 < 1000 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 solid organ transplant
  8. Diagnosis of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection, or positive serology at screening Visits 1/3a and 3b (previous HBV or HCV diagnosis cleared by treatment is allowed)
  9. 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
  10. Secondary IgA nephropathy: e.g. due to lupus, liver cirrhosis, IgA Vasculitis (Henoch-Schonlein purpura)
  11. Membranous Nephropathy secondary to another known disease
  12. Current unstable kidney function for other reasons, e.g. toxin induced acute kidney injury
  13. Presence or suspicion of active bacterial or viral infection or severe recurrent bacterial infections
  14. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period
  15. Pregnant, breast-feeding, or intending to conceive during the course of the study
  16. 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
  17. Unwillingness to receive or intolerant of SC infusions of study medication or known allergy to ingredients in APL-2.
  18. Positive results for drug abuse (upon urinary drug screen) or alcohol dependence at screening (Visit 1/3a). Documented evidence of prescribed marijuana use is not exclusionary.

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

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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: August 14, 2018
Last Verified: August 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