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Angelman SyndromePhase 2AldebaranNCT05630066

A Study to Investigate the Pharmacokinetics (PK) 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.

A Phase 2 study testing Alogabat in people with Angelman Syndrome, run by Hoffmann-La Roche at 19 sites in 6 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 5 to 17 years
Conditions the study is for
Angelman Syndrome
What is being tested?
Alogabat
Could I receive a placebo?
No, everyone receives an active treatment
How many people
48 participants
Where
19 sitesin 6 countries
Study dates
27 Jul 2023to 2 Dec 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Clinical diagnosis of AS and a genetic subtype of deletion on chromosome 15q11q13 confirmed by a historical molecular diagnosis
  • The participant's general health status, in the context of the disease under study, allows them to participate in a clinical trial in the opinion of the investigator
  • The reliability of sexual abstinence for male and/or female enrollment eligibility needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or post-ovulation methods) and withdrawal are not acceptable methods of preventing drug exposure
  • Female participants:
  • A female participant is eligible to participate if she is not pregnant, not breastfeeding, and non-childbearing or remain abstinent and/or Hormonal contraceptive methods must be supplemented
  • -Male participants: Male contraception is not required in this study because of the minimal seminal dose transmitted through sexual intercourse

Who cannot join

  • A molecular diagnosis of AS with genotypic classification of any type besides the molecular diagnosis as specified in Inclusion Criterion
  • Concurrent cardiovascular disease considered not well controlled by drug treatment, including participants with clinically significant hypertension, bradycardia and arrhythmias, myocardial infarction (MI) within 12 months of screening or uncompensated heart failure
  • Confirmed clinically significant abnormality on 12-lead electrocardiogram (ECG), including:
  • a QT corrected for heart rate using the Fridericia's correction factor (QTcF) of >/= 450 ms (based on the average of 3 consecutive measurements) for participants older than 10 years old
  • a QT corrected for heart rate using Bazett's formula (QTcB) of >/= 450 ms (based on the average of 3 consecutive measurements) for participants up to, and including, the age of 10 years old
  • Congenital heart diseases not treated and congenital QT corrected for heart rate (QTc) prolongation or family history of Long QT Syndrome
Show all 20Show fewer
  • Medical history of malignancy if not considered cured or if occurred within the last 5 years with the exception of fully excised non-melanoma skin cancers or in-situ carcinoma of the cervix that has been successfully treated
  • Concomitant disease, condition, or treatment that would either interfere with the conduct of the study or pose an unacceptable risk to the participant in the opinion of the investigator
  • Known active or uncontrolled bacterial, viral, or other infection (excluding fungal infections of nail beds) or any major episode of infection or hospitalization (relating to the completion of the course of antibiotics) within 6 weeks prior to the start of drug administration. Rescreening is allowed once the infection is cured and if the rescreening criteria are met
  • Any concomitant condition that might interfere with the clinical evaluation of AS and that is not related to AS
  • Known history of human immunodeficiency virus (HIV) or hepatitis B virus (HBV) or hepatitis C virus (HCV)
  • Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study. Rescreening is allowed not earlier than 12 weeks after the surgery and if the rescreening criteria are met.
  • Use of prohibited medications within 6 weeks or 5 half-lives (t1/2) prior to start of study medication on Day 1 (whichever is longer)
  • Clinically significant loss of blood within 3 months prior to screening defined by participant age and weight per recommendations from Duke University (2012)
  • Any prior or current treatment with an investigational study drug within 6 weeks or 5 times the t1/2 of the investigational molecule (whichever is longer) prior to baseline or prior or current use of an investigational medical device within 6 weeks prior to baseline or if the device is still active. Concurrent or planned concurrent participation in any clinical study (including observational and non-interventional studies) without approval of the Investigator.
  • Previous participation in a cellular therapy, gene therapy, or gene editing clinical study
  • Clinically significant vital sign or ECG abnormalities at Screening
  • Confirmed clinically significant abnormality in hematological, chemistry or coagulation laboratory parameters
  • Uncorrected hypokalemia or hypomagnesaemia
  • Positive test result at screening for hepatitis B surface antigen (HBsAg), HCV (untreated), or HIV-1/2. Participants with HCV who have been successfully treated and who test negative for HCV ribonucleic acid (HCV RNA) may be considered eligible for entry into the study

Trial locations and contacts

19 sites in 6 countries.

19 sites in 6 countries

United States6 sites

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

  • Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine

    Carrboro, North Carolina, 27510

  • Rush Medical Center

    Chicago, Illinois, 60612

  • Vanderbilt Children's Hospital

    Nashville, Tennessee, 37232-9119

  • Columbia University Medical Center

    New York, New York, 10032

  • Multicare Institute for Research and Innovation

    Tacoma, Washington, 98405

Other trials in United States

France4 sites

  • CHRU de Brest

    Brest, 29609

  • CHU Dijon Bourgogne Hôpital François Mitterand

    Dijon, 21000

  • Hopital la Timone Enfants

    Marseille, 13005

  • Groupe Hospitalier Necker Enfants Malades

    Paris, 75015

Other trials in France

Spain4 sites

  • Hospital Sant Joan de Deu

    Esplugues de Llobregat · Barcelona, Barcelona, 08950

  • Hospital Universitario Puerta De Hierro Majadahonda

    Madrid, 28222

  • Hospital Universitario de Navarra;Unidad de Neuropediatría

    Pamploa, Navarre, 31008

  • Corporacio Sanitaria Parc Tauli

    Sabadell, Barcelona, 08208

Other trials in Spain

Italy3 sites

  • IRCCS Eugenio Medea

    Conegliano Veneto (TV), Veneto, 31015

  • IRCCS Istituto G. Gaslini

    Genoa, Liguria, 16147

  • Ospedale Pediatrico Bambino Gesù

    Rome, Lazio, 00165

Other trials in Italy

Australia1 site

  • Queensland Children?s Hospital

    South Brisbane, Queensland, 4101

Other trials in Australia

Germany1 site

  • Dr. Von Haunersches Kinderspital

    München, 80337

Other trials in Germany

About this study

Official title: A Phase IIa Multicenter, Open-label, 12-week 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 AS aged 5-17 years (inclusive) will be enrolled in the study.

Read the detailed description

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.

Being tested
Alogabat
Medical subject headings
Angelman Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AlogabatDrug

    Alogabat will be administered QD with dose depending on cohort and age of the participant.

Study groups in NCT05630066
Study groupRoleWhat they receive
Part 1 Adult Alogabat Dose (Age 15-17)ExperimentalIn Part 1 of the study participants will receive alogabat once a day (QD).
Receives: Drug: Alogabat
Part 1 Age-adjusted Dose (Age 10-14)ExperimentalIn Part 1 of the study, participants will receive age-adjusted QD doses of alogabat.
Receives: Drug: Alogabat
Part 1 Age-adjusted Dose (Age 5-9)ExperimentalIn Part 1 of the study, participants will receive age-adjusted QD doses of alogabat.
Receives: Drug: Alogabat
Part 2 Cohort 1ExperimentalIn Part 2 of the study, the dosing will depend upon the results of Part 1 with two different dose levels per cohort. Doses can be age-adjusted.
Receives: Drug: Alogabat
Part 2 Cohort 2ExperimentalIn Part 2 of the study, the dosing will depend upon the interim results with two different dose levels per cohort. Doses can be age-adjusted.
Receives: Drug: Alogabat
Part 1 Optional CohortExperimentalIf 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.
Receives: Drug: Alogabat
Part 2 Optional CohortExperimentalIn Part 2 of the study, the dosing will depend upon the interim results with two different dose levels per cohort. Doses can be age-adjusted.
Receives: Drug: Alogabat

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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)

    Measured: Up to 12 Weeks

  2. 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)

    Measured: Up to 12 Weeks

  3. Part 2: Change From Baseline to Week 2, 4, and 12 in Resting State EEG Power in the Beta Band

    Measured: Week 2, 4, and 12

Secondary outcomes (6)

  1. 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

    Measured: Up to 12 Weeks

  2. Parts 1 and 2: Plasma PK Parameter of Alogabat, AUC

    Plasma PK parameter of alogabat AUC as derived using a popPK model

    Measured: Up to 12 Weeks

  3. Parts 1 and 2: Plasma PK Parameter of Alogabat, CL/F

    Plasma PK parameter of alogabat CL/F derived using a popPK model

    Measured: Up to 12 Weeks

  4. Parts 1 and 2: Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Incidence and severity of AEs and SAEs

    Measured: Up to 18 Weeks

  5. Parts 1 and 2: Incidence of Treatment Discontinuations due to AEs

    Incidence of treatment discontinuations due to AEs

    Measured: Up to 18 Weeks

  6. 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.

    Measured: Up to 12 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Nov 2022
  2. Study start27 Jul 2023
  3. Primary completion2 Dec 2025
  4. Study completion2 Dec 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Model description
There will be up to 7 cohorts. The adolescents aged 15-17 will receive the adult dose. Dosing in Part 1 is pre-specified. Cohorts in Part 1 enroll a specific age range (15-17, 10-14, 5-9), with older cohorts being initiated prior to younger ones. Part 2 features mixed-age cohorts, with age-adjusted doses. Within each Part 2 cohort, two different doses of alogabat may be used: one up to Week 2 (Dose A), and the other from Week 3 to Week 12 (Dose B). Part 2 opens enrolment for ages 10-17 years following analysis of the 2-week data from the Part 1 cohorts aged 15-17 years and 10-14 years. Ages 5-9 years may enroll in Part 2 following analysis of the 2-week data from the Part 1 cohort with participants aged 5-9 years. Part 2 Cohort 2 will open enrollment across ages following the review of Week 2 and Week 4 data in Part 2 Cohort 1. A total of two optional cohorts may be utilized in this study, allocated to Part 1 and/or Part 2.
Masking
None (open label)
Enrolment
48 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05630066
Sponsor's protocol ID
BP41315
Other IDs
2022-501844-14-00
Acronym
Aldebaran
Status last verified
Dec 2025
First submitted
9 Nov 2022
Last updated
4 Dec 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Alogabat in people with Angelman Syndrome, run by Hoffmann-La Roche at 19 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in United States, France, Spain, Italy, Australia and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part 1: Age-group Based Ratio of Plasma PK Parameter, Area Under the Concentration-time Curve (AUC) (time frame: Up to 12 Weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05630066.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2025.

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