FDA previously reviewed the AP-003-A (SPRING) study and confirmed it is the first of two required pivotal studies in support of a Biologics License Application (BLA) for Ampion™. FDA has now reviewed the AP-003-B (PIVOT) study and has agreed, “[t]he trial is adequate to serve as [Ampio’s second] pivotal trial in support of a future BLA”. The study will evaluate the efficacy and safety of an intra-articular (IA) injection of Ampion™ in adults with pain due to osteoarthritis (OA) of the knee. This study is a randomized, placebo-controlled, double-blind study with a 28-day screening period for each patient followed by a 12-week participation period. A total of 484 subjects with osteoarthritis knee pain will be randomized 1:1 across 2 study arms: 4mL placebo and 4mL Ampion™ at multiple sites across the country. The primary trial objective is to evaluate the efficacy of 4 mL of Ampion versus 4 mL of placebo IA injection in improving knee pain, when administered to subjects suffering from OA of the knee. The secondary trial objective is evaluation of the safety of an IA injection of Ampion. At the conclusion of the AP-003-B (PIVOT) study, Ampio will have conducted the two required well-controlled and adequate trials in support of a BLA for FDA approval of Ampion.
A Special Protocol Assessment (SPA) is a declaration from the FDA that the planned Phase III trial’s design, clinical endpoints, and statistical analyses are acceptable for support of regulatory approval. It’s the FDA’s way of saying: If you execute this trial exactly as you promise you will, and the data are there, this drug will get approved (Redrearn, 2010). To read more about the FDA’s SPA please click the link below: