Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3
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|ClinicalTrials.gov Identifier: NCT04637282|
Recruitment Status : Not yet recruiting
First Posted : November 19, 2020
Last Update Posted : December 4, 2020
|Condition or disease||Intervention/treatment||Phase|
|Juvenile Neuronal Ceroid Lipofuscinosis||Drug: PLX-200||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Open-label, non-comparator, within subject, dose-titration trial|
|Masking:||None (Open Label)|
|Official Title:||A Safety, Tolerability, and Efficacy Study of PLX-200 in Participants With Mild-to-Moderate Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Disease|
|Estimated Study Start Date :||February 1, 2021|
|Estimated Primary Completion Date :||February 1, 2023|
|Estimated Study Completion Date :||August 1, 2024|
This is an open-label, non-comparator, non-randomized study of PLX-200 in patients with CLN3 disease.
15 mg/mL oral PLX-200 solution administered twice daily
- Efficacy of PLX-200 in CLN3 as assessed by the mean rate of increase in the Physical domain sub-score of the Unified Batten Disease Rating Scale (UBDRS) clinical rating scale compared with that of the natural history data. [ Time Frame: 149 weeks ]The rate of increase in the Physical Domain sub-score of the UBDRS clinical rating scale will be calculated as the change from Baseline to the last assessment, divided by the length of follow-up (treatment period). Descriptive statistics of the rate of increase will be provided in the PLX-200 and historical cohorts. The scale is from 0 (normal physical assessment) to 104 (severe physical impairment).
- Number of patients with treatment-related adverse events, as assessed by CTCAE v5.0, abnormal laboratory results, and abnormal cardiovascular and/or abdominal findings. [ Time Frame: 149 weeks ]The aggregate of clinical chemistries, hematology, urinalysis, electrocardiogram readings, abdominal ultrasound findings, and tabulation of the number and severity of adverse events will be compared with baseline values throughout the study to evaluate the safety and tolerability of PLX-200 at escalating oral doses in children with CLN3 disease.
- Efficacy of PLX in each of the domains of the Unified Batten Disease Rating Scale (UBDRS) including seizure, behavioral, and functional capabilities. [ Time Frame: 149 weeks ]To evaluate the efficacy of PLX-200 using the individual domain scores of the Unified Batten Disease Rating Scale: seizure, behavioral, and functional capabilities, compared with the Batten Disease Registry at the University of Rochester (BDR-UR) periodically throughout the study period. The seizure domain is a series of items with a maximal total score of 54 (the higher the score, the more severe the disease). The behavioral domain is a series of items with a maximal total score of 56 (the higher the score, the more severe the disease). The capabilities domain is a series of items with a maximal total score of 28 (the higher the score, the more severe the disease).
- Evaluate the Clinical Global Impression (CGI) scale of symptoms [ Time Frame: 149 weeks ]To evaluate the Clinical Global Impression (CGI) scale of symptom severity and change from baseline periodically throughout the study. The scale is from the Unified Batten Disease Rating Scale where each domain (seizures, cognitive function, behavior, mood, motor, and overall) is ranked 0 (normal function) to 5 (severe impairment) with a minimum score of 0 and maximum score of 30.
- Evaluate the efficacy of PLX-200 using quality of life (QoL) assessments, namely the PedsQL Generic Core Scales and the PedsQL Family Impact Module where the higher score reflects better outcomes. [ Time Frame: 149 weeks ]To evaluate the efficacy of PLX-200 using Quality of Life assessments in the pediatric populations utilizing the PedsQL™ Generic Core Scales Version 4.0 and PedsQL™ Family Impact Module Version 2.0. These scales consist of 23 items applicable for pediatric populations with acute and chronic health conditions, encompassing physical function (8 items), emotional functioning (5 items), social functioning (5 items), and school functioning (if applicable; 5 items). The PedsQL Family Impact module is similarly constructed involving the child and parent(s). Items are reverse-scored and linearly transformed to a 0 to 100 scale (0 = 100, 1 = 75, 2 = 50,3 = 25, and 4 = 0), so that higher scores indicate better HRQOL. Scale Scores are computed as the sum of the items divided by the number of items answered (this accounts for missing data). To summarize, a higher score in each of the individual items and total score reflect a healthier child.
- PLX-200 pharmacokinetics (PK) will be evaluated through periodic blood draws during the trial. PLX-200 levels will be determined to estimate area under the curve, maximal concentration, time to maximal concentration, half-life, and dosing interval. [ Time Frame: 12 weeks ]To evaluate PLX-200 plasma concentrations in blood collected from participants during the Titration and Maintenance Periods for PK analyses, namely Cmax (maximal PLX-200 concentration) and Tmax (time to Cmax), elimination half-life (t1/2), area under the plasma concentration-time curve (AUC), and the dosing interval.
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): NCT04637282
|Contact: David R Luke, PharmD||8606085296||DrDavidRLuke@Polaryx.com|
|Contact: Hahn-Jun Lee, PhD||2017241786||HahnJun7@Polaryx.com|
|Study Chair:||Hahn-Jun Lee, PhD||Polaryx Therapeutics|