Niemann-Pick C disease (NPC)
Conditions
Interventions
IMP:
Part 1:
ESB1609 jetmilled Form A capsules.
ESB1609 jetmilled Form A suspension in 20 mL volume, 50 wt% Captisol, 0.2 wt%
HPMC-E50
ESB1609 jetmilled Form A tablet, 60 wt% ESB1609, 500 mg
Compar
Sponsors
ESCAPE Bio
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: 1. The subject has provided written informed consent. 2. The subject is a male aged between 18 to 55 years (inclusive) at the time of Screening. 3. The subject*s BMI is between 18.0 and 30.0 kg/m2 (inclusive) at Screening and on Day -1 of the (first) treatment period. 4. If the subject is sexually active, he agrees to comply with the contraceptive requirements outlined in the protocol during the study and for 3 months after last study drug dosing.
Exclusion criteria
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 any QTc >= 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 bradycardia syndromes (e.g., sinus bradycardia, sick sinus syndrome) or sinus bradycardia at Screening or Day -1. 4. The subject has a resting heart rate of 40 beats per minute or less at Screening, on Day -1, or prior to dosing on Day 1 of the (first) treatment period. 5. The subject has a history of risk factors for Torsades de Pointes (e.g., heart failure, hypokalemia, family history of Long QT Syndrome).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part 1: • PK parameters for ESB1609 include: Cmax, AUC0-t, AUC0-inf, geometric mean ratios (GMRs) and associated 90% confidence intervals (CIs) of observed PK exposure measures for treatments A/B and A/C (Cohort 1a). • For additional forms and/or formulations of ESB1609, PK parameters for ESB1609 include: Cmax, AUC0-t, AUC0-inf, GMRs and associated 90% CIs of observed PK exposure measures for the treatments for additional cohorts between treatment periods following the same endpoint analysis plan for treatments as Cohort 1a. Part 2: • PK parameters for ESB1609 include: Cmax, AUC0-t, AUC0-inf, GMRs and associated 90% CIs of observed PK exposure measures with and without fluconazole. Part 3: • PK parameters for ESB1609 include: Cmax, AUC0-t, AUC0-inf, GMRs and associated 90% CIs of observed PK exposure measures with and without rifampicin. | — |
Secondary
| Measure | Time frame |
|---|---|
| All parts: • Safety and tolerability parameters include: physical examinations, vital signs, 12-lead ECGs, clinical laboratory tests, C-SSRS, and AEs. | — |
Countries
Netherlands
Outcome results
None listed