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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

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 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

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

Do I qualify?Apply below to find out

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Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor

2,432 Previous Clinical Trials

1,090,105 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this investigation seeking out elderly participants?

"This study is recruiting 375 minors and 1000 adults. Specifically, the age range for participants is 5 to 17 years old."

Answered by AI

What potential adverse effects may arise from Part 1 Age adjusted high dose (age 10-14)?

"Although no clinical data is available to support its efficacy, part 1 age-adjusted high dose (age 10-14) was rated a 2 on the safety scale due to preliminary evidence that suggests it can be safely administered."

Answered by AI

What is the upper limit of participants enrolled in this research?

"Affirmative. According to the details on clinicaltrials.gov, this trial was first launched in July 31st 2023 and recently edited on June 23rd 2023 is currently recruiting patients. The study requires 56 participants split between two separate medical centres."

Answered by AI

Are there any opportunities to join this trial currently?

"Indeed, per the data hosted by clinicaltrials.gov this medical trial is currently accepting participants. The study was initially advertised on July 31st 2023 and modified most recently on June 23rd 2023. A total of 56 patients are needed from 2 different sites to complete enrollment."

Answered by AI

What are researchers attempting to determine with this exploration?

"As per the data provided by Hoffmann-La Roche, this study's primary endpoint is an age-based ratio of alogabat clearance (CL/F) that will be monitored for a period up to 12 weeks. Secondary outcomes include: CL/F and Cmax derived from population pharmacokinetic modeling, as well as incidence and severity of adverse events (AEs) and serious adverse events (SAEs)."

Answered by AI

Is it possible to become a participant in this clinical trial?

"This research trial is looking for 56 people aged between 5 and 17 with Angelman syndrome. To be eligible, individuals must have a confirmed genetic deletion of UBE3A, GABRB3, GABRA5 and GABRG3 on the maternally inherited chromosome 15q11q13; BMI within normal limits; evidence that they are able to abstain from sexual activity throughout the study period or use acceptable contraceptive methods if necessary; female participants must not be pregnant nor breastfeeding. Male contraception is unnecessary as the amount of drug exposure through intercourse would likely be minimal."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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.

2023. "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.". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05630066.

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