Carrboro, NCNCT07782827Now EnrollingIRB Ready

Angelman Syndrome Clinical Trial in Carrboro, NC

Access cutting-edge angelman syndrome treatment through this phase 3 clinical trial at a research site in Carrboro. Study-provided care at no cost to qualified participants.

Sponsored by Ionis Pharmaceuticals, Inc.

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Expert Care in Carrboro

Access angelman syndrome specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related angelman syndrome treatment provided free

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Check if you qualify for this angelman syndrome clinical trial in Carrboro, NC

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Carrboro

    Convenient for NC residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Carrboro site if eligible
  4. 4Begin participation

About This Angelman Syndrome Study in Carrboro

The primary purpose of the study is to evaluate efficacy of obudanersen in participants with AS due to uniparental disomy or imprinting defects (UPD/ID) as measured through expressive communication.

Sponsor: Ionis Pharmaceuticals, Inc.

Who Can Participate

Inclusion Criteria

Clinical diagnosis of AS with molecular result indicating either paternal UPD of 15q11.2-q13 or ID of the maternal 15q11.2-q13 region, provided by the Investigator and confirmed by either a qualified central vendor or a qualified local geneticist at the site.
The participant's caregiver(s)/legally-authorized representative (LAR) must have given written informed consent and any authorizations required by local law and be able and willing to comply with all study requirements.
Medically stable and can undergo sedation and/or general anesthesia without intubation.
Male or female between 2 and ≤ 50 years of age, depending on the specific cohort, at the time of the in-clinic Screening visit.
If applicable, is currently receiving stable doses of concomitant medications typically prescribed for AS, such as anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and special diets, supplements, or nutritional support for at least 8 weeks prior to the Baseline visit. If recent changes (\< 8 weeks stable) in medications, the participant may be allowed per Investigator judgment if the change is not expected to have an impact on the signs and symptoms of AS.
LAR/caregiver(s) agree(s) not to post any of the participant's personal medical data or information related to the study on any website or social media site (e.g., Facebook, Instagram, X, YouTube, TikTok, WhatsApp) from the time of enrollment until they are notified that the study is completed. Key

Exclusion Criteria

Participant has a clinical diagnosis of AS with molecular confirmation of a UBE3A deletion or UBE3A mutation.
Any clinically significant abnormalities in medical history (e.g., major surgery within 3 months of Screening), or on physical examination for which treatment with an antisense oligonucleotide (ASO) would be contraindicated or which, in the opinion of the Investigator, could confound the results of this study.
Known brain or spinal disease that would interfere with the lumbar puncture (LP) procedure, cerebrospinal fluid (CSF) circulation, or presence of other factors that would affect the safety of the LP procedure, including tumors or abnormalities by MRI or computed tomography (CT), subarachnoid hemorrhage, suggestion of raised intracranial pressure (ICP) on magnetic resonance imaging (MRI) or ophthalmic examination, Chiari malformation, obstructive hydrocephalus, syringomyelia, tethered spinal cord syndrome, or connective tissue disorders such as Ehlers-Danlos syndrome and Marfan syndrome.
Any laboratory abnormalities or any other clinically significant abnormalities that would, as assessed by the Investigator, at Screening or Baseline, render a participant unsuitable for inclusion.
Previous treatment with an oligonucleotide (including small interfering ribonucleic acid \[siRNA\] and ASOs) or gene therapy or gene editing. This exclusion criterion does not apply to approved nucleic acid-based vaccines, including messenger Ribonucleic Acid (mRNA) vaccines, which are allowed. Other inclusion/exclusion criteria may apply

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Carrboro?

Yes, this clinical trial (NCT07782827) has an active research site in Carrboro, NC that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Angelman Syndrome Treatment Options in Carrboro, NC

If you're searching for angelman syndrome treatment options in Carrboro, NC, this clinical trial (NCT07782827) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Carrboro research site is actively enrolling participants for this phase 3 clinical trial. You'll receive care from experienced angelman syndrome specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all angelman syndrome clinical trials near you to find additional studies recruiting in your area.

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