- ICH GCP
- Yhdysvaltain kliinisten tutkimusten rekisteri
- Kliininen tutkimus NCT07655492
Developing a Tailored Neuropsychological Rehabilitation for Sturge-Weber Syndrome (NPS-SWS)
Exploring the Neuropsychological Profile of Sturge-Weber Syndrome for Developing Tailored Rehabilitation.
The goal of this observational study is to learn about the neuropsychological profile of Sturge-Weber Syndrome (SWS) in children and adults with this rare neurocutaneous condition. SWS affects approximately 1 in 50,000 live births and is characterized by brain blood vessel malformations, facial port-wine stains, and abnormal vascularization in the brain, skin, and eyes. Patients are at high risk for epileptic seizures, stroke-like episodes, glaucoma, and motor and cognitive difficulties. The main questions it aims to answer are:
- What are the specific cognitive strengths and weaknesses in visuospatial abilities, working memory, and executive functions in individuals with SWS?
- What is the detailed neuropsychological profile of patients with SWS who do not have intellectual disability?
- Are there different cognitive-behavioral phenotypes between patients with and without the characteristic facial port-wine stain (PWS)?
- How do clinical variables such as seizure history and brain involvement patterns relate to specific cognitive deficits?
Participants will undergo a comprehensive neuropsychological assessment battery that includes:
- Intellectual functioning tests (K-BIT2) to measure verbal and non-verbal intelligence
- Executive function evaluation (BRIEF2) assessing behavioral regulation, emotional regulation, and cognitive regulation
- Language assessment including receptive vocabulary (PPVT) and grammatical comprehension (TCGB-2)
- Visuospatial skills testing (Beery-Buktenica VMI) evaluating visual-motor integration
- Working memory assessment (WISC-IV/WAIS-IV digit span and spatial span subtests)
- Learning abilities evaluation including reading (Battery for the Assessment of - Developmental Dyslexia and Dysorthography), writing, and mathematical skills (ABCA test)
- Additional assessments for attention, verbal memory, and spatial memory as needed
The study will recruit SWS patients through collaboration with patient associations and Telethon support. Participants will continue their standard medical care throughout the study, and all medications and therapies will be recorded. The neuropsychological testing will be conducted at IRCCS San Raffaele and Sapienza University Department of Psychology.
The study aims to identify a cognitive-behavioral phenotype for SWS, develop evidence-based guidelines for neuropsychological monitoring, create personalized recommendations for educational adaptations, produce training materials for healthcare professionals and educators, and establish a multidisciplinary framework for supporting individuals with SWS. This research addresses a critical knowledge gap, as previous studies have focused mainly on general intellectual functioning and the prevalence of intellectual disability and language disorders, without providing detailed neuropsychological profiles, particularly for patients without intellectual disability.
Tutkimuksen yleiskatsaus
Tila
Opintotyyppi
Ilmoittautuminen (Arvioitu)
Yhteystiedot ja paikat
Opiskeluyhteys
- Nimi: Laura Piccardi, PhD
- Puhelinnumero: +39 06 49917527
- Sähköposti: laura.piccardi@uniroma1.it
Opiskelupaikat
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Roma
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Roma, Roma, Italia, 00185
- Rekrytointi
- Department of Psychology University of Rome
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Ottaa yhteyttä:
- Laura Piccardi, PhD
- Puhelinnumero: +39 06 49917527
- Sähköposti: laura.piccardi@uniroma1.it
-
-
Osallistumiskriteerit
Kelpoisuusvaatimukset
Opintokelpoiset iät
- Lapsi
- Aikuinen
- Vanhempi Aikuinen
Hyväksyy terveitä vapaaehtoisia
Näytteenottomenetelmä
Tutkimusväestö
Kuvaus
Inclusion Criteria:
- Confirmed diagnosis of Sturge-Weber Syndrome (SWS) of any type (Type I, Type II, or Type III) based on clinical and/or neuroimaging findings
- Age 2 years or older at the time of enrollment (children, adolescents, and adults are eligible)
- Ability to cooperate with neuropsychological testing procedures
- Willingness to complete all study assessments within the designated timeframe
Exclusion Criteria:
- Absence of confirmed Sturge-Weber Syndrome (SWS)
- Age younger than 2 years at the time of enrollment
- Inability to cooperate with neuropsychological testing procedures even with accommodations or modifications
Opintosuunnitelma
Miten tutkimus on suunniteltu?
Suunnittelun yksityiskohdat
Mitä tutkimuksessa mitataan?
Ensisijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
|---|---|---|
|
1. Composite neuropsychological profile across visuospatial, working memory, and executive function domains
Aikaikkuna: At baseline assessment
|
Assessment of previously uncharacterized cognitive domains in Sturge-Weber Syndrome using three standardized measures administered at baseline: (1) Beery-Buktenica VMI for visuospatial integration; (2) WISC-IV/WAIS-IV Working Memory Index for working memory capacity; (3) BRIEF2 Global Executive Composite for executive functioning. Each measure is scored according to its own normative standard (standard scores Mean=100 SD=15, or T-scores Mean=50 SD=10), then converted to a uniform z-score scale to allow cross-domain comparison and aggregation into a single composite profile score (mean z-score across the three domains). Performance on the composite is classified as impaired (z < -1.5), borderline (z between -1.5 and -1.0), average (z between -1.0 and +1.0), or above average (z > +1.0). Unit of Measure: Mean z-score (composite across three domains) |
At baseline assessment
|
Toissijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
|---|---|---|
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2. Composite cognitive, linguistic, and academic functioning profile in Sturge-Weber Syndrome
Aikaikkuna: At baseline assessment
|
Multidimensional assessment of language, intellectual, and academic functioning using standardized measures administered at baseline. Domains assessed: (1) Language: receptive vocabulary (PPVT) and grammatical comprehension (TCGB-2); (2) General intellectual functioning: K-BIT2 Composite IQ; (3) Academic skills: reading accuracy/speed, writing/spelling accuracy, and mathematical abilities (Battery for Dyslexia/Dysorthographia and ABCA); (4) Supplementary attention and memory when clinically indicated (BVN subtests). All measures are converted to a uniform z-score scale relative to age-based norms and aggregated into a domain-level composite score for each of the four areas above. A global secondary composite (mean z-score across all available domains) summarizes overall functioning. Performance classified as impaired (z < -1.5), borderline (z between -1.5 and -1.0), average, or above average (z > +1.0) per domain. Unit of Measure: Mean z-score (composite across domains) |
At baseline assessment
|
Yhteistyökumppanit ja tutkijat
Sponsori
Tutkijat
- Päätutkija: Laura Piccardi, PhD, University of Roma La Sapienza
Opintojen ennätyspäivät
Opi tärkeimmät päivämäärät
Opiskelun aloitus (Arvioitu)
Ensisijainen valmistuminen (Arvioitu)
Opintojen valmistuminen (Arvioitu)
Opintoihin ilmoittautumispäivät
Ensimmäinen lähetetty
Ensimmäinen toimitettu, joka täytti QC-kriteerit
Ensimmäinen Lähetetty (Todellinen)
Tutkimustietojen päivitykset
Viimeisin päivitys julkaistu (Todellinen)
Viimeisin lähetetty päivitys, joka täytti QC-kriteerit
Viimeksi vahvistettu
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Muita asiaankuuluvia MeSH-ehtoja
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