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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07213960
- Acronym / sponsor ID
- APL2-FSG-319
- Conditions
- FSGS
- Keywords
- Glomerulosclerosis, Focal Segmental, Child, Adult, Peptides, Cyclic, Complement Inactivator Proteins
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 270 participants (estimated)
- Lead sponsor
- Apellis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Dec 2026 (estimated)
- Primary completion
- Oct 2029 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 9 Oct 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Phase 2 - APL2 | Experimental | Drug: APL2Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly |
| Phase 3 - APL2 | Experimental | Drug: APL2Adults: 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) |
| Phase 3 - Placebo | Placebo comparator | Other: PlaceboSubcutaneous infusions of a sterile solution, twice-weekly, and equivalent in volume to the active arm based on participant's age and weight |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Age
- Weight ≥30 kg and ≤100 kg at screening
- FSGS diagnosis
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| APL2Complement (C3) Inhibitor | Drug | Phase 2 - APL2, Phase 3 - APL2 |
| PlaceboSterile solution of equal volume to active arm | Other | Phase 3 - Placebo |
Primary outcome
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)
4 secondary outcomes
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
Phase 3: Change from Baseline in log-transformed urine protein to creatinine ratio (uPCR)
Time frame: Baseline to Week 104
Phase 3: Proportion of participants achieving Complete Remission
Time frame: Week 104
Phase 3: Slope of estimated Glomerular Filtration Rate (eGFR)
Time frame: Baseline to Week 104
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing APL2 in people with FSGS, run by Apellis Pharmaceuticals, Inc. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 2 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Apellis Clinical Trial Information Line (833-284-6361, clinicaltrials@apellis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2026.