Placebo: Sterile solution of equal volume to active arm
Study 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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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
Primary outcome measure(s)
Phase 2: Evaluate the efficacy of APL2 in terms of change from baseline in log-transformed urine protein to creatinine ratio (uPCR) — 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) — Baseline to Week 52 Change from baseline in log-transformed uPCR will be based on triplicate first morning urine (FMU)
Trial sites (2)
Facility
City
Region
Status
Investigator Site 1
Chicago
Illinois
Recruiting
Investigator Site 2
New York
New York
Recruiting
More Apellis Pharmaceuticals, Inc. trials in the USA
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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