Ignite Creation Date:
2024-05-05 @ 9:40 PM
Last Modification Date:
2024-10-26 @ 10:08 AM
Study NCT ID:
NCT00947297
Status:
COMPLETED
Last Update Posted:
2024-07-10
First Post:
2009-07-24
Brief Title:
Study of the Safety of HPN Hyperion-100 for the Long-Term Treatment of Urea Cycle Disorders Treat UCD
Sponsor:
Amgen
Organization:
Amgen
Study Overview
Official Title:
A Phase 3 Open-Label Study of the Safety of HPN-100 for the Long-Term Treatment of Urea Cycle Disorders Treat UCD
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:
This was a long-term safety study HPN-100 in urea cycle disorder UCD subjects Subjects were assessed regularly for safety and control of their venous ammonia Hyperammonemic events were characterized with respect to contributing factors such as intercurrent illness diet and noncompliance with medication
Detailed Description:
This was a one year long-term safety study of HPN-100 in UCD subjects Subjects were assessed regularly for safety and control of their venous ammonia Hyperammonemic events were characterized with respect to contributing factors such as intercurrent illness diet and noncompliance with medication
Forty subjects with a diagnosis of UCD who completed Study HPN-100-006 were enrolled
Twenty additional UCD subjects 6 years of age were enrolled These subjects included those who did not qualify for HPN-100-006 eg subjects between the ages of 6-17 subjects with other UCD subtypes or adult subjects who have not taken sodium phenylbutyrate NaPBA in the past 6 months etc For adult subjects not receiving NaPBA in the past 6 months subjects must in the judgment of the investigator be anticipated to benefit from the addition of a nitrogen-scavenging agent to their current treatment See the inclusion criteria for examples of clinical evidence of potential benefit
Monthly assessments included safety laboratory tests amino acid panel vital signs electrocardiogram ECG monitoring venous ammonia and blood and urine metabolites Adverse events AEs and concomitant medications were recorded on an ongoing basis
Study acquired from Horizon in 2024
Forty subjects with a diagnosis of UCD who completed Study HPN-100-006 were enrolled
Twenty additional UCD subjects 6 years of age were enrolled These subjects included those who did not qualify for HPN-100-006 eg subjects between the ages of 6-17 subjects with other UCD subtypes or adult subjects who have not taken sodium phenylbutyrate NaPBA in the past 6 months etc For adult subjects not receiving NaPBA in the past 6 months subjects must in the judgment of the investigator be anticipated to benefit from the addition of a nitrogen-scavenging agent to their current treatment See the inclusion criteria for examples of clinical evidence of potential benefit
Monthly assessments included safety laboratory tests amino acid panel vital signs electrocardiogram ECG monitoring venous ammonia and blood and urine metabolites Adverse events AEs and concomitant medications were recorded on an ongoing basis
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