Ignite Creation Date:
2024-05-05 @ 9:42 PM
Last Modification Date:
2024-10-26 @ 10:08 AM
Study NCT ID:
NCT00947544
Status:
COMPLETED
Last Update Posted:
2024-07-11
First Post:
2009-07-24
Brief Title:
Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
Sponsor:
Amgen
Organization:
Amgen
Study Overview
Official Title:
A Phase 2 Fixed-Sequence Open-Label Switch-Over Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children 6-17 Years of Age With Urea Cycle Disorders With a Long-Term Safety Extension
Status:
COMPLETED
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Protocol HPN-100-005 was the first study of HPN-100 in pediatric subjects with urea cycle disorders UCDs and was a fixed-sequence open-label switch over study of HPN-100 with a long-term 12 month safety extension designed to assess the safety of HPN-100 and to prospectively assess its ability to control blood ammonia as compared with Sodium Phenylbutyrate NaPBA Upon DSMB review of the first ten subjects who completed the switch over part of the study and with DSMB approval up to an additional 20 subjects were enrolled into the safety extension part of the study HPN-100 is a triglyceride that has a similar mechanism of action as NaPBA It is a liquid with minimal taste and odor Three teaspoons of HPN-100 174mL delivers an equivalent amount of PBA to 40 tablets of NaPBA
Detailed Description:
This was a fixed-sequence open-label switch over study of HPN-100 with a long-term 12 month safety extension part designed to assess the safety of HPN-100 in pediatric subjects and to prospectively assess the ability of HPN-100 to control blood ammonia compared with NaPBA
For those subjects who participated in the switch over NaPBA was dosed three times daily TID with meals during the first week and the same PBA mole-equivalent dose of HPN-100 during the second week If there were safety concerns regarding a single-step transition from NaPBA to HPN-100 at the investigators discretion the transition could occur in 2 steps such that in the second week subjects might receive 50 of the PBA equivalent dose as NaPBA and 50 as HPN-100 before receiving 100 of the PBA equivalent dose as HPN-100 in the third week Serial blood samples were collected for PK and blood ammonia assessments after each drug reached steady state which was achieved approximately 4 days after initiation of 100 NaPBA or HPN-100 treatment
The subjects who completed the switch over part of the study and up to 20 additional subjects were offered the opportunity to continue in the study by entering the safety extension part of the study to continue receiving open-label HPN-100 for up to 12 months
Subjects who prematurely terminated the study during the switch-over period after enrollment had safety assessments including safety labs and a single blood sample drawn for measurement of phenylbutyrate PBA the active metabolite phenylacetate PAA and the terminal metabolite phenylacetylglutamine PAGN Subjects who had enrolled in the safety extension period of the study either directly or following the switch over but prematurely terminated the study prior to completing the extension period had Month 12 procedures performed or at a minimum had safety assessments including safety labs and ammonia had drawn The time of day at which the blood sample was drawn was recorded as well as the time since the last dose of medication was taken
Subjects followed a stable diet throughout the study as prescribed by the investigator and dietary compliance was recorded at each study visit for both the switch over and safety extension parts of the study
Study acquired from Horizon in 2024
For those subjects who participated in the switch over NaPBA was dosed three times daily TID with meals during the first week and the same PBA mole-equivalent dose of HPN-100 during the second week If there were safety concerns regarding a single-step transition from NaPBA to HPN-100 at the investigators discretion the transition could occur in 2 steps such that in the second week subjects might receive 50 of the PBA equivalent dose as NaPBA and 50 as HPN-100 before receiving 100 of the PBA equivalent dose as HPN-100 in the third week Serial blood samples were collected for PK and blood ammonia assessments after each drug reached steady state which was achieved approximately 4 days after initiation of 100 NaPBA or HPN-100 treatment
The subjects who completed the switch over part of the study and up to 20 additional subjects were offered the opportunity to continue in the study by entering the safety extension part of the study to continue receiving open-label HPN-100 for up to 12 months
Subjects who prematurely terminated the study during the switch-over period after enrollment had safety assessments including safety labs and a single blood sample drawn for measurement of phenylbutyrate PBA the active metabolite phenylacetate PAA and the terminal metabolite phenylacetylglutamine PAGN Subjects who had enrolled in the safety extension period of the study either directly or following the switch over but prematurely terminated the study prior to completing the extension period had Month 12 procedures performed or at a minimum had safety assessments including safety labs and ammonia had drawn The time of day at which the blood sample was drawn was recorded as well as the time since the last dose of medication was taken
Subjects followed a stable diet throughout the study as prescribed by the investigator and dietary compliance was recorded at each study visit for both the switch over and safety extension parts of the study
Study acquired from Horizon in 2024
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None