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NCT Number: NCT07213960

A Sequential Phase 2/3 Study of APL2 in Patients With Focal Segmental Glomerulosclerosis

This is a sequential phase 2/3 study to evaluate the efficacy and safety of twice-weekly subcutaneous (SC) infusions of APL2 in patients diagnosed with FSGS. The initial phase 2 portion is a single-arm, open-label study in adults diagnosed with FSGS. Phase 2 will commence prior to randomizing for phase 3. The phase 3 portion of the study is a randomized, placebo-controlled, double-blinded, multicenter study in adults and adolescents diagnosed with FSGS.

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Key information

Conditions

FSGS

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Investigator Site 1, Chicago, Illinois, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age
  • Phase 2: adults aged ≥18 years
  • Phase 3: adults aged ≥18 years; if and where approved, adolescents (aged 12--17 years) at the time of signing the informed consent and assent form
  • Weight ≥30 kg and ≤100 kg at screening
  • FSGS diagnosis
  • Phase 2: primary, genetic, or undetermined FSGS diagnosed by kidney biopsy
  • Phase 3: primary, genetic, or undetermined FSGS diagnosed by kidney biopsy or by recognized podocyte genetic mutation
  • At least 1.5 g/day of proteinuria on a screening 24-hour urine collection and a uPCR of at least 1.5 g/g in at least 2 FMU samples collected during screening
  • Estimated glomerular filtration rate (eGFR) ≥25 mL/min/1.73 m2
  • Stable regimen for FSGS treatment for at least 12 weeks prior to randomization, with no planned or anticipated adjustments or dose changes to the stable treatment regimen

Exclusion criteria

  • Previous exposure to APL2
  • Evidence of improving kidney disease in the 8 weeks prior to screening or during the screening period according to available data
  • FSGS secondary to another condition (eg, infectious, diabetic, drug-induced, obesity, prematurity, sickle-cell, vesicoureteral reflux, congenital anomalies of the kidney, and urinary tract)
  • Type 1 or uncontrolled (HbA1C ≥8%) type 2 diabetes mellitus
  • History of kidney transplant
  • Current or prior diagnosis of HIV, hepatitis B, or hepatitis C infection or positive serology or viral load during screening that is indicative of active infection with any of these viruses
  • Hypersensitivity to APL2 or to any of the excipients
  • Significant other kidney disease that would, in the opinion of the investigator, confound interpretation of study results
  • Use of rituximab, belimumab, or any approved or investigational anticomplement therapy within 5 half-lives of that product prior to the screening period

Treatment and study plan

APL2

Drug

Complement (C3) Inhibitor

Placebo

Other

Sterile solution of equal volume to active arm

Primary outcomes

  1. Phase 2: Evaluate the efficacy of APL2 in terms of change from baseline in log-transformed urine protein to creatinine ratio (uPCR)

    Time frame: Baseline to Week 12

    Change from Baseline in Log-Transformed uPCR will be based on triplicate first morning urine (FMU)

  2. Phase 3: Change from baseline in log-transformed urine protein to creatinine ratio (uPCR)

    Time frame: Baseline to Week 52

    Change from baseline in log-transformed uPCR will be based on triplicate first morning urine (FMU)

Secondary outcomes

  1. Phase 2: Evaluate the efficacy of APL2 in terms of change from baseline in log-transformed urine albumin to creatinine ratio (uACR)

    Time frame: Baseline to Week 12

    Change From Baseline in Log-Transformed uACR will be based on first morning urine (FMU)

  2. Phase 3: Change from Baseline in log-transformed urine protein to creatinine ratio (uPCR)

    Time frame: Baseline to Week 104

    Change from baseline in log-transformed uPCR will be based on triplicate first morning urine (FMU)

  3. Phase 3: Proportion of participants achieving Complete Remission

    Time frame: Week 104

    Complete Remission is defined as participants who have achieved uPCR <0.3 g/g

  4. Phase 3: Slope of estimated Glomerular Filtration Rate (eGFR)

    Time frame: Baseline to Week 104

    The annualized eGFR slope

Study contacts

Contact information is provided by the study sponsor or research team.

Apellis Clinical Trial Information Line

CONTACT

[email protected]

833-284-6361

Sponsors and collaborators

Lead sponsor

Apellis Pharmaceuticals, Inc.

Industry

Registry information

Official study title

A Sequential Phase 2/3, Single-Arm, Open-Label Study in Adults Followed by a Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of APL2 in Adults and Adolescents With Focal Segmental Glomerulosclerosis

Important dates

Study start
2026
Primary completion
2029
Study completion
2029
First posted
Oct 9, 2025
Registry last updated
Sep 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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