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REVEAL Study: Clinical Trial Investigating Potential Treatment for people with Angelman Syndrome

Pediatric | Angelman Syndrome | Adult | Clinical Trial

The REVEAL study is a clinical trial evaluating an study drug, ION582, in individuals with Angelman syndrome. Currently, there are no approved therapies that address the underlying cause of Angelman syndrome. ION582 is being studied as a potential treatment for Angelman syndrome.

This study is done to find out how safe and effective ION582 is in treating Angelman syndrome compared to placebo. Placebo looks the same as ION582 but does not have any medicine-related effects on the body.

The REVEAL study will also evaluate ION582’s impact on communication, intellectual ability, motor skills, seizures, and sleep among other important factors.

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Conditions de participation

  • Sexe:

    Any

  • Âges admissibles:

    2 to 50

Critères de participation

Inclusion Criteria:
- Aged 2-50 years
- Able to undergo conscious sedation
- Confirmed diagnosis of Angelman syndrome with either a UBE3A deletion or UBE3A mutation
- On stable doses of symptom treatment (example: behavioral management medications, sleep medications, etc.) as applicable
Exclusion Criteria:
- No known brain or spinal disease that can interfere with the safety of a lumbar puncture procedure
- No cancer within 5 years (skin cancer may be an exception)
- HIV infection
- Active hepatitis C or B infection
- Uncontrolled high blood pressure
- Previous treatment with an oligonucleotide (including small interfering RNA [siRNA], ASOs) or gene therapy.
- History of experimental brain surgery
- Have a central nervous system catheter
- Pregnancy or planned pregnancy

Lieu de l'étude

University of Alberta
University of Alberta
Edmonton, Alberta
Canada

Contactez l'équipe d'étude

Primary Contact

Casey Brodie

[email protected]
780-492-9277
Étude parrainée par
University of Alberta
Participants recherchés
Plus d'informations
ID de l'étude: Pro00156950