Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to healthy male adult volunteers
ADULT;
AREA UNDER THE CURVE;
ARTICLE;
CONCENTRATION AT STEADY-STATE;
CONSTIPATION;
CONTROLLED STUDY;
DIET RESTRICTION;
DOUBLE BLIND PROCEDURE;
DRUG BIOAVAILABILITY;
DRUG BLOOD LEVEL;
DRUG CLEARANCE;
DRUG DOSE ESCALATION;
DRUG HALF LIFE;
DRUG SAFETY;
DRUG TOLERABILITY;
HEADACHE;
HEART PALPITATION;
HUMAN;
HUMAN EXPERIMENT;
LEG CRAMP;
LOOSE FECES;
MALE;
MAXIMUM PLASMA CONCENTRATION;
MORNING DOSAGE;
MULTIPLE CYCLE TREATMENT;
NORMAL HUMAN;
PHASE 1 CLINICAL TRIAL;
PLASMA CONCENTRATION-TIME CURVE;
SINGLE DRUG DOSE;
TIME TO MAXIMUM PLASMA CONCENTRATION;
VOLUME OF DISTRIBUTION;
ADOLESCENT;
ANALOGS AND DERIVATIVES;
BIOSYNTHESIS;
CHEMISTRY;
CLINICAL TRIAL;
DOSE RESPONSE;
DRUG ADMINISTRATION;
HALF LIFE TIME;
MIDDLE AGED;
MUSCULAR DYSTROPHY, DUCHENNE;
NO-OBSERVED-ADVERSE-EFFECT LEVEL;
ORAL DRUG ADMINISTRATION;
YOUNG ADULT;
ADMINISTRATION, ORAL;
ADOLESCENT;
ADULT;
AREA UNDER CURVE;
ARGININE;
BENZOTHIAZOLES;
BENZOXAZOLES;
DOSE-RESPONSE RELATIONSHIP, DRUG;
DOUBLE-BLIND METHOD;
DRUG ADMINISTRATION SCHEDULE;
HALF-LIFE;
HEALTHY VOLUNTEERS;
HUMANS;
MALE;
MIDDLE AGED;
MUSCULAR DYSTROPHY, DUCHENNE;
NO-OBSERVED-ADVERSE-EFFECT LEVEL;
UTROPHIN;
YOUNG ADULT;
Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse
Tinsley JM, Fairclough RJ, Storer R, et al. Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse. PLoS ONE. 2011; 6: e19189.
Orphan drug development in muscular dystrophy: Update on two large clinical trials of dystrophin rescue therapies
Hoffman EP, Connor EM,. Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies. Discov Med. 2013; 16: 233-239.
Prevention of pathology in mdx mice by expression of utrophin: Analysis using an inducible transgenic expression system
Squire S, Raymackers JM, Vandebrouck C, et al. Prevention of pathology in mdx mice by expression of utrophin: analysis using an inducible transgenic expression system. Hum Mol Genet. 2002; 11: 3333-3344.
Expression of utrophin A mRNA correlates with the oxidative capacity of skeletal muscle fiber types and is regulated by calcineurin/NFAT signaling
Chakkalakal JV, Stocksley MA, Harrison MA, et al. Expression of utrophin A mRNA correlates with the oxidative capacity of skeletal muscle fiber types and is regulated by calcineurin/NFAT signaling. Proc Natl Acad Sci USA. 2003; 100: 7791-7796.
Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy
Chancellor DR, Davies KE, De Moor O, et al. Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy. J Med Chem. 2011; 54: 3241-3250.
3rd ed. Stockholm, Sweden: Swedish Pharmaceutical Press
Gabrielsson J, Weiner D,. Pharmacokinetic and Pharmacodynamic Data Analysis: Concepts and Applications. 3rd ed. Stockholm, Sweden: Swedish Pharmaceutical Press; 2000: 141-153.
Pharmacokinetic analysis of mizolastine in healthy young volunteers after single oral and intravenous doses: Noncompartmental approach and compartmental modeling
Mesnil F, Dubruc C, Mentre F, et al. Pharmacokinetic analysis of mizolastine in healthy young volunteers after single oral and intravenous doses: noncompartmental approach and compartmental modeling. J Pharmacokinet Biopharm. 1997; 25: 125-147.
