← Back to Search

GABA Receptor Agonist

Alogabat for Angelman Syndrome (Aldebaran Trial)

Phase 2
Recruiting
Research Sponsored by Hoffmann-La Roche
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 2, 4, and 12
Awards & highlights

Aldebaran Trial Summary

This trial will test a new therapy for children 5-17 with Angelman Syndrome (AS) over a 12-week period. Up to 56 participants will be enrolled.

Who is the study for?
This trial is for children and adolescents aged 5-17 with Angelman Syndrome (AS) who have a specific genetic deletion. They must have a BMI within the normal range for their age and sex, not be pregnant or breastfeeding, and either practice abstinence or use contraception. Participants should not have heart issues like QTc prolongation, recent major infections or surgeries, other conditions that could affect the study's results, or a history of certain diseases.Check my eligibility
What is being tested?
The trial is testing different doses of Alogabat in two parts over 12 weeks to understand how it works in kids with AS. It's an open-label study where everyone knows what treatment they're getting. The goal is to find out how safe Alogabat is and how the body processes it.See study design
What are the potential side effects?
While specific side effects are not listed here, common concerns may include reactions at the site of administration, potential impacts on growth due to hormone changes from medication intake at such young ages, general discomforts like headaches or stomachaches typical with new medications.

Aldebaran Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 2, 4, and 12
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 2, 4, and 12 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Age-group based ratio of plasma PK parameter, apparent clearance (CL/F)
Age-group based ratio of plasma PK parameter, area under the concentration-time curve (AUC)
Change from baseline to Week 2, 4, and 12 in resting state EEG power in the beta band
Secondary outcome measures
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs).
Incidence of daytime sleepiness assessed with the Karolinska Sleepiness Scale (KSS), and incidence of sudden onset of sleep assessed with somnolence diary
Incidence of treatment discontinuations due to AEs
+3 more

Aldebaran Trial Design

7Treatment groups
Experimental Treatment
Group I: Part 2 Optional CohortExperimental Treatment1 Intervention
If dose adjustments (e.g., increase or decrease in dose) are required, particularly due to uncertainty of the clearance estimates (e.g., due to high variability) or over-/underprediction of the pediatric clearance versus adult clearance, participants from any of the 3 age-groups may enroll in order to confirm the exposure equivalence. A total of two optional cohorts may be utilized in this study, allocated to Part 1 and/or Part 2.
Group II: Part 2 Cohort 2Experimental Treatment1 Intervention
In Part 2 of the study, the dosing will depend upon the results of Part 1, with age-adjusted dose equivalents of up to 100 mg being administered.
Group III: Part 2 Cohort 1Experimental Treatment1 Intervention
In Part 2 of the study, the dosing will depend upon the results of Part 1, with age-adjusted dose equivalents of up to 100 mg being administered.
Group IV: Part 1 Optional CohortExperimental Treatment1 Intervention
If dose adjustments (e.g., increase or decrease in dose) are required, particularly due to uncertainty of the clearance estimates (e.g., due to high variability) or over-/underprediction of the pediatric clearance versus adult clearance, additional participants may be recruited in any of the of the 3 age-groups in order to confirm the exposure equivalence. A total of two optional cohorts may be utilized in this study, allocated to Part 1 and/or Part 2.
Group V: Part 1 Age adjusted high dose (age 10-14)Experimental Treatment1 Intervention
In Part 1 of the study, participants will receive age-adjusted dose 60 mg QD alogabat.
Group VI: Part 1 Age Adjusted Low Dose (age 5-9)Experimental Treatment1 Intervention
In Part 1 of the study, participants will receive age-adjusted dose 20 mg QD alogabat.
Group VII: Part 1 Adult Alogabat High Dose (aged 15-17)Experimental Treatment1 Intervention
In Part 1 of the study, participants will receive age-adjusted dose 60 mg QD alogabat

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Angelman Syndrome (AS) focus on managing symptoms and targeting the genetic mechanisms underlying the disorder. Pharmacological agents like Alogabat, currently under study, aim to modulate neurotransmitter systems to improve neurological function. These treatments are crucial for AS patients as they can potentially enhance cognitive and motor functions, reduce seizures, and improve overall quality of life. By addressing the genetic and symptomatic aspects of AS, these therapies offer hope for more effective management of this complex neurogenetic disorder.
Immunophenotypes and karyotypes of leukemic cells in children with Down syndrome and acute lymphoblastic leukemia.Therapeutic Approaches in Adult Primary Spinal Cord Astrocytoma: A Systematic Review.The Limb-Girdle Muscular Dystrophies: Is Treatment on the Horizon?

Find a Location

Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor
2,434 Previous Clinical Trials
1,091,336 Total Patients Enrolled
3 Trials studying Angelman Syndrome
135 Patients Enrolled for Angelman Syndrome

Media Library

Alogabat (GABA Receptor Agonist) Clinical Trial Eligibility Overview. Trial Name: NCT05630066 — Phase 2
~27 spots leftby Mar 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top