FSGSPhase 2/3NCT07213960
A Sequential Phase 2/3 Study of APL2 in Patients With Focal Segmental Glomerulosclerosis
A Phase 2/3 study testing APL2 in people with FSGS, run by Apellis Pharmaceuticals, Inc. at 2 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- FSGS
- What is being tested?
- APL2
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 270 participants
- Where
- 2 sitesUnited States
- Study dates
- Dec 2026to Dec 2029
- Phase
- Phase 2/3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Investigator Site 1
Chicago, Illinois, 60643
- Recruiting
Investigator Site 2
New York, New York, 10032
About this study
Official 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
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.
- Conditions
- FSGS
- Being tested
- APL2, Placebo
- Keywords
- Glomerulosclerosis, Focal Segmental, Child, Adult, Peptides, Cyclic, Complement Inactivator Proteins
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APL2Drug
Complement (C3) Inhibitor
PlaceboOther
Sterile solution of equal volume to active arm
| Study group | Role | What they receive |
|---|---|---|
| Phase 2 - APL2 | Experimental | Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly Receives: Drug: APL2 |
| Phase 3 - APL2 | Experimental | Adults: Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly
Adolescents:
≥50 Kg: Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly 35 to <50kg: First sub-cutaneous infusion of 648mg (12 mL) followed by 810mg (15 mL) every infusion thereafter (i.e. twice weekly) 30 to <35kg: First \& Second sub-cutaneous infusion of 540mg (10mL) followed by 648mg (12 mL) every infusion thereafter (twice weekly) Receives: Drug: APL2 |
| Phase 3 - Placebo | Placebo comparator | Subcutaneous infusions of a sterile solution, twice-weekly, and equivalent in volume to the active arm based on participant's age and weight Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 2: Evaluate the efficacy of APL2 in terms of change from baseline in log-transformed urine protein to creatinine ratio (uPCR)
Change from Baseline in Log-Transformed uPCR will be based on triplicate first morning urine (FMU)
Measured: Baseline to Week 12
Phase 3: Change from baseline in log-transformed urine protein to creatinine ratio (uPCR)
Change from baseline in log-transformed uPCR will be based on triplicate first morning urine (FMU)
Measured: Baseline to Week 52
Secondary outcomes (4)
Phase 2: Evaluate the efficacy of APL2 in terms of change from baseline in log-transformed urine albumin to creatinine ratio (uACR)
Change From Baseline in Log-Transformed uACR will be based on first morning urine (FMU)
Measured: Baseline to Week 12
Phase 3: Change from Baseline in log-transformed urine protein to creatinine ratio (uPCR)
Change from baseline in log-transformed uPCR will be based on triplicate first morning urine (FMU)
Measured: Baseline to Week 104
Phase 3: Proportion of participants achieving Complete Remission
Complete Remission is defined as participants who have achieved uPCR <0.3 g/g
Measured: Week 104
Phase 3: Slope of estimated Glomerular Filtration Rate (eGFR)
The annualized eGFR slope
Measured: Baseline to Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2025
- Study startDec 2026 (estimated)
- Primary completionOct 2029 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for FSGSStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 270 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07213960
- Sponsor's protocol ID
- APL2-FSG-319
- Status last verified
- Aug 2026
- First submitted
- 15 Sep 2025
- Last updated
- 2 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No