What is the mechanism of action of this treatment?

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.

What side effects are associated with this type of intervention?

Common treatments for Angelman Syndrome include melatonin and clonidine for sleep disturbances, and clonazepam for mild seizures. Melatonin is generally well-tolerated but can cause daytime drowsiness and headaches. Clonidine may lead to side effects such as dry mouth, drowsiness, and low blood pressure. Clonazepam can cause drowsiness, dizziness, and coordination problems. These treatments aim to manage symptoms rather than address the genetic cause of AS.

What prior FDA approvals exist?

There are no specific FDA-approved drugs mentioned in the provided context for the treatment of Angelman Syndrome. The context does reference a study investigating the pharmacokinetics and safety of Alogabat in children and adolescents with Angelman Syndrome, which suggests ongoing research into potential treatments targeting the symptoms or genetic mechanisms of the condition.

What other types of research exist?

One promising novel alternative for treating Angelman Syndrome is Alogabat, which is currently being investigated in a Phase IIa clinical trial. This study aims to evaluate the pharmacokinetics, safety, and proof of mechanism of Alogabat in children and adolescents with the deletion genotype of Angelman Syndrome. This drug represents a targeted approach to managing the condition by potentially addressing the underlying genetic mechanisms.

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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

No Placebo-Only Group

Summary

This trial tests Alogabat, a medication, to see if it can help children aged 5-17 with deletion Angelman Syndrome. Researchers will give the medication daily and check for improvements in brain activity over a few months.

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.

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.

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 2, 4, and 12

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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 study objectives

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

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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.

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