Pharmacokinetic Study of Sub-q and IV Treprostinil in Kids With Pulmonary Arterial Hypertension (PAH)
| Status: | Completed |
|---|---|
| Conditions: | High Blood Pressure (Hypertension) |
| Therapuetic Areas: | Cardiology / Vascular Diseases |
| Healthy: | No |
| Age Range: | Any - 18 |
| Updated: | 10/7/2017 |
| Start Date: | October 2013 |
| End Date: | August 1, 2017 |
Multi-center, Open-label Pharmacokinetic Study of Subcutaneously and Intravenously Administered Treprostinil in Children With Pulmonary Arterial Hypertension (PAH)
Abstract
This is a multi-center, open-label pharmacokinetic (PK) study examining the relationship
between the steady-state plasma concentration and dose of treprostinil delivered
intravenously or subcutaneously in children with pulmonary arterial hypertension (PAH).
Subjects will be divided into 5 cohorts by age. A blood sample will be obtained from each
subject at steady state. Additional blood samples will be obtained from a small subset of
subjects with a 15% increase or with at least a 15ng/kg/min increase in dose from steady
state. Samples will be sent to a pharmacokinetic laboratory for analysis. Linear regression
analysis will be used to determine the relationship between the steady state plasma
concentration and drug dose. A power model will be used to assess dose proportionality.
This is a multi-center, open-label pharmacokinetic (PK) study examining the relationship
between the steady-state plasma concentration and dose of treprostinil delivered
intravenously or subcutaneously in children with pulmonary arterial hypertension (PAH).
Subjects will be divided into 5 cohorts by age. A blood sample will be obtained from each
subject at steady state. Additional blood samples will be obtained from a small subset of
subjects with a 15% increase or with at least a 15ng/kg/min increase in dose from steady
state. Samples will be sent to a pharmacokinetic laboratory for analysis. Linear regression
analysis will be used to determine the relationship between the steady state plasma
concentration and drug dose. A power model will be used to assess dose proportionality.
Background Information and Rationale
Treprostinil has not been adequately studied to determine its safety and efficacy in children
≤ 16 years old. However, the drug's use and tolerance in children with PAH has been
demonstrated in studies with small sample sizes.
Although the pharmacokinetic relationship of treprostinil has been established in adult
patients with PAH, the relationship between the steady-state plasma concentration and dose
for children requires further investigation because of physiologic differences, such as the
maturity of enzyme systems and drug clearance mechanisms, between children and adults. The
subjects in this study will be divided into cohorts by age to address the physiologic changes
that occur throughout childhood.
Currently, no data exists demonstrating the relationship between the steady-state plasma
concentration and dose for children treated with intravenously or subcutaneously delivered
treprostinil. Understanding the pharmacokinetics of treprostinil among different age cohorts
in children will provide the data to make an informed recommendation for dosing based on age
(and possibly weight).
Treprostinil has not been adequately studied to determine its safety and efficacy in children
≤ 16 years old. However, the drug's use and tolerance in children with PAH has been
demonstrated in studies with small sample sizes.
Although the pharmacokinetic relationship of treprostinil has been established in adult
patients with PAH, the relationship between the steady-state plasma concentration and dose
for children requires further investigation because of physiologic differences, such as the
maturity of enzyme systems and drug clearance mechanisms, between children and adults. The
subjects in this study will be divided into cohorts by age to address the physiologic changes
that occur throughout childhood.
Currently, no data exists demonstrating the relationship between the steady-state plasma
concentration and dose for children treated with intravenously or subcutaneously delivered
treprostinil. Understanding the pharmacokinetics of treprostinil among different age cohorts
in children will provide the data to make an informed recommendation for dosing based on age
(and possibly weight).
Inclusion Criteria:
1. Patients must be on continuous intravenous or subcutaneous treprostinil for the
treatment of pulmonary arterial hypertension, defined as mean pulmonary artery
pressure >25mmHg at rest with a PVRi > 3 Wood units.
2. Patients must be between the ages of 0 to 16 years at the time of study enrollment.
3. Written informed consent and assent, when applicable, must be completed.
Exclusion Criteria:
1. Patients with severe liver or renal diseases.
2. Female patients who may be pregnant or breastfeeding
3. Written informed consent and assent not completed due to patient and/or parent or
legal guardian unwilling to participate.
4. Patients on concomitant use of a CYP2C inhibitor or inducer.
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