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Tests treatment safety and results for Angelman Syndrome
Official title A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
ClinicalTrials.gov ID: NCT07157254
What this study is testing
What is GTX-102?
GTX-102 is an investigational medicine, given as a treatment applied to the skin, being studied as a potential treatment for angelman syndrome.
Also referred to as apazunersen.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 64
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:
- Subprotocol A: ≥ 1 to \< 4 years of age with a genetically confirmed diagnosis of deletion-type Angelman syndrome
- Subprotocol B: ≥ 4 to \< 18 years of age with a genetically confirmed diagnosis of UPD/ICD Angelman syndrome
- Subprotocol C: ≥ 18 to \< 65 years of age with a genetically confirmed diagnosis of Angelman syndrome, any genotype
You likely can't join if
- Any change in medications or diet/supplements intended to treat symptoms of Angelman Syndrome (eg, sleeping aids, antiseizure medications...
- Any condition that creates an increased risk of unsuccessful lumbar puncture
- 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...
- Presence or history of any condition, lab abnormality, or infection that, in the judgment of the Investigator, would interfere with study...
- Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
See the full eligibility criteria
- Signed informed consent from parent(s) or legal guardian(s)
- Males and females of the following ages and genotypes at time of informed consent:
- Subprotocol A: ≥ 1 to \< 4 years of age with a genetically confirmed diagnosis of deletion-type Angelman syndrome
- Subprotocol B: ≥ 4 to \< 18 years of age with a genetically confirmed diagnosis of UPD/ICD Angelman syndrome
- Subprotocol C: ≥ 18 to \< 65 years of age with a genetically confirmed diagnosis of Angelman syndrome, any genotype
- Subprotocol D: ≥ 4 to \< 18 years of age with a genetically confirmed diagnosis of mutation-type Angelman syndrome
- Weight ≥ 8 kg at Screening Visit
- Platelet count, prothrombin time / international normalized ratio, and partial thromboplastin time \ 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 lumbar puncture (LP) procedure, magnetic resonance imaging (MRI) and tolerating...
- 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...
- Any change in medications or diet/supplements intended to treat symptoms of Angelman Syndrome (eg, sleeping aids, antiseizure medications, supplements, dietary change including ketogenic or low-glycemic index diet...
- Any condition that creates an increased risk of unsuccessful lumbar puncture
- 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 judgment 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 treatment study
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Chicago, Illinois, United States
- Baltimore, Maryland, United States
- Kansas City, Missouri, United States
- Hillsborough, North Carolina, United States
- Akron, Ohio, United States
- Austin, Texas, United States
- Dallas, Texas, United States
- Pilar, Buenos Aires, Argentina
- Curitiba, Paraná, Brazil
- Santa Cecília, Porto Alegre, Brazil
- Marseille, France
- Paris, France
- Ramat Gan, Israel
- Florence, Italy
- Milan, Italy
- Rome, Italy
- Lisbon, Portugal
- Porto, Portugal
- London, United Kingdom
+ 1 more site(s).
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, 1 year to 64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Chicago, Illinois, United States; Baltimore, Maryland, United States; Kansas City, Missouri, United States; Hillsborough, North Carolina, United States; Akron, Ohio, United States and 15 more location(s). 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 ClinicalTrials.gov; the study team decides eligibility.