Study on the Long-Term Safety of GTX-102 for Patients with Angelman Syndrome

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What is this study about?

This clinical trial is focused on studying the long-term safety and effectiveness of a treatment called GTX-102 for individuals with Angelman Syndrome. Angelman Syndrome is a genetic disorder that affects the nervous system and can cause developmental delays, speech difficulties, and balance issues. The treatment being tested, GTX-102, is a special type of medication known as an antisense oligonucleotide, which is designed to target specific genetic material in the body. It is administered as a solution for injection directly into the spinal fluid, a method known as intrathecal use.

The purpose of this study is to evaluate how safe GTX-102 is for patients over a long period. Participants in the trial will receive the treatment and be monitored for any side effects or changes in their condition. The study will also involve the use of Elliotts B Solution, a buffered intrathecal electrolyte/dextrose injection, which contains substances like calcium chloride, glucose, and sodium chloride. This solution is used to help deliver the treatment safely.

Throughout the study, participants will undergo regular assessments to track their progress and any changes in their symptoms. These assessments will include evaluations of cognitive abilities, communication skills, and motor functions. The study aims to provide valuable information on the long-term use of GTX-102 in managing Angelman Syndrome, helping to determine its potential benefits and any risks associated with its use.

1 joining the study

Participation begins after prior involvement in a clinical trial with GTX-102. Completion of the loading phase in the previous study is required.

The screening visit must occur within 6 months of the last visit in the prior GTX-102 study.

2 medication administration

The medication used in this trial is GTX-102, also known as ELLIOTTS B SOLUTION, which is a buffered intrathecal electrolyte/dextrose injection.

The medication is administered as a solution for injection through intrathecal use, which means it is injected into the space around the spinal cord.

3 safety monitoring

The main objective is to evaluate the long-term safety profile of GTX-102 in patients with Angelman Syndrome.

Monitoring includes tracking any treatment-emergent adverse events (AEs) and serious adverse events (SAEs), their frequency, severity, and relationship to the investigational product throughout the study.

4 efficacy assessment

Secondary assessments involve measuring changes from the start of the long-term extension (LTE) phase and pretreatment in various developmental scores.

These scores include Bayley-4 Cognition, Receptive Communication, Expressive Communication, Gross Motor, and Fine Motor raw scores.

5 trial duration

The estimated end date for the trial is October 2, 2029.

The estimated start date for recruitment is October 2, 2024.

Who Can Join the Study?

The patient must have a signed informed consent from their parent(s) or legal guardian(s). This means that the parent or guardian agrees to let the patient participate in the study after understanding all the details.
The patient must have previously participated in a clinical trial with GTX-102 and completed a specific part of that trial called the Loading phase. The patient must also have approval from the Ultragenyx Medical Monitor. The first visit for this study must happen within 6 months after the last visit in the previous GTX-102 study.
Females who can have children and are sexually active must use highly effective birth control methods or choose not to have sexual intercourse from the time they agree to join the study until at least 6 months after the last dose of GTX-102. Males can participate if they agree to not have sexual intercourse or use acceptable birth control methods during the study and for at least 3 months after the last dose of GTX-102.

Who Cannot Join the Study?

Patients who have a history of severe allergic reactions to the study medication.
Patients who are currently participating in another clinical trial.
Patients who have a medical condition that the study doctors believe would make it unsafe for them to participate.
Patients who are unable to follow the study procedures or instructions.
Patients who are pregnant or breastfeeding.
Patients who have a history of substance abuse that could interfere with the study.
Patients who have a serious mental health condition that could affect their ability to participate.
Patients who have had a major surgery within the last 3 months.
Patients who have an infection that requires treatment with antibiotics.
Patients who have a history of certain heart conditions.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Hopital Necker Enfants Malades	Paris	France
Universitaet Leipzig	Leipzig	Germany

Other Sites

Site Name	City	Country
Hospital Universitario Puerta De Hierro De Majadahonda	Majadahonda	Spain
Uzpvqzewlq Mgfmvkg Cfzhyq Hgkrbtpvjezmrkvod	Hamburg	Germany
Pcgq Txxhv Hfsdinit Utsubjzzlzmi	Sabadell	Spain
Aabiczxyww Psuijjnb Hiwheofj Dc Mfykuaxej	Marseille	France

Trial status

Country	Status	Recruitment Start
France	Recruiting	02.10.2024
Germany	Recruiting	02.10.2024
Spain	Recruiting	02.10.2024

Trial locations

Investigated drugs:

GTX-102 is a medication being studied for its safety and effectiveness in patients with Angelman Syndrome. This trial aims to understand how well patients tolerate the medication over a long period and to gather information on any potential side effects. Angelman Syndrome is a genetic disorder that affects the nervous system, and this medication is being tested to see if it can help manage symptoms associated with the condition.

Investigated diseases:

Angelman's syndrome

Angelman Syndrome – Angelman Syndrome is a rare genetic disorder that primarily affects the nervous system. It is characterized by developmental delays, intellectual disability, severe speech impairment, and problems with movement and balance. Individuals with this condition often exhibit a happy demeanor, frequent smiling, and laughter. The syndrome is caused by a loss of function of the UBE3A gene on chromosome 15. Symptoms typically become noticeable in early childhood, and while they can vary, they generally include seizures and sleep disturbances. The progression of the disorder involves persistent developmental challenges and requires ongoing support.

Trial ID:

2024-510917-14-00

Protocol code:

GTX-102-CL302

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Long-Term Safety of GTX-102 for Patients with Angelman Syndrome

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?