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
Status: Completed
Location: See all (19) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Maximum Age: 17
Healthy Volunteers: f
View:
⁃ 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
Locations
United States
Illinois
Rush Medical Center
Chicago
Massachusetts
Boston Children's Hospital
Boston
North Carolina
Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine
Carrboro
New York
Columbia University Medical Center
New York
Tennessee
Vanderbilt Children's Hospital
Nashville
Washington
Multicare Institute for Research and Innovation
Tacoma
Other Locations
Australia
Queensland Children?s Hospital
South Brisbane
France
CHRU de Brest
Brest
CHU Dijon Bourgogne Hôpital François Mitterand
Dijon
Hopital la Timone Enfants
Marseille
Groupe Hospitalier Necker Enfants Malades
Paris
Germany
Dr. Von Haunersches Kinderspital
München
Italy
IRCCS Eugenio Medea
Conegliano Veneto (tv)
IRCCS Istituto G. Gaslini
Genoa
Ospedale Pediatrico Bambino Gesù
Rome
Spain
Hospital Sant Joan de Deu
Esplugues De Llobregat · Barcelona
Hospital Universitario Puerta De Hierro Majadahonda
Madrid
Hospital Universitario de Navarra;Unidad de Neuropediatría
Pamploa
Corporacio Sanitaria Parc Tauli
Sabadell
Time Frame
Start Date:2023-07-27
Completion Date:2025-12-02
Participants
Target number of participants:48
Treatments
Experimental: Part 1 Adult Alogabat Dose (Age 15-17)
In Part 1 of the study participants will receive alogabat once a day (QD).
Experimental: Part 1 Age-adjusted Dose (Age 10-14)
In Part 1 of the study, participants will receive age-adjusted QD doses of alogabat.
Experimental: Part 1 Age-adjusted Dose (Age 5-9)
In Part 1 of the study, participants will receive age-adjusted QD doses of alogabat.
Experimental: Part 2 Cohort 1
In 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.
Experimental: Part 2 Cohort 2
In 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.
Experimental: Part 1 Optional Cohort
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.
Experimental: Part 2 Optional Cohort
In 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.