A Stabilized Release Neuropeptide Exhibits Efficacy in a Phase II Double Blind Placebo Controlled Clinical Trial of Secondary Progressive Multiple Sclerosis (P3.167)

BACKGROUND A phase II trial to explore the efficacy, safety and tolerability of a stabilised neuropeptide - SR-CRH-01 in Secondary Progressive MS (SPMS) DESIGN AND METHODS A phase II double-blind placebo controlled (DBPC) crossover clinical trial was performed with n=20 SPMS patients who received SR-CRH-01 [known as AIMSPRO®] (n=10), or placebo (n=10) by s.c. injection with 1 mL (4.5 mg/mL) twice weekly for 4-weeks, followed by a 6-week washout, and then a crossover to the other treatment for 4-weeks. Post-DBPC phase, SR-CRH-01 was given in an open-label phase (OL) for an additional 38 weeks. RESULTS Primary endpoints; in the DBPC phase SR-CRH-01 did not show significantly altered bladder function. The therapy was safe and well tolerated throughout the trial period. In the OL phase, SR-CRH-01 significantly decreased the mean voiding frequency (p<0.0001) and mean urinary incontinence (p=0.0009). Secondary endpoints; in the DBPC phase, SR-CRH-01 significantly increased the total MS Functional Composite (MSFC) versus placebo (p=0.0215); the 25-foot walk (p=0.049) and mean 9-hole peg test [9-HPT] (p=0.046). Analysis of the mean PASAT 3 in the OL phase showed that SR-CRH-01 also significantly increased the mean score from baseline to 26 weeks (p<0.0001). SR-CRH-01 significantly decreased the mean MS Impact (physical) scale in the DBPC phase (p=0.007), which continued through to week 26 (p<0.0001). The visual acuity (LogMar - both eyes combined) by treatment in the DBPC phase was significantly improved by SR-CRH-01 versus placebo (p=0.026). This effect persisted to the OL phase (p=0.005). The mean colour vision score also showed a strong trend towards improvement in the DBPC phase (p=0.06). SR-CRH-01 significantly increased the overall I-QOL during the OL phase (p=0.007). CONCLUSION The medication SR-CRH-01 was well tolerated. Although bladder function in the short DBPC phase was not significantly altered, the trial results implied that longer term administration had a therapeutic effect on bladder instability. The multiple significant secondary outcome results support future development of larger, longer-term studies.