NCT00043329

Brief Summary

The purpose of this study is to establish a registry of all children with severe, malignant osteopetrosis who are treated with Actimmune (IFN-g 1b or Interferon gamma-1b) to monitor the effects of IFN-g 1b on preventing progression of this disease and to follow the safety of patients receiving it on a long-term basis. In addition, evaluation of the possible effect of Actimmune therapy on the humoral response to normal childhood vaccinations in this same patient population will be examined.Interferon gamma is a substance that the body makes naturally.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jan 2002

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2002

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

August 7, 2002

Completed
13 days until next milestone

First Posted

Study publicly available on registry

August 20, 2002

Completed
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2005

Completed
Last Updated

November 1, 2007

Status Verified

October 1, 2007

First QC Date

August 7, 2002

Last Update Submit

October 30, 2007

Conditions

Osteopetrosis

Keywords

RegistryActimmuneosteopetrosis

Interventions

Actimmune RegistryDRUG

as administered by physician

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Osteopetrosis patients receiving Actimmune therapy

* Male or female * Diagnosis of severe, malignant osteopetrosis * Currently receiving or planning to initiate therapy with Actimmune (Interferon gamma-1b) * Willing to attend follow-up appointments every 6 months following enrollment into the study, if clinically indicated

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

InterMune, Inc.

Brisbane, California, 94005, United States

Location

MeSH Terms

Conditions

Osteopetrosis

Condition Hierarchy (Ancestors)

OsteosclerosisOsteochondrodysplasiasBone Diseases, DevelopmentalBone DiseasesMusculoskeletal Diseases

Study Officials

  • Steven Porter, MD

    InterMune

    STUDY DIRECTOR

Study Design

Study Type
observational
Time Perspective
RETROSPECTIVE
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

August 7, 2002

First Posted

August 20, 2002

Study Start

January 1, 2002

Study Completion

September 1, 2005

Last Updated

November 1, 2007

Record last verified: 2007-10

Locations

US(1)