This is a two-part, Phase IIa, multicenter, 12-week, open-label study. Up to 56 participants with deletion AS aged 5-17 years (inclusive) will be enrolled in the study.
The study will have Part 1-dose confirmations and Part 2 with dose levels to be decided based on the cumulative PK, electroencephalography (EEG), and safety data emerging from Part 1. The dose levels for the first cohort of Part 2 will be decided based on the cumulative PK, EEG, and safety data emerging from Part 1. Part 2 will explore the change in EEG beta-band power relative to baseline at Week 2, Week 4 (i.e., approximately 2 weeks after the start of the Dose B), and at the end of the 12-week treatment period after daily administration of alogabat.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
48
Alogabat will be administered QD with dose depending on cohort and age of the participant.
Rush Medical Center
Chicago, Illinois, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Part 1: Age-group Based Ratio of Plasma PK Parameter, Area Under the Concentration-time Curve (AUC)
Age-group based ratio of plasma PK parameters in pediatric participants with AS versus data collected from adult healthy volunteers and participants with autism spectrum disorder (ASD) (AUC)
Time frame: Up to 12 Weeks
Part 1: Age-group Based Ratio of Plasma PK Parameter, Apparent Clearance (CL/F)
Age-group based ratio of plasma PK parameters in pediatric participants with AS versus data collected from adult healthy volunteers and participants with ASD (CL/F)
Time frame: Up to 12 Weeks
Part 2: Change From Baseline to Week 2, 4, and 12 in Resting State EEG Power in the Beta Band
Time frame: Week 2, 4, and 12
Parts 1 and 2: Plasma PK Parameter of Alogabat, Maximum Concentration (Cmax)
Plasma PK parameter of alogabat Cmax as derived using a population-pharmacokinetic (popPK) model
Time frame: Up to 12 Weeks
Parts 1 and 2: Plasma PK Parameter of Alogabat, AUC
Plasma PK parameter of alogabat AUC as derived using a popPK model
Time frame: Up to 12 Weeks
Parts 1 and 2: Plasma PK Parameter of Alogabat, CL/F
Plasma PK parameter of alogabat CL/F derived using a popPK model
Time frame: Up to 12 Weeks
Parts 1 and 2: Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Incidence and severity of AEs and SAEs
Time frame: Up to 18 Weeks
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Columbia University Medical Center
New York, New York, United States
Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine
Carrboro, North Carolina, United States
Vanderbilt Children's Hospital
Nashville, Tennessee, United States
Multicare Institute for Research and Innovation
Tacoma, Washington, United States
Queensland Children?s Hospital
South Brisbane, Queensland, Australia
CHRU de Brest
Brest, France
CHU Dijon Bourgogne Hôpital François Mitterand
Dijon, France
Hopital la Timone Enfants
Marseille, France
...and 9 more locations
Parts 1 and 2: Incidence of Treatment Discontinuations due to AEs
Incidence of treatment discontinuations due to AEs
Time frame: Up to 18 Weeks
Parts 1 and 2: Incidence of Daytime Sleepiness Assessed With the Karolinska Sleepiness Scale (KSS), and Incidence of Sudden Onset of Sleep Assessed With Somnolence Diary
Incidence of daytime sleepiness assessed with the KSS, and incidence of sudden onset of sleep assessed with somnolence diary.
Time frame: Up to 12 Weeks