Ignite Creation Date:
2025-12-25 @ 3:46 AM
Ignite Modification Date:
2025-12-26 @ 2:33 AM
Study NCT ID:
NCT00004402
Status:
COMPLETED
Last Update Posted:
2015-03-25
First Post:
1999-10-18
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
Sponsor:
FDA Office of Orphan Products Development
Organization:
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 ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| MUSC-FDR000768 | None | None | View |