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Study to Investigate the Pharmacokinetics and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.
This is a two-part, Phase IIa, multicenter, 12-week, open-label study. Up to 56 participants with deletion Angelman Syndrome (AS) aged 5-17 years (inclusive) will be enrolled in the study.
Study details:
The study will have Part 1-dose confirmations and Part 2 with dose levels to be decided based on the cumulative PK, 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 second dose), and at the end of the 12-week treatment period after daily administration of Alogabat.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 5 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2023-07-27
Primary completion: 2025-09-12
Study completion finish: 2025-09-12
Study type
TREATMENT
Phase
PHASE2
Trial ID
NCT05630066
Intervention or treatment
DRUG: 60 mg QD Alogabat
DRUG: 40 mg QD Alogabat
DRUG: 7 mg QD Alogabat
DRUG: Part 2 Adult Alogabat High Dose (aged 15-17)
DRUG: Alogabat
DRUG: Alogabat
DRUG: Part 2 Optional Cohort
Conditions
- • Angelman Syndrome
Find a site
Closest Location:
Queensland Children?s Hospital
Research sites nearby
Select from list below to view details:
Queensland Children?s Hospital
South Brisbane, Queensland, Australia
Flinders Medical Centre
Bedford Park, South Australia, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: Part 1 Adult Alogabat High Dose (aged 15-17)
| DRUG: 60 mg QD Alogabat
|
EXPERIMENTAL: Part 1 Age adjusted high dose (age 10-14)
| DRUG: 40 mg QD Alogabat
|
EXPERIMENTAL: Part 1 Age Adjusted Low Dose (age 5-9)
| DRUG: 7 mg QD Alogabat
|
EXPERIMENTAL: Part 2 Cohort 1
| DRUG: Part 2 Adult Alogabat High Dose (aged 15-17)
|
EXPERIMENTAL: Part 2 Cohort 2
| DRUG: Alogabat
|
EXPERIMENTAL: Part 1 Optional Cohort
| DRUG: Alogabat
|
EXPERIMENTAL: Part 2 Optional Cohort
| DRUG: Part 2 Optional Cohort
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
Age-group based ratio of plasma PK parameter, area under the concentration-time curve (AUC) | In Part 1 only. Age-group based ratio of plasma PK parameters in pediatric participants with AS versus data collected from adult healthy volunteers and participants with ASD (area under the concentration-time curve \[AUC\]) | Up to 12 Weeks |
Age-group based ratio of plasma PK parameter, apparent clearance (CL/F) | In Part 1 only. Age-group based ratio of plasma PK parameters in pediatric participants with AS versus data collected from adult healthy volunteers and participants with ASD (apparent clearance \[CL/F\]) | Up to 12 Weeks |
Change from baseline to Week 2, 4, and 12 in resting state EEG power in the beta band | In Part 2 only | Week 2, 4, and 12 |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
Plasma pharmacokinetic parameter of alogabat, maximum concentration (Cmax) | In Part 1 and 2. Plasma pharmacokinetic parameter of alogabat maximum concentration (Cmax) as derived using a population-pharmacokinetic (popPK) model | Up to 12 Weeks |
Plasma pharmacokinetic parameters of alogabat area under the concentration-time curve (AUC) | In Part 1 and 2. Plasma pharmacokinetic parameter of alogabat area under the concentration-time curve (AUC) as derived using a population-pharmacokinetic (popPK) model | Up to 12 Weeks |
Plasma pharmacokinetic parameter of alogabat, apparent clearance (CL/F) | In Part 1 and 2. Plasma pharmacokinetic parameter of alogabat apparent clearance (CL/F) derived using a population-pharmacokinetic (popPK) model | Up to 12 Weeks |
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs). | In Part 1 and 2. Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) | Up to 18 Weeks |
Incidence of treatment discontinuations due to AEs | Incidence of treatment discontinuations due to AEs in Part 1 and 2 | Up to 18 Weeks |
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 Karolinska Sleepiness Scale (KSS), and incidence of sudden onset of sleep assessed with somnolence diary in Part 1 and 2. | Up to 21 Weeks |
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