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A Safety and Efficacy Study of GTX-102 in Subjects with Deletion- or Nondeletion-type Angelman Syndrome (Aurora)
EU CTIS ID: 2024-519393-39-00
What this study is testing
Primary Safety Objective: evaluate the safety of GTX-102. Primary Efficacy Objective: evaluate the efficacy of GTX-102.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Signed informed consent from parent(s) or legal guardian(s)
- Males and females of the following ages and genotypes at time of informed consent: a. Subprotocol A: ≥ 1 to < 4 years of age with a genetically confirmed diagnosis of deletion-type Angelman Syndrome b. Subprotocol B: ≥ 4 to < 18 years of age with a genetically confirmed diagnosis of UPD/ICD Angelman Syndrome c. Subprotocol C: ≥ 18 to < 65 years of age with a genetically confirmed diagnosis of Angelman Syndrome, any genotype d. Subprotocol D: ≥ 4 to < 18 years of age with a genetically confirmed diagnosis of mutation-type Angelman Syndrome.
- Weight ≥ 8 kg at Screening Visit.
- Prothrombin time / international normalized ratio, and partial thromboplastin time < 1.5x the upper limit of normal and platelets > 75,000 cells/mm3 at the Screening Visit.
- Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, and all study procedures, including LP procedure, MRI, and tolerating anesthesia without intubation.
- From the time of informed consent through to at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102.
You likely can't join if
- Any change in medications or diet/supplements intended to treat symptoms of AS (eg, sleeping aids, antiseizure medications, supplements, dietary change including ketogenic or low-glycemic index diet, other) within the month prior to the Screening Visit (excluding weight-based adjustments).
- Any condition that creates an increased risk of unsuccessful LP.
- Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors).
- Known hypersensitivity to GTX-102 or its excipients or required premedication that, in the judgment of the Investigator, places the subject at increased risk for adverse effects.
- Presence or history of any condition, lab abnormality, or infection that, in the judgement of the Investigator, would interfere with study participation, pose undue safety risk, or would confound interpretation of results.
- Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study.
See the full eligibility criteria
Who can join
- Signed informed consent from parent(s) or legal guardian(s)
- Males and females of the following ages and genotypes at time of informed consent: a. Subprotocol A: ≥ 1 to < 4 years of age with a genetically confirmed diagnosis of deletion-type Angelman Syndrome b. Subprotocol B: ≥ 4 to < 18 years of age with a genetically confirmed diagnosis of UPD/ICD Angelman Syndrome c. Subprotocol C: ≥ 18 to < 65 years of age with a genetically confirmed diagnosis of Angelman Syndrome, any genotype d. Subprotocol D: ≥ 4 to < 18 years of age with a genetically confirmed diagnosis of mutation-type Angelman Syndrome.
- Weight ≥ 8 kg at Screening Visit.
- Prothrombin time / international normalized ratio, and partial thromboplastin time < 1.5x the upper limit of normal and platelets > 75,000 cells/mm3 at the Screening Visit.
- Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, and all study procedures, including LP procedure, MRI, and tolerating anesthesia without intubation.
- From the time of informed consent through to at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102.
What rules you out
- Any change in medications or diet/supplements intended to treat symptoms of AS (eg, sleeping aids, antiseizure medications, supplements, dietary change including ketogenic or low-glycemic index diet, other) within the month prior to the Screening Visit (excluding weight-based adjustments).
- Any condition that creates an increased risk of unsuccessful LP.
- Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors).
- Known hypersensitivity to GTX-102 or its excipients or required premedication that, in the judgment of the Investigator, places the subject at increased risk for adverse effects.
- Presence or history of any condition, lab abnormality, or infection that, in the judgement of the Investigator, would interfere with study participation, pose undue safety risk, or would confound interpretation of results.
- Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study.
- Use of any investigational product or investigational medical device within 6 months or 5 half-lives prior to the Screening Visit, or any prior use of gene therapy or an ASO regardless of length of time since last use.
- Concurrent participation in any interventional study.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
- Israel
- Brazil
- Argentina
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom; Israel; Brazil; Argentina. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.