The Clinical Study GTX-102-001

What is the purpose of the clinical study (KIK-AS study)?

The purpose of this study is to see if the investigational drug, GTX-102, is safe and well tolerated in children with Angelman syndrome.

What does KIK-AS stand for?

Knockdown of UBE3A-antisense in kids with Angelman syndrome

Who can take part in the study?

Study participants must be between the ages of 4 and 17 years, have Angelman syndrome, deletion positive genotype, and meet other study related criteria. 

How long does the study last?

Study participation will last approximately 22 weeks.

What is informed consent?

You and your child will be prescreened by the study site for possible enrollment into the clinical trial.  If your child is potentially eligible, you will be given an informed consent form by the study site. Informed consent is the process of learning the key facts and details about a clinical research study. This information will be provided to you before you decide to participate and as new information becomes available during your continued participation throughout the study. Once you have read the informed consent form and understand the details, including the risks and benefits, you can decide whether or not to sign the document. The informed consent process includes opportunities for you to ask questions about the study. No study-related procedures will take place without a signed informed consent form.

What is an investigational drug?

An investigational drug or research study drug is a drug that has not yet been approved by government agencies (such as the USA Food & Drug Administration) for prescription by doctors.

Where can I find additional information about this study?

In addition to this website, has additional information about this study at the following link:

Does my child qualify for study participation?

Please review the following key criteria to determine if your child may be potentially eligible for this study.
  1. Your child must have documented molecular confirmation (laboratory report) of a diagnosis of Angelman syndrome, with maternal deletion of the UBE3A gene. (If you do not have a copy of your child's confirmed diagnosis, you should contact the doctor who confirmed your child's diagnosis and ask for a copy of the laboratory report.)
  2. Your child's seizure activity and management must be stable for 1 month or more.
  3. Your child must be able to tolerate a lumbar puncture procedure (spinal tap), under anesthesia, 4 times during the course of the study.
  4. Your child must be between the ages of 4 and 17.
  5. To the best of your knowledge, your child must have normal kidney and liver function.
  6. Your child must be able to walk independently or with an assistive device.
  7. You are able to travel to one of the study sites 9 times over the course of the study.
  8. Your child must not have any bleeding problems or take a blood thinner medicine.


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