OsteopetrosisNCT00043329
Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis
An observational study of Osteopetrosis, run by InterMune at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Osteopetrosis
- What is being tested?
- Actimmune Registry
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2002to Sep 2005
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Post-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) 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.
Read the detailed description
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
- Osteopetrosis
- Being tested
- Actimmune Registry
- Keywords
- Registry, Actimmune, osteopetrosis
- Medical subject headings
- Osteopetrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Actimmune RegistryDrug
as administered by physician
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2002 (estimated)
- Study startJan 2002
- Study completionSep 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for OsteopetrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Time perspective
- Retrospective
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- InterMune
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Steven Porter, MD, Study director, InterMune
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00043329
- Sponsor's protocol ID
- GIOS-003
- Status last verified
- Oct 2007
- First submitted
- 7 Aug 2002
- Last updated
- 1 Nov 2007
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Nov 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) in Patients With Severe Malignant Osteopetrosis
- ClinicalTrials.gov ID
- NCT00043329
- Acronym / sponsor ID
- GIOS-003
- Conditions
- Osteopetrosis
- Keywords
- Registry, Actimmune, osteopetrosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- InterMuneIndustry (pharma, biotech, devices)
- Study start
- Jan 2002
- Study completion
- Sep 2005 (actual)
- First posted
- 20 Aug 2002 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Osteopetrosis patients receiving Actimmune therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Actimmune Registryas administered by physician | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Nov 2007.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven Porter, MD study director
InterMune
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Osteopetrosis, run by InterMune at 1 site in United States.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Nov 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Nov 2007.