Ignite Creation Date:
2024-05-05 @ 10:17 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004402
Status:
COMPLETED
Last Update Posted:
2015-03-25
First Post:
1999-10-18
Brief Title:
Phase III Randomized Study of Interferon Gamma in Children With Severe Congenital Osteopetrosis
Sponsor:
FDA Office of Orphan Products Development
Organization:
FDA Office of Orphan Products Development
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2001-01
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:
OBJECTIVES I Compare the rate of treatment failure in osteopetrosis patients receiving interferon gamma in combination with calcitriol to the rate of treatment failure in patients receiving calcitriol alone
II Compare the number of adverse events or clinical manifestations of disease progression occurring in these patients
III Assess the effects of interferon gamma on hematopoiesis cranial nerve function and rate of infection in these patients
II Compare the number of adverse events or clinical manifestations of disease progression occurring in these patients
III Assess the effects of interferon gamma on hematopoiesis cranial nerve function and rate of infection in these patients
Detailed Description:
PROTOCOL OUTLINE This is a randomized placebo controlled open label study Patients are randomized to one of two arms interferon gamma in combination with calcitriol or calcitriol alone
Arm I Patients receive calcitriol once daily Interferon gamma is administered by subcutaneous injection three times a week
Arm II Patients receive calcitriol once daily Patients may continue treatment in the absence of toxicity and disease progression If disease progression is diagnosed in the control group patients will then receive interferon gamma in combination with calcitriol
Patients are followed every 4 weeks
Arm I Patients receive calcitriol once daily Interferon gamma is administered by subcutaneous injection three times a week
Arm II Patients receive calcitriol once daily Patients may continue treatment in the absence of toxicity and disease progression If disease progression is diagnosed in the control group patients will then receive interferon gamma in combination with calcitriol
Patients are followed every 4 weeks
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| MUSC-FDR000768 | None | None | None |