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

NCT ID: NCT07213960

Last Updated: 2025-10-09

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 270 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-12-31
• Study Completion Date: 2029-12-31

Brief 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. 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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Phase 2 - APL2

Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly

Group Type: EXPERIMENTAL

APL2

Intervention Type: DRUG

Complement (C3) Inhibitor

Phase 3 - APL2

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)

Group Type: EXPERIMENTAL

APL2

Intervention Type: DRUG

Complement (C3) Inhibitor

Phase 3 - Placebo

Subcutaneous infusions of a sterile solution, twice-weekly, and equivalent in volume to the active arm based on participant's age and weight

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Sterile solution of equal volume to active arm

Interventions

APL2

Complement (C3) Inhibitor

Intervention Type: DRUG

Placebo

Sterile solution of equal volume to active arm

Intervention Type: OTHER

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Apellis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Investigator Site 1

Chicago, Illinois, United States

Investigator Site 2

New York, New York, United States

Countries

United States

Central Contacts

Apellis Clinical Trial Information Line

Role: CONTACT

clinicaltrials@apellis.com

833-284-6361

Other Identifiers

APL2-FSG-319

Identifier Type: -

Identifier Source: org_study_id