NCT07213960 · Apellis Pharmaceuticals, Inc.
A Sequential Phase 2/3 Study of APL2 in Patients With Focal Segmental Glomerulosclerosis
What this study is about
This is a sequential phase 2/3 study to evaluate the effectiveness and safety of twice-weekly injected under the skin (SC) infusions of APL2 in patients diagnosed with FSGS. The initial phase 2 portion is a single-treatment group$1, where both patients and doctors know the treatment given study in adults diagnosed with FSGS. Phase 2 will commence prior to randomizing for phase 3.
View original scientific description
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.
Interventions
DRUG
APL2
Complement (C3) Inhibitor
OTHER
Placebo
Sterile solution of equal volume to active arm
Primary outcome measures
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)
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)
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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
Where
- Chicago, Illinois
- New York, New York
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Sep 2, 2026 · Source of record for eligibility and locations