This is a phase 1/2a randomised, placebo controlled, double-blind study investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of ALE1 on healthy adult subjects and adult patients with Hypophosphatasia (HPP).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
120
New Zealand Clinical Research
Grafton, Auckland, New Zealand
RECRUITINGFortrea Clinical Research Unit
Leeds, United Kingdom
RECRUITINGEvaluate the safety of ALE1 by assessing the number of treatment emergent adverse events (TEAEs)
Time frame: From baseline up to day 16
Evaluate safety of ALE1 by assessing the presence of clinically significant changes in participants haematology parameters post-dose
Time frame: From baseline up to day 16
Evaluate safety of ALE1 by assessing the presence of clinically significant changes in participants biochemistry parameters post-dose
Time frame: From baseline up to day 16
Evaluate safety of ALE1 by assessing changes in heart rhythms via electrocardiogram
Time frame: From baseline up to day 16
Evaluate safety of ALE 1 by assessing the presence of clinically significiant changes in participants vital signs
Time frame: From baseline up to day 16
Pharmacokinetic parameter: area under the plasma concentration versus time curve (AUC (D0 - INF))
Time frame: From baseline up to day 16
Pharmacokinetic parameter: time at which maximum plasma concentration occurs (Tmax)
Time frame: From baseline up to day 16
Pharmacokinetic parameter: terminal elimination phase half-life (t(1/2))
Time frame: From baseline up to day 16
Pharmacokinetic parameter: total clearance (CL/F)
Time frame: From baseline up to day 16
Pharmacokinetic parameter: volume of distribution (Vd/F)
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Time frame: From baseline up to day 16
Change in pharmacodynamic biomarker levels in blood samples of ALE1
Time frame: From baseline up to day 16
Dose proportionality of maximum observed plasma concentration (Cmax) Time at which maximum plasma concentration occurs (Tmax)
Time frame: From baseline up to day 16
Dose proportionality of area under the plasma concentration versus time curve (AUC (D0 - INF))
Time frame: From baseline up to day 16
Effect of food on area under the plasma concentration versus time curve (AUC (D0 - INF))
Time frame: From baseline up to 16 days post dose
Effect of food on maximum observed plasma concentration (Cmax)
Time frame: From baseline up to day 16