Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)
- Registration Number
- NCT04085666
- Lead Sponsor
- Société des Produits Nestlé (SPN)
- Brief Summary
This study is an international, multi-center, randomized, double-blind, placebo-controlled, two-treatment, two-period cross-over study to evaluate the pharmacodynamics, safety, tolerability and pharmacokinetics of a single oral dose of CDX-6114 in patients with phenylketonuria (PKU).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 18
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description 2nd Confinement Period in Unit Matching Placebo Randomized to treatment with either CDX-6114 or matching Placebo 1st Confinement Period in Unit CDX 6114 Randomized to treatment with either CDX-6114 or matching Placebo 1st Confinement Period in Unit Matching Placebo Randomized to treatment with either CDX-6114 or matching Placebo 2nd Confinement Period in Unit CDX 6114 Randomized to treatment with either CDX-6114 or matching Placebo
- Primary Outcome Measures
Name Time Method Change in concentration of post parandial plasma level of Phe will be summarized over time for each treatment Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1hours, 1.5, 2,4 and 5hours after dosing on both Day 1 and Day 8 Blood samples will be collected at the following time points to determine the postprandial plasma levels of Phe following single does of CDX-6114
Change in concentration of post parandial plasma level of CA will be summarized over time for each treatment Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1hours, 1.5, 2,4 and 5hours after dosing on both Day 1 and Day 8 Blood samples will be collected at the following time points to determine the postprandial plasma levels of CA following single does of CDX-6114
Change in the peak Phe concentration in Plasma will be summarized by treatment Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8 Blood samples will be collected at the following time points to determine the Peak Phe plasma concentration follwoing a single, oral dose of CDX-6114
Change in the peak CA concentration in Plasma will be summarized by treatment Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8 Blood samples will be collected at the following time points to determine the Peak CA plasma concentration following a single, oral dose of CDX-6114
Phe Area under the plasma concentration versus time curve (AUC) , over a 5-hour period, following dosing and the standardized breakfast Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8 Blood samples will be collected at the following time points to determine the Phe AUC following a single, oral dose of CDX-6114
CA Area under the plasma concentration versus time curve (AUC) , over a 5-hour period, following dosing and the standardized breakfast Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8 Blood samples will be collected at the following time points to determine the CA AUC following a single, oral dose of CDX-6114
- Secondary Outcome Measures
Name Time Method Incidence of Treatment-Emergent Adverse Events (AEs) will be measured Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8 The safety and tolerability of CDX-6114 following single dose oral administration assesed by Adverse events monitoring
The serum levels of CDX-6114 will be summarized descriptively over time Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8 Blood will be collected for pharmacokinetic analysis of CDX-6114 at the following time points follwoing a single oral dose of CDX-6114
Absolute values and changes from baseline in blood pressure measurements will be summarized over time for each treatment Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8. The safety and tolerability of CDX-6114 following single dose oral administration assesed by blood pressure monitoring
Absolute values and changes from baseline in Heart rate measurements will be summarized over time for each treatment Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8. The safety and tolerability of CDX-6114 following single dose oral administration assesed by Heart rate monitoring
Absolute values and changes from baseline in Respiratory rate measurements will be summarized over time for each treatment Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8. The safety and tolerability of CDX-6114 following single dose oral administration assesed by Respiratory rate monitoring
Absolute values and changes from baseline in body temperature (in Fahrenheit or Celsius) measurements will be summarized over time for each treatment Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8. The safety and tolerability of CDX-6114 following single dose oral administration assesed by Body temperature monitoring
Absolute values and changes from baseline in 12 lead Electrocardiogram (ECG) measurements will be summarized over time for each treatment Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8. The safety and tolerability of CDX-6114 following single dose oral administration assesed by 12 lead ECG including P Wave, QRS Complex, QT Interval
Absolute values of Weight measurements will be summarized over time for each treatment using a weighing scale in Kg or pounds Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1hr, 1.5hr, 2hr, 4hr and 5hr after dosing on both Day 1 and Day 8. The safety and tolerability of CDX-6114 following single dose oral administration assesed by weight monitoring
Absolute blood composition values and changes from baseline to the last post-dose time-point will be summarized for each treatment Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8. The safety and tolerability of CDX-6114 following single dose oral administration assesed by laboratory assessments as Haematology and Coagulation
Absolute urine composition values and changes from baseline to the last post-dose time-point will be summarized for each treatment Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8. The safety and tolerability of CDX-6114 following single dose oral administration assesed by routine urinalysis laboratory assessments
Assesment of the incidence of Treatment-Emergent Antibodies Assesments will be done on day 1 and day 8. Blood will be collected for analysis within 30 minutes prior to dosing on Day 1 and Day 8 and again at the End of Study Visit ( through study completion , an avearge of 8 to 10 weeks) The safety and tolerability of CDX-6114 following single dose oral administration assesed by assessment for development of anti-CDX-6114 antibodies
absolute values of height measurements will be summarized over time for each treatment using length measurement scale in centimeters or inches Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1hr, 1.5hr, 2hr, 4hr and 5hr after dosing on both Day 1 and Day 8. The safety and tolerability of CDX-6114 following single dose oral administration assesed by height examination using Lenght scale
Trial Locations
- Locations (2)
Profil Institut für Stoffwechselforschung GmbH
🇩🇪Neuss, Germany
Linear Clinical Research Ltd
🇦🇺Nedlands, Western Australia, Australia