A Phase 1, Multi-center, Randomized, Double-blind, Placebo-controlled, Cross-over Study to Evaluate the Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of a Single Oral Dose of CDX-6114 in Patients With Phenylketonuria (PKU).
Overview
- Phase
- Phase 1
- Intervention
- CDX 6114
- Conditions
- Phenylketonuria
- Sponsor
- Société des Produits Nestlé (SPN)
- Enrollment
- 18
- Locations
- 2
- Primary Endpoint
- Change in concentration of post parandial plasma level of Phe will be summarized over time for each treatment
- Status
- Completed
- Last Updated
- 5 years ago
Overview
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).
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
1st Confinement Period in Unit
Randomized to treatment with either CDX-6114 or matching Placebo
Intervention: CDX 6114
1st Confinement Period in Unit
Randomized to treatment with either CDX-6114 or matching Placebo
Intervention: Matching Placebo
2nd Confinement Period in Unit
Randomized to treatment with either CDX-6114 or matching Placebo
Intervention: CDX 6114
2nd Confinement Period in Unit
Randomized to treatment with either CDX-6114 or matching Placebo
Intervention: Matching Placebo
Outcomes
Primary Outcomes
Change in concentration of post parandial plasma level of Phe will be summarized over time for each treatment
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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 Outcomes
- 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 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)
- 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.)
- 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.)
- 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.)
- 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.)
- 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.)
- 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.)
- 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.)
- 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.)
- 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))
- 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.)