SEC Filings

8-K
AVEXIS, INC. filed this Form 8-K on 12/13/2017
Entire Document
 

Phase 1 Trial of AVXS-101 in SMA Type 2 6 Route of Administration One-time intrathecal dose Prednisolone 1 mg/kg 1 day Pre-GT Trial Design 27-patient, Dose-comparison AVXS-101 PHASE 1 TRIAL OVERVIEW – SMA TYPE 2 Study Sites 11 centers in U.S. Safety Safety and Tolerability of intrathecal administration of AVXS-101 Determination of optimal dose SAFETY OBJECTIVE Primary Ability to stand alone for at least 3 seconds Secondary Ability to walk without assistance, defined as taking at least 5 steps independently displaying coordination and balance Exploratory Hammersmith Functional Motor Scale – Expanded Change from baseline in fine and gross motor components EFFICACY OBJECTIVES: PATIENTS LESS THAN 24 MONTHS* Primary Change from baseline in Hammersmith Functional Motor Scale – Expanded Secondary Ability to walk without assistance, defined as taking at least 5 steps independently displaying coordination and balance Exploratory Change from baseline in fine and gross motor components EFFICACY OBJECTIVES: PATIENTS AT LEAST 24 MONTHS* *Additionally, compelling, demonstrable, documented evidence of efficacy as determined by changes in developmental abilities as captured during videotaping sessions during site visits and/or captured/provided by parent/legal guardian will be collected for all patients in all age groups.

GRAPHIC

 


© AveXis, Inc. All Rights Reserved.