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

Apellis Pharmaceuticals, Inc.

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

Age range: 18+ years
Sex: All
Healthy volunteers: No

Detailed 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 s…

Interventions

Drug
APL2
Complement (C3) Inhibitor
Other
Placebo
Sterile solution of equal volume to active arm

Locations (2)

Investigator Site 1
Chicago, Illinois
Investigator Site 2
New York, New York

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

Summary

Eligibility

Interventions

Locations (2)

Compensation

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Details

Conditions