This (DEEp SEA Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DS. The study consists of 3 main phases: Screening, Titration period, and Maintenance period, followed by a Taper period and Follow-Up. Participants will be randomized to LP352 or placebo. The total duration of the study will be approximately 24 months.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
160
LP352 will be administered orally or through G-tube/ percutaneous endoscopic gastrostomy (PEG) tube
Participants will be administered with matching placebo orally or through G-tube/ PEG tube
Arkansas Children's Hospital - PIN
Little Rock, Arkansas, United States
RECRUITINGDavid Geffen School of Medicine at UCLA
Los Angeles, California, United States
RECRUITINGThe Stanford Division of Child Neurology
Palo Alto, California, United States
RECRUITINGUCSF Children's Hospital
San Francisco, California, United States
Frequency Percent Change in Countable Motor Seizures During Treatment Compared to Baseline
The percent change from Baseline in countable motor seizure frequency during Treatment will be calculated as countable motor seizure frequency during Treatment minus countable motor seizure frequency during Screening and divided by seizure frequency during Screening and multiplied by 100 where each seizure frequency will be based on number of seizures.
Time frame: Baseline and up to 15 Weeks
Safety and Tolerability of LP352
Safety and tolerability as measured by incidence and severity of non-serious Treatment Emergent Adverse Events (TEAEs), Serious Adverse events (SAEs), AEs leading to discontinuation and clinically significant changes in laboratory parameters (hematology, serum chemistry and Urinalysis), physical examination findings, vital signs, growth parameters (height and weight), 12-lead electrocardiograms (ECGs), C-SSRS responses, and PHQ-9 total score and Question 9 score.
Time frame: Up to 21 Weeks
Percentage of participants with ≥ 50% Reduction in countable motor seizures during Treatment compared to Baseline
Time frame: Baseline and up to 15 Weeks
Frequency Percent Change in Countable Motor Seizures during Maintenance compared to Baseline
Time frame: Baseline and up to 15 Weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Children's Hospital Colorado.
Aurora, Colorado, United States
RECRUITINGNW FL Clinical Research Group, LLC
Gulf Breeze, Florida, United States
RECRUITINGNicklaus Children's Hospital - PIN
Miami, Florida, United States
RECRUITINGResearch Institute of Orlando LLC
Orlando, Florida, United States
COMPLETEDPediatric Epilepsy and Neurology Specialists
Tampa, Florida, United States
RECRUITINGRare Disease Research, LLC - Atlanta - RDR - PIN
Atlanta, Georgia, United States
COMPLETED...and 67 more locations