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

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.

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Key information

Conditions

Osteopetrosis Bone Diseases Bone Diseases, Developmental Musculoskeletal Diseases Osteochondrodysplasias Osteosclerosis

Sex eligibility

All sexes

Study type

Observational

Primary location

InterMune, Inc.

Brisbane, California, 94005, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Actimmune Registry

Drug

as administered by physician

Sponsors and collaborators

Lead sponsor

InterMune

Industry

Registry information

Official study title

Post-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) in Patients With Severe Malignant Osteopetrosis

Important dates

Study start: 2002
Study completion: 2005
First posted: Aug 20, 2002
Registry last updated: Nov 1, 2007

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed