Posaconazole to Treat Invasive Fungal Infections
| Status: | Completed |
|---|---|
| Conditions: | Infectious Disease, Infectious Disease |
| Therapuetic Areas: | Immunology / Infectious Diseases |
| Healthy: | No |
| Age Range: | 2 - Any |
| Updated: | 4/21/2016 |
| Start Date: | April 2002 |
| End Date: | February 2007 |
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.
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.
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.
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.
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.
- INCLUSION CRITERIA:
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.
EXCLUSION CRITERIA:
As set out in the company protocol.
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