Niemann-Pick C disease (NPC)
Conditions
Interventions
Approximately 32 healthy volunteers will be enrolled into 1 of 3 cohorts.
Cohort 1 will receive either 800 mg of ESB1609 (n=6) or placebo (n=3), daily
for 14
Cohort 2 will receive either 1600 mg of
Sponsors
ESCAPE Bio, Inc.
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Part 1 Subject Inclusion Criteria 1. The subject has provided written informed consent. 2. The subject is a male 18 to 55 years of age (inclusive at screening) 3. For Cohort 3 B only the subject is a male aged 55-65 years of age
Exclusion criteria
Exclusion criteria: Part 1 Subject Exclusion Criteria 1. The subject has a current or recurrent disease (e.g., cardiovascular, renal, liver, gastrointestinal, malignancy or other conditions) that could affect the action, absorption or disposition of the investigational product or could affect clinical or laboratory assessments. 2. The subject has a clinically significant 12-lead ECG abnormality, including QTc of >450 msec (average of triplicate measures via Fridericia*s corrections) for any Screening or Day -1 ECG assessment. 3. The subject has a history of clinically significant bradycardia syndromes (e.g., sick sinus syndrome) or sinus bradycardia at Screening or Day -1.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The PK analysis will be performed for the PK population. The plasma and CSF concentration-time data will be analyzed by non-compartmental methods, using commercial software such as Phoenix® WinNonlin®. The analysis will be performed using data from all subjects and actual times at which blood samples are obtained will be used in all calculations, except for the determination of Tmax. Plasma and CSF PK parameters will include the following as appropriate based on the observed data: Area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration (AUC0-last) Area under the concentration-time curve from time 0 to 24 hours (AUC0-24) Area under the concentration-time curve from time 0 to infinity (AUC0-*) Area under the concentration-time curve over from time 0-18 hours (Cohort 3B only) (AUC0-18) Maximum plasma drug concentration (Cmax) Time to maximum plasma drug concentration (Tmax) Terminal elimination rate constant (*z) Half-life (t*) Apparent total clearance of the drug from plasma (CL/F) Apparent volume distribution (Vz/F) All safety analyses will be performed on the safety population. Interim safety data will be examined on an ongoing basis to ensure subject safety and to comply with the clinical study dose escalation rules. Summary statistics will be presented descriptively for the following safety endpoints by dose group: Adverse Events - the number of adverse events, the proportion of subjects having at least one adverse event and adverse events by MedDRA coded terms will be presented. Serious Adverse Events - the number of serious adverse events, the proportion of subjects having at least one serious adverse event and serious adverse events by coded terms will be presented. Related Adverse Events - the number of related adverse events, the proportion of subjects having at least one related adverse event and related adverse events by coded terms will be presented. The following wil | — |
Countries
Netherlands
Outcome results
None listed