Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects
NCT Number: NCT06481709
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE1
Conditions: Bioavailability
Interventions: DRUG: RLF-OD032|DRUG: Kuvan
Sponsor: APR Applied Pharma Research s.a.
Sex: ALL
Age: ADULT
Enrollment: 16
Study Design: Allocation: RANDOMIZED|Intervention Model: CROSSOVER|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-07-30
Completion Date: 2024-10-30
Locations: Pharma Medica Research Inc. 4770 Sheppard Ave E,, Toronto, Ontario, M1S3V6, Canada

Summary:
This is a single center, Phase 1, randomized, open-label, single-dose, 4 treatment, 4-period, 4-sequence, crossover study designed to compare the pharmacokinetics (PK) of sapropterin from the Test and Reference products, and to evaluate the effect of food and the effect of water administration on the bioavailability of sapropterin from the Test product in healthy subjects.

.

Primary Outcome Measures: Uncorrected and baseline-corrected sapropterin AUC0-t, Area under the concentration-time curve from time zero until the last observed concentration, as calculated by the linear up/log down variant of the trapezoidal method, Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.|Uncorrected and baseline-corrected sapropterin AUC0-inf, Area under the concentration-time curve from time zero to infinity (extrapolated), Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.|Uncorrected and baseline-corrected sapropterin Cmax, Maximum observed concentration, Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Secondary Outcome Measures: Uncorrected and baseline-corrected sapropterin Tmax, Time when the maximal concentration is observed, Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.|Uncorrected and baseline-corrected sapropterin Tlag, Time of observation prior to the first observation with a measurable (non-zero) concentration, Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.|Uncorrected and baseline-corrected sapropterin T½ el, Terminal elimination half-life, Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.|Uncorrected and baseline-corrected sapropterin Residual Area, Percentage of AUC0-inf due to extrapolation from the time of the last observed concentration to infinity, calculated as \[1 - (AUC0-t/AUC0-inf)\] x 100, Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.|Uncorrected and baseline-corrected sapropterin Kel, Terminal elimination rate constant, Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.|Uncorrected and baseline-corrected sapropterin Cl/F, Apparent clearance, Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.|Safety and Tolerability Analysis of sapropterin dihydrochloride when administered to healthy subjects., Assessment of AEs. AEs will be coded using the latest version of the Medical Dictionary for Regulatory Activities (MedDRA)., Safety monitored from the signature of the Informed Consent through study completion, approximately 24 days