Kansas City, MONCT07157254Now EnrollingIRB Ready

Angelman Syndrome Clinical Trial in Kansas City, MO

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

Sponsored by Ultragenyx Pharmaceutical Inc

Quick Self-Assessment

See if you qualify for this Kansas City location

Preparing your pre-screening questions…

Expert Care in Kansas City

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

Apply for This Kansas City Location

Check if you qualify for this angelman syndrome clinical trial in Kansas City, MO

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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 Kansas City

    Convenient for MO 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 Kansas City site if eligible
  4. 4Begin participation

About This Angelman Syndrome Study in Kansas City

The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.

Sponsor: Ultragenyx Pharmaceutical Inc

Who Can Participate

Inclusion 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 \< 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 lumbar puncture (LP) procedure, magnetic resonance imaging (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

Exclusion Criteria

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, other) within the month prior to the Screening Visit (excluding weight-based adjustments)
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 interventional study

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 Kansas City?

Yes, this clinical trial (NCT07157254) has an active research site in Kansas City, MO 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 Kansas City, MO

If you're searching for angelman syndrome treatment options in Kansas City, MO, this clinical trial (NCT07157254) 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 Kansas City research site is actively enrolling participants for this 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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