Granulomatous Disease, ChronicPhase 3NCT00033982
Posaconazole to Treat Invasive Fungal Infections
A Phase 3 study testing Posaconazole in people with Granulomatous Disease, Chronic and Job's Syndrome, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial summary
This study will evaluate the safety and effectiveness of posaconazole for treating invasive fungal infections. New therapies for these infections are needed for patients who do not respond, to or cannot tolerate, standard treatment. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Granulomatous Disease, Chronic, Job's Syndrome, Infection
- What is being tested?
- Posaconazole
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Apr 2002to 20 Feb 2007
- Phase
- Phase 3
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- As set out in the company protocol.
- All subjects must have a proven or probable fungal infection that is resistant to standard therapy, or to which therapy the patient is significantly intolerant. (Intolerance must include signs of intolerance such as fever, rigors, laboratory, abnormalities, as well as subjective symptoms).
- Only subjects age 2 years and older will be eligible at this time.
- Pediatric Inclusion:
- Children age 2 and over are eligible. Children under 2 years are not currently eligible because of lack of any data on dose and tolerance.
Who cannot join
- As set out in the company protocol.
Trial locations and contacts
1 site in 1 country.
United States1 site
Schering-Plough Research Institute
Kenilworth, New Jersey, 07033
About this study
Official title: Open Label, Limited Access Protocol of Posaconazole in Invasive Fungal Infections
This study will evaluate the safety and effectiveness of posaconazole for treating invasive fungal infections. New therapies for these infections are needed for patients who do not respond, to or cannot tolerate, standard treatment. These patients include those with immune defects who have significant side effects from treatment with amphotericin or other antifungals.
Patients 13 years of age or older who are on other primary NIH protocols with an invasive fungal infection 1) that does not respond to standard antifungal therapies; 2) for which there is no effective therapy; 3) who develop serious side effects from their current treatment; or 4) who have organ dysfunction that does not permit use of standard antifungal treatments may be eligible for this study. Candidates will be screened with a medical history, including a review of current and previous antifungal treatments, pregnancy test for women of childbearing potential, electrocardiogram (EKG), and detailed neurologic examination.
Read more
Participants will take either 200 mg (1 teaspoonful) of liquid posaconazole by mouth four times a day or 400 mg (two teaspoonfuls) twice a day for a period of 28 days to 24 months. (The physician will determine the duration of treatment.) Patients will have monthly follow-up visits during the treatment period and 1 month after treatment is completed for the following procedures:
- Detailed neurologic exam every 3 months
- Blood tests every month
- EKG every month
- Imaging studies, including chest x-ray, computed tomography (CT), magnetic resonance imaging (MRI) radionuclide scanning or ultrasound, every month until the infection has been stable for three determinations. Thereafter, imaging studies will be done every 3 months as long as the infection remains stable or improves.
On the last day of the study treatment period, participants will have a detailed neurologic exam and review of medications and medical complaints since their last visit.
Read the detailed description
We seek to use the experimental triazole antifungal, Posaconazole, in the treatment of patients with invasive fungal infections that are resistant or refractory to the best available treatment, or who are unable to tolerate the best available treatment. We have observed significant short and long term toxicities from amphotericin and its various preparations in our patients with chronic granulomatous disease (CGD) and Hyper IgE recurrent infection syndrome (HIE or Job's). We now have now had 5 CGD patients on dialysis, and several more have progressive renal failure, all thought due to high dose, prolonged amphotericin. In addition, many of our more severely affected CGD patients have had multiple thoracic surgeries, leading to reduced lung capacity. Several of our HIE patients have persistent fungal lung infections that have not cleared with extensive therapy with amphotericin and/or other new triazole antifungals. Therefore, agents of high tolerability and high potency are still desperately needed. Posaconazole has a broad spectrum of action against a large number of molds and fungi at easily achievable concentrations in plasma. It has a favorable toxicity profile and has performed well in clinical trials to date. This drug will be administered orally twice daily. Close monitoring of tolerance, toxicity, and efficacy will be performed. We anticipate enrollment of up to 50 patients over the next 2 years.
- Conditions
- Granulomatous Disease, Chronic, Job's Syndrome, Infection
- Being tested
- Posaconazole
- Keywords
- Triazole, Refractory, Antifungal, Fungal Infection
- Medical subject headings
- Granulomatous Disease, Chronic, Job Syndrome, Infections, Mycoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PosaconazoleDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2002 (estimated)
- Study start11 Apr 2002
- Study completion20 Feb 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Granulomatous Disease, ChronicStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Enrolment
- 50 participants
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00033982
- Sponsor's protocol ID
- 020171
- Other IDs
- 02-I-0171
- Status last verified
- 20 Feb 2007
- First submitted
- 18 Apr 2002
- Last updated
- 2 Jul 2017
- Results posted
- No
Publications
Nguyen MH, Peacock JE Jr, Morris AJ, Tanner DC, Nguyen ML, Snydman DR, Wagener MM, Rinaldi MG, Yu VL. The changing face of candidemia: emergence of non-Candida albicans species and antifungal resistance. Am J Med. 1996 Jun;100(6):617-23. doi: 10.1016/s0002-9343(95)00010-0.
PubMed 8678081
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label, Limited Access Protocol of Posaconazole in Invasive Fungal Infections
- ClinicalTrials.gov ID
- NCT00033982
- Acronym / sponsor ID
- 020171
- Conditions
- Granulomatous Disease, Chronic, Job's Syndrome, Infection
- Keywords
- Triazole, Refractory, Antifungal, Fungal Infection
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- 11 Apr 2002
- Study completion
- 20 Feb 2007
- First posted
- 19 Apr 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.
This study will evaluate the safety and effectiveness of posaconazole for treating invasive fungal infections. New therapies for these infections are needed for patients who do not respond, to or cannot tolerate, standard treatment. These patients include those with immune defects who have significant side effects from treatment with amphotericin or other antifungals.
Patients 13 years of age or older who are on other primary NIH protocols with an invasive fungal infection 1) that does not respond to standard antifungal therapies; 2) for which there is no effective therapy; 3) who develop serious side effects from their current treatment; or 4) who have organ dysfunction that does not permit use of standard antifungal treatments may be eligible for this study. Candidates will be screened with a medical history, including a review of current and previous antifungal treatments, pregnancy test for women of childbearing potential, electrocardiogram (EKG), and detailed neurologic examination.
Participants will take either 200 mg (1 teaspoonful) of liquid posaconazole by mouth four times a day or 400 mg (two teaspoonfuls) twice a day for a period of 28 days to 24 months. (The physician will determine the duration of treatment.) Patients will have monthly follow-up visits during the treatment period and 1 month after treatment is completed for the following procedures:
- Detailed neurologic exam every 3 months
- Blood tests every month
- EKG every month
- Imaging studies, including chest x-ray, computed tomography (CT), magnetic resonance imaging (MRI) radionuclide scanning or ultrasound, every month until the infection has been stable for three determinations. Thereafter, imaging studies will be done every 3 months as long as the infection remains stable or improves.
On the last day of the study treatment period, participants will have a detailed neurologic exam and review of medications and medical complaints since their last visit.
Detailed description
We seek to use the experimental triazole antifungal, Posaconazole, in the treatment of patients with invasive fungal infections that are resistant or refractory to the best available treatment, or who are unable to tolerate the best available treatment. We have observed significant short and long term toxicities from amphotericin and its various preparations in our patients with chronic granulomatous disease (CGD) and Hyper IgE recurrent infection syndrome (HIE or Job's). We now have now had 5 CGD patients on dialysis, and several more have progressive renal failure, all thought due to high dose, prolonged amphotericin. In addition, many of our more severely affected CGD patients have had multiple thoracic surgeries, leading to reduced lung capacity. Several of our HIE patients have persistent fungal lung infections that have not cleared with extensive therapy with amphotericin and/or other new triazole antifungals. Therefore, agents of high tolerability and high potency are still desperately needed. Posaconazole has a broad spectrum of action against a large number of molds and fungi at easily achievable concentrations in plasma. It has a favorable toxicity profile and has performed well in clinical trials to date. This drug will be administered orally twice daily. Close monitoring of tolerance, toxicity, and efficacy will be performed. We anticipate enrollment of up to 50 patients over the next 2 years.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- As set out in the company protocol.
- All subjects must have a proven or probable fungal infection that is resistant to standard therapy, or to which therapy the patient is significantly intolerant. (Intolerance must include signs of intolerance such as fever, rigors, laboratory, abnormalities, as well as subjective symptoms).
- Only subjects age 2 years and older will be eligible at this time.
- Children age 2 and over are eligible. Children under 2 years are not currently eligible because of lack of any data on dose and tolerance.
Key exclusion criteria (1 listed)
- As set out in the company protocol.
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 |
|---|---|---|
| Posaconazole | 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 2 Jul 2017.
- Background: PubMed 8678081Nguyen MH, Peacock JE Jr, Morris AJ, Tanner DC, Nguyen ML, Snydman DR, Wagener MM, Rinaldi MG, Yu VL. The changing face of candidemia: emergence of non-Candida albicans species and antifungal resistance. Am J Med. 1996 Jun;100(6):617-23. doi: 10.1016/s0002-9343(95)00010-0.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Posaconazole in people with Granulomatous Disease, Chronic and Job's Syndrome, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Jul 2017. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
On the ClinicalTrials.gov record for NCT00033982, which also lists 1 linked publication.
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 2 Jul 2017.