Methods for increasing growth in pediatric subjects having cholestatic liver disease

Abstract

Provided herein are methods for increasing growth in a pediatric subject having a cholestatic liver disease. The method includes administering to the subject an effective amount of an ASBTI.

1 . A method for increasing growth in a pediatric subject having a cholestatic liver disease, the method comprising administering to the subject an effective amount of an ASBTI, wherein the ASBTI is selected from the group consisting of 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof. 
     
     
         2 . (canceled) 
     
     
         3 . The method of  claim 1 , wherein the ASBTI is 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable alternative salt thereof. 
     
     
         4 . The method of  claim 1 , wherein the ASBTI is volixibat, or a pharmaceutically acceptable salt thereof. 
     
     
         5 .- 7 . (canceled) 
     
     
         8 . The method of  claim 1 , wherein the cholestatic liver disease is progressive familial intrahepatic cholestasis (PFIC), biliary atresia, Alagille syndrome (ALGS), or any pediatric cholestatic condition resulting in below normal growth, height, or weight. 
     
     
         9 . The method of  claim 1 , wherein the cholestatic liver disease is biliary atresia. 
     
     
         10 . The method of  claim 1 , wherein the cholestatic liver disease is PFIC. 
     
     
         11 . The method of  claim 10 , wherein the PFIC is selected from PFIC type 1, PFIC type 2, and PFIC type 3. 
     
     
         12 . The method of  claim 11 , wherein the PFIC is PFIC type 2. 
     
     
         13 . The method of  claim 12 , wherein the subject has an ABCB11 gene with a missense mutation and no truncating mutation. 
     
     
         14 . The method of  claim 1 , wherein the cholestatic liver disease is ALGS. 
     
     
         15 . (canceled) 
     
     
         16 . The method of  claim 1 , wherein the ASBTI is administered once daily (QD). 
     
     
         17 . The method of  claim 16 , wherein the ASBTI is administered in an amount of about 140 μg/kg/day to about 1400 μg/kg/day. 
     
     
         18 . The method of  claim 1 , wherein the ASBTI is administered twice daily (BID). 
     
     
         19 . The method of  claim 18 , wherein the ASBTI is administered in an amount of about 70 μg/kg to about 700 μg/kg per dose. 
     
     
         20 . The method of  claim 1 , wherein an increase in growth is measured as an increase in height or weight Z-score. 
     
     
         21 . The method of  claim 20 , wherein the administration results in an increase in height or weight Z-score of at least 0.1 relative to baseline. 
     
     
         22 . The method of  claim 21 , wherein the administration results in an increase in height or weight Z-score of at least 0.25 relative to baseline. 
     
     
         23 . The method of  claim 1 , wherein the increase in growth is observed within 1 year of first administration of the ASBTI. 
     
     
         24 . The method of  claim 1 , wherein an increase in height or weight Z-score is maintained over a period of up to 20 weeks. 
     
     
         25 . The method of  claim 24 , wherein the increase in height or weight Z-score is maintained over a period of up to 2 years. 
     
     
         26 .- 37 . (canceled)