Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies (ENVISION)
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|ClinicalTrials.gov Identifier: NCT04537832|
Recruitment Status : Active, not recruiting
First Posted : September 3, 2020
Last Update Posted : May 16, 2022
|Condition or disease||Intervention/treatment|
|Dravet Syndrome||Other: No Intervention|
|Study Type :||Observational|
|Actual Enrollment :||58 participants|
|Official Title:||ENVISION: Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies|
|Actual Study Start Date :||January 18, 2021|
|Estimated Primary Completion Date :||March 2024|
|Estimated Study Completion Date :||March 2024|
SCN1A-positive Dravet Syndrome
Participants aged between 6 and 60 months of age who have SCN1A-positive Dravet Syndrome. Clinical, neurocognitive, laboratory, the burden of disease, and health care resource utilization will be assessed.
Other: No Intervention
- Seizure burden [ Time Frame: Change from Baseline at 24 months ]Measured using monthly seizure frequency derived from seizure diaries.
- Seizure freedom [ Time Frame: Change from Baseline at 24 months ]Measured using the proportion of seizure-free days observed.
- Use of anti-seizure medication(s) [ Time Frame: Baseline through Month 24 ]Measured using the incidence of anti-seizure medication usage observed during the 60 days leading up to each nominal visit.
- Use of Special Diet [ Time Frame: Change from Baseline at 24 months ]Measured using the incidence of ketogenic/high-fat diet usage observed during the 60 days leading up to each nominal visit.
- Cognitive functioning [ Time Frame: Change from Baseline at 24 months ]
Measured using composite scores from 3 domains in the Bayley Scales of Infant and Toddler Development (3rd Edition) instrument. Domains include: (1) Cognitive; (2) Language; (3) Motor.
Composite scores are normalized to a mean and SD of 100 and 15, respectively (range is not applicable as the scores are unbounded). Higher scores correspond to better outcomes compared to a normal population.
- Behavioral and social functioning [ Time Frame: Change from Baseline at 24 months ]
Measured using raw scores from 2 domains in the Brief Infant Toddler Social Emotional Assessment. Domains include: (1) Problem; and (2) Competence.
Domain raw scores range from 31 to 93 and 11 to 33 for the Problem and Competence domains, respectively. Higher Problem scores correspond to worse outcomes. Higher Competence scores correspond to better outcomes
- Motor functioning [ Time Frame: Baseline through Month 24 ]Measured using categorical outcomes of 7 motor items adapted from the Bayley Scales of Infant and Toddler Development instrument and NorthStar Ambulatory Assessment. Motor milestones include: (1) Sit unassisted for 30 seconds; (2) Walk with assistance; (3) Stand alone; (4) Walk alone; (5) Walk upstairs; (6) Run with Coordination; and (7)Jump forward.
- Incidence of Adverse Events [ Time Frame: Baseline through Month 24 ]Measured using the incidence of adverse events and serious adverse events (broken down by preferred term) observed during the study.
- Overall survival [ Time Frame: Baseline through Month 24 ]Measured using the incidence of death observed by a given time point during the study.
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04537832
|United States, California|
|Children's Hospital Los Angeles|
|Los Angeles, California, United States, 90027|
|UCSF Benioff Children's Hospital|
|San Francisco, California, United States, 94143|
|United States, Colorado|
|Children's Hospital Colorado|
|Aurora, Colorado, United States, 80045|
|United States, Florida|
|Nicklaus Children's Hospital|
|Miami, Florida, United States, 33155|
|United States, Illinois|
|Ann and Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60611|
|United States, New Jersey|
|Northeast Regional Epilepsy Group|
|Hackensack, New Jersey, United States, 07601|
|United States, Ohio|
|Abigail Wexner Research Institute at Nationwide Children's Hospital|
|Columbus, Ohio, United States, 43205|
|United States, Oregon|
|Oregon Health and Science University|
|Portland, Oregon, United States, 97239|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|United States, Tennessee|
|Le Bonheur Children's Hospital|
|Memphis, Tennessee, United States, 38103|
|United States, Texas|
|Cook Children's Medical Center|
|Fort Worth, Texas, United States, 76104|
|United States, Washington|
|Multicare Institute for Research and Innovation|
|Tacoma, Washington, United States, 98405|
|Austin Hospital - Melbourne Brain Centre|
|Heidelberg, Victoria, Australia, 3084|
|Hospital de la Santa Creu i Sant Pau|
|Hospital Universitari i Politècnic La Fe|
|Queen Elizabeth Hospital|
|Glasgow, United Kingdom, G51 4TF|
|Study Director:||Salvador Rico, M.D., Ph.D||Encoded Therapeutics|