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.

Completed · Phase 2

Conditions studied: Angelman Syndrome

In brief

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.

Key facts

Study ID

NCT05630066

Run by

Hoffmann-La Roche

People needed

48

Starts

2023-07-27

Expected to finish

2025-12-02

Last updated by the study team

2025-12-04

Who can join

Age: 5 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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
• 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

Where it is running

• Rush Medical Center — Chicago, Illinois, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Columbia University Medical Center — New York, New York, United States
• Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine — Carrboro, North Carolina, United States
• Vanderbilt Children's Hospital — Nashville, Tennessee, United States
• Multicare Institute for Research and Innovation — Tacoma, Washington, United States
• Queensland Children?s Hospital — South Brisbane, Queensland, Australia
• CHRU de Brest — Brest, France
• CHU Dijon Bourgogne Hôpital François Mitterand — Dijon, France
• Hopital la Timone Enfants — Marseille, France
• Groupe Hospitalier Necker Enfants Malades — Paris, France
• Dr. Von Haunersches Kinderspital — München, Germany
• Ospedale Pediatrico Bambino Gesù — Rome, Lazio, Italy
• IRCCS Istituto G. Gaslini — Genoa, Liguria, Italy
• IRCCS Eugenio Medea — Conegliano Veneto (TV), Veneto, Italy
• Hospital Sant Joan de Deu — Esplugues de Llobregat · Barcelona, Barcelona, Spain
• Corporacio Sanitaria Parc Tauli — Sabadell, Barcelona, Spain
• Hospital Universitario de Navarra;Unidad de Neuropediatría — Pamploa, Navarre, Spain
• Hospital Universitario Puerta De Hierro Majadahonda — Madrid, Spain

Full record on ClinicalTrials.gov

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