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Posaconazole to Treat Invasive Fungal Infections

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00033982
Recruitment Status : Completed
First Posted : April 19, 2002
Last Update Posted : July 2, 2017
Information provided by:
National Institutes of Health Clinical Center (CC)

Brief 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. 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.

Condition or disease Intervention/treatment Phase
Granulomatous Disease, Chronic Job's Syndrome Infection Drug: Posaconazole Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 50 participants
Primary Purpose: Treatment
Official Title: Open Label, Limited Access Protocol of Posaconazole in Invasive Fungal Infections
Study Start Date : April 11, 2002
Study Completion Date : February 20, 2007

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.


As set out in the company protocol.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00033982

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United States, New Jersey
Schering-Plough Research Institute
Kenilworth, New Jersey, United States, 07033
Sponsors and Collaborators
National Institute of Allergy and Infectious Diseases (NIAID)

Layout table for additonal information Identifier: NCT00033982     History of Changes
Other Study ID Numbers: 020171
First Posted: April 19, 2002    Key Record Dates
Last Update Posted: July 2, 2017
Last Verified: February 20, 2007

Keywords provided by National Institutes of Health Clinical Center (CC):
Fungal Infection

Additional relevant MeSH terms:
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Communicable Diseases
Chronic Disease
Invasive Fungal Infections
Job Syndrome
Granulomatous Disease, Chronic
Disease Attributes
Pathologic Processes
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Antifungal Agents
Anti-Infective Agents
Trypanocidal Agents
Antiprotozoal Agents
Antiparasitic Agents
14-alpha Demethylase Inhibitors
Cytochrome P-450 Enzyme Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Steroid Synthesis Inhibitors
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs