Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

6 participants

Study Classification

OBSERVATIONAL

Study Start Date

2002-01-31

Study Completion Date

2005-09-30

Brief Summary

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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.

Detailed Description

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It is made by white blood cells and appears to be involved in regulating the body's ability to fight off infection. Actimmune is a synthetic form of Interferon gamma which is similar to that normally made by white blood cells.

IFN-g 1b (Actimmune®) is currently approved by the United States Food and Drug Administration (FDA) for the treatment of patients with chronic granulomatous disease (CGD) to reduce the frequency and severity of serious infections. It is also approved in patients with severe, malignant osteopetrosis to delay the time to disease progression. In research trials, IFN-g 1b has been given to over 2,000 patients in diseases such as CGD, osteopetrosis, atopic dermatitis, pulmonary fibrosis, atypical mycobacteria and various cancers.

Conditions

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Osteopetrosis

Keywords

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Registry Actimmune osteopetrosis

Study Design

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Study Time Perspective

RETROSPECTIVE

Interventions

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Actimmune Registry

as administered by physician

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Principal Investigators

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Steven Porter, MD

Role: STUDY_DIRECTOR

InterMune

Locations

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InterMune, Inc.

Brisbane, California, United States

Site Status

Countries

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United States

Other Identifiers

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GIOS-003

Identifier Type: -

Identifier Source: org_study_id