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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. (Aldebaran)

Primary Purpose

Angelman Syndrome

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
60 mg QD Alogabat
40 mg QD Alogabat
7 mg QD Alogabat
Part 2 Adult Alogabat High Dose (aged 15-17)
Alogabat
Alogabat
Part 2 Optional Cohort
Sponsored by
Hoffmann-La Roche
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Angelman Syndrome

Eligibility Criteria

5 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Clinical diagnosis of AS and a genetic subtype of deletion on the maternally inherited chromosome 15q11q13 confirmed by a historical molecular diagnosis. The deletion must include UBE3A, GABRB3, GABRA5, and GABRG3 genes, and be less than 7 Mb in size. Body mass index (BMI) below the 97th percentile and above the 3rd percentile for the same age and sex 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 Exclusion Criteria: 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 within 12 months of screening or uncompensated heart failure Confirmed clinically significant abnormality on 12-lead ECG, including: a QTcF of >/= 450 ms (based on the average of 3 consecutive measurements) for participants older than 10 years old a 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 QTc prolongation or family history of Long QT Syndrome 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 RNA may be considered eligible for entry into the study.

Sites / Locations

  • Rush Medical CenterRecruiting
  • Columbia University Medical CenterRecruiting
  • Carolina Institute for Development Disabilities University of North Carolina/School of MedicineRecruiting
  • Flinders Medical CentreRecruiting
  • Hopital la Timone Enfants; Service de Pediatrie et Neurologie PediatriqueRecruiting
  • Groupe Hospitalier Necker Enfants MaladesRecruiting
  • Ospedale Pediatrico Bambino Gesù; Dip. Neuroscienze e NeuroriabilitazioneRecruiting
  • IRCCS Istituto G. Gaslini; UOC Neurologia Pediatrica e Malattie MuscolariRecruiting
  • Corporacio Sanitaria Parc TauliRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm 7

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

Part 1 Adult Alogabat High Dose (aged 15-17)

Part 1 Age adjusted high dose (age 10-14)

Part 1 Age Adjusted Low Dose (age 5-9)

Part 2 Cohort 1

Part 2 Cohort 2

Part 1 Optional Cohort

Part 2 Optional Cohort

Arm Description

In Part 1 of the study, participants will receive age-adjusted dose 60 mg QD alogabat

In Part 1 of the study, participants will receive age-adjusted dose 60 mg QD alogabat.

In Part 1 of the study, participants will receive age-adjusted dose 20 mg QD alogabat.

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.

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.

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.

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.

Outcomes

Primary Outcome Measures

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])
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])
Change from baseline to Week 2, 4, and 12 in resting state EEG power in the beta band
In Part 2 only

Secondary Outcome Measures

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
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
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
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)
Incidence of treatment discontinuations due to AEs
Incidence of treatment discontinuations due to AEs in Part 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 Karolinska Sleepiness Scale (KSS), and incidence of sudden onset of sleep assessed with somnolence diary in Part 1 and 2.

Full Information

First Posted
November 9, 2022
Last Updated
October 11, 2023
Sponsor
Hoffmann-La Roche
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1. Study Identification

Unique Protocol Identification Number
NCT05630066
Brief Title
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.
Acronym
Aldebaran
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 27, 2023 (Actual)
Primary Completion Date
March 21, 2025 (Anticipated)
Study Completion Date
March 21, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hoffmann-La Roche

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
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.
Detailed Description
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Angelman Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
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, and the other from week 2 to week 12. 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. A total of two optional cohorts may be utilized in this study, allocated to Part 1 and/or Part 2.
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
56 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Part 1 Adult Alogabat High Dose (aged 15-17)
Arm Type
Experimental
Arm Description
In Part 1 of the study, participants will receive age-adjusted dose 60 mg QD alogabat
Arm Title
Part 1 Age adjusted high dose (age 10-14)
Arm Type
Experimental
Arm Description
In Part 1 of the study, participants will receive age-adjusted dose 60 mg QD alogabat.
Arm Title
Part 1 Age Adjusted Low Dose (age 5-9)
Arm Type
Experimental
Arm Description
In Part 1 of the study, participants will receive age-adjusted dose 20 mg QD alogabat.
Arm Title
Part 2 Cohort 1
Arm Type
Experimental
Arm Description
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.
Arm Title
Part 2 Cohort 2
Arm Type
Experimental
Arm Description
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.
Arm Title
Part 1 Optional Cohort
Arm Type
Experimental
Arm Description
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.
Arm Title
Part 2 Optional Cohort
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
60 mg QD Alogabat
Intervention Description
Participants aged 15-17 years or above receiving the adult 60 mg of alogabat dose. I
Intervention Type
Drug
Intervention Name(s)
40 mg QD Alogabat
Intervention Description
Participants aged 10-14 years receiving the equivalent of the adult 60 mg alogabat dose.
Intervention Type
Drug
Intervention Name(s)
7 mg QD Alogabat
Intervention Description
Participants aged 5-9 years receiving the equivalent of the adult 20 mg alogabat dose.
Intervention Type
Drug
Intervention Name(s)
Part 2 Adult Alogabat High Dose (aged 15-17)
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Alogabat
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Alogabat
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Part 2 Optional Cohort
Intervention Description
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
Primary Outcome Measure Information:
Title
Age-group based ratio of plasma PK parameter, area under the concentration-time curve (AUC)
Description
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])
Time Frame
Up to 12 Weeks
Title
Age-group based ratio of plasma PK parameter, apparent clearance (CL/F)
Description
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])
Time Frame
Up to 12 Weeks
Title
Change from baseline to Week 2, 4, and 12 in resting state EEG power in the beta band
Description
In Part 2 only
Time Frame
Week 2, 4, and 12
Secondary Outcome Measure Information:
Title
Plasma pharmacokinetic parameter of alogabat, maximum concentration (Cmax)
Description
In Part 1 and 2. Plasma pharmacokinetic parameter of alogabat maximum concentration (Cmax) as derived using a population-pharmacokinetic (popPK) model
Time Frame
Up to 12 Weeks
Title
Plasma pharmacokinetic parameters of alogabat area under the concentration-time curve (AUC)
Description
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
Time Frame
Up to 12 Weeks
Title
Plasma pharmacokinetic parameter of alogabat, apparent clearance (CL/F)
Description
In Part 1 and 2. Plasma pharmacokinetic parameter of alogabat apparent clearance (CL/F) derived using a population-pharmacokinetic (popPK) model
Time Frame
Up to 12 Weeks
Title
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs).
Description
In Part 1 and 2. Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
Time Frame
Up to 18 Weeks
Title
Incidence of treatment discontinuations due to AEs
Description
Incidence of treatment discontinuations due to AEs in Part 1 and 2
Time Frame
Up to 18 Weeks
Title
Incidence of daytime sleepiness assessed with the Karolinska Sleepiness Scale (KSS), and incidence of sudden onset of sleep assessed with somnolence diary
Description
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.
Time Frame
Up to 21 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Clinical diagnosis of AS and a genetic subtype of deletion on the maternally inherited chromosome 15q11q13 confirmed by a historical molecular diagnosis. The deletion must include UBE3A, GABRB3, GABRA5, and GABRG3 genes, and be less than 7 Mb in size. Body mass index (BMI) below the 97th percentile and above the 3rd percentile for the same age and sex 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 Exclusion Criteria: 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 within 12 months of screening or uncompensated heart failure Confirmed clinically significant abnormality on 12-lead ECG, including: a QTcF of >/= 450 ms (based on the average of 3 consecutive measurements) for participants older than 10 years old a 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 QTc prolongation or family history of Long QT Syndrome 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 RNA may be considered eligible for entry into the study.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Reference Study ID Number: BP41315 https://forpatients.roche.com/
Phone
888-662-6728 (U.S. Only)
Email
global-roche-genentech-trials@gene.com
Facility Information:
Facility Name
Rush Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Individual Site Status
Recruiting
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Individual Site Status
Recruiting
Facility Name
Carolina Institute for Development Disabilities University of North Carolina/School of Medicine
City
Carrboro
State/Province
North Carolina
ZIP/Postal Code
27510
Country
United States
Individual Site Status
Recruiting
Facility Name
Flinders Medical Centre
City
Bedford Park
State/Province
South Australia
ZIP/Postal Code
5042
Country
Australia
Individual Site Status
Recruiting
Facility Name
Hopital la Timone Enfants; Service de Pediatrie et Neurologie Pediatrique
City
Marseille
ZIP/Postal Code
13005
Country
France
Individual Site Status
Recruiting
Facility Name
Groupe Hospitalier Necker Enfants Malades
City
Paris
ZIP/Postal Code
75015
Country
France
Individual Site Status
Recruiting
Facility Name
Ospedale Pediatrico Bambino Gesù; Dip. Neuroscienze e Neuroriabilitazione
City
Roma
State/Province
Lazio
ZIP/Postal Code
00165
Country
Italy
Individual Site Status
Recruiting
Facility Name
IRCCS Istituto G. Gaslini; UOC Neurologia Pediatrica e Malattie Muscolari
City
Genova
State/Province
Liguria
ZIP/Postal Code
16147
Country
Italy
Individual Site Status
Recruiting
Facility Name
Corporacio Sanitaria Parc Tauli
City
Sabadell
State/Province
Barcelona
ZIP/Postal Code
08208
Country
Spain
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

Learn more about this trial

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.

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