RecruitingNCT06115109

Angelman Natural History Study - FAST Spain

Observational Study, Prospective Multicentric, From the Natural History of Patients With Angelman Syndrome in Spain

Sponsor

Puerta de Hierro University Hospital

Enrollment

40 participants

Start Date

Dec 1, 2023

Study Type

OBSERVATIONAL

Conditions

Angelman Syndrome

Summary

The goal of this study is to conduct a prospective, longitudinal study to observe the natural clinical progression and disease outcome of AS patients receiving no disease-modified intervention, with the purpose of obtaining data that will be useful for future clinical trials.

Eligibility

Min Age: 3 MonthsMax Age: 99 Years

Inclusion Criteria4

Male or female between 3 months and 99 years of age.
Clinical diagnosis of Angelman Syndrome and molecular confirmation of the diagnosis.
The participant has an acceptable guardian capable of giving consent on behalf of the participant,
Willingness and ability of their guardians to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.

Exclusion Criteria3

Patients with any comorbidity that, in the clinical judgment of the principal investigator, may affect the results of the study. Any confirmed chronic or acute condition or illness affecting any system(s) that could interfere with study results and/or compliance with study procedures is included.
Patients who are participating in parallel studies with investigational drugs.
Unwillingness or inability of their guardians to follow the procedures outlined in the protocol.

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