ImmunocompromisedPhase 1MicafunginNCT02057289
A Pharmacokinetic Study of Pediatric Micafungin Prophylaxis
A Phase 1 study testing Micafungin in people with Immunocompromised and Bone Marrow Transplant, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The purpose of this research study is to examine the pharmacokinetics (the process by which a drug is absorbed, distributed, metabolized, and eliminated by the body) of micafungin when it is given at 5mg/kg dose to immunocompromised children as anti-fungal prophylaxis. Read more about this study
- Who is it for?
- Ages 6 months to 10 years
- Conditions the study is for
- Immunocompromised, Bone Marrow Transplant, Cancer
- What is being tested?
- Micafungin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2014to Nov 2014
- Phase
- Phase 1
- 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 6 months to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients who are at risk for fungal infection and require prophylaxis. Example: patients undergoing blood and marrow transplant, immunodeficiency patients, patients with aplastic anemia.
- Age >= 6 months to <= 10 years (at time of enrollment).
- Patients with adequate organ function (documented within 2 weeks prior to start of micafungin):
- Creatinine < 2 times upper limit normal
- Total bilirubin and AST < 3 times upper limit normal
Who cannot join
- Patients who have history of past or evidence of active fungal disease (by either radiological studies or biopsy proven) or are being treated for presumed fungal infection.
- Patients who have history of allergy to micafungin or other echinocandin preparations, such as Caspofungin or Anidulafungin.
- Patients who have received micafungin or other echinocandin preparations in the previous two weeks.
- Patients receiving antifungal prophylaxis other than Fluconazole at the time of enrollment. This is due to the fact that during transplant, Fluconazole is usually switched to agents with better coverage. This will avoid the possibility of reducing effective antifungal coverage for the purpose of the study.
- Failure to sign informed consent, or inability to undergo informed consent process.
- It is not medically advisable to obtain the specimens necessary for this study.
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: Micafungin Anti-Fungal Prophylaxis in Immunocompromised Pediatric Patients: A Pharmacokinetic Study
The purpose of this research study is to examine the pharmacokinetics (the process by which a drug is absorbed, distributed, metabolized, and eliminated by the body) of micafungin when it is given at 5mg/kg dose to immunocompromised children as anti-fungal prophylaxis. These children are at high risk for developing invasive fungal disease due to their compromised immunity and associated variable degree and duration of neutropenia. Currently, children who receive micafungin are given daily or alternate day dosing. The investigators will give a ONE TIME dose of micafungin and draw PK levels up to 96 hours post-infusion. The investigators goal is to obtain comparable micafungin drug concentrations at the end of 96 hours (4 days) as compared to lower dose at every 24 hour dosing. The investigators dosing proposal is likely to be effective prophylaxis for immunocompromised patients and would broaden its applicability to larger populations.
Read the detailed description
Disseminated fungal infection is a major cause of morbidity and mortality in immunocompromised children. Many of the drugs used for fungal prophylaxis have been associated with renal and hepatic toxicity. Also, breakthrough infections have been reported with the use of some of the oral agents due to poor oral absorption. An alternative approach is the use of intravenous micafungin for fungal prophylaxis. Micafungin has a distinct advantage due to its better safety profile, specifically in terms of hepatic and renal toxicity. Currently, children who receive micafungin are given daily or alternate day dosing (based on their last Pk study, Mehta et al 2010). The investigators objective is to examine the pharmacokinetics of micafungin when it is given on a less frequent schedule. The investigators hypothesize that a single dose of micafungin (at 5mg/kg) every 4 days will provide drug exposure equivalent to daily dosing (at 1mg/kg) while reducing administration costs and improving patient convenience (with essentially twice a week dosing regimen). Both animal and adult data support the use of this approach. Fifteen patients will be enrolled on this study and will be given a SINGLE DOSE of micafungin antifungal prophylaxis (5 mg/kg). Blood samples will be drawn for pharmacokinetic measurements after administration of micafungin. Plasma concentration data will be analyzed by compartmental and non-compartmental pharmacokinetic analysis. The data will also be analyzed by a population pharmacokinetic approach.
- Conditions
- Immunocompromised, Bone Marrow Transplant, Cancer
- Being tested
- Micafungin
- Keywords
- pediatric, antifungal, bone marrow, AML or relapsed ALL
- Medical subject headings
- Neoplasms, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MicafunginDrug
Also called Mycamine
Micafungin (trade name Mycamine) is an echinocandin antifungal drug developed by Astellas Pharma. It inhibits the production of beta-1,3-glucan, an essential component of fungal cell walls. Micafungin is administered intravenously. It received final approval from the U.S. Food and Drug Administration on March 16, 2005, and gained approval in the European Union on April 25, 2008.
| Study group | Role | What they receive |
|---|---|---|
| Micafungin | Experimental | Subjects will be administered 5 mg/kg of micafungin intravenously as a ONE TIME dose.
For patients undergoing Hematopoietic Stem Cell Transplant (HSCT), Micafungin will be given on during rest days (i.e. days when no chemotherapy is administered) and blood for pharmacokinetic measurements will be drawn over next 96 hours.
Following this, further anti-fungal coverage will be at the discretion of the patient's attending physician. (I.e. other antifungal agent(s) or Micafungin at a standard clinical dose; repeat doses of 5mg/kg will NOT be administered.) Receives: Drug: Micafungin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetics to measure the levels of micafungin
Blood samples from the above time points will be analyzed to study the bodily absorption, distribution, metabolism, and excretion of micafungin
Measured: Prior to the micafungin infusion at hour 0, at the end of the infusion (60 min), then at 11/2, 2, 4, 6, 8, 10, 24, 36, 48, 60, 72, 84 and 96 hours after the start of the micafungin infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2014 (estimated)
- Study startJan 2014
- Primary completionNov 2014
- Study completionNov 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ImmunocompromisedStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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 1
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Astellas Pharma Inc
- Responsible party
- Sponsor
- Investigators
- Parinda Mehta, MD, Principal investigator, CCHMC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02057289
- Sponsor's protocol ID
- 2013-4868
- Acronym
- Micafungin
- Status last verified
- May 2018
- First submitted
- 29 Jan 2014
- Last updated
- 23 May 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Micafungin Anti-Fungal Prophylaxis in Immunocompromised Pediatric Patients: A Pharmacokinetic Study
- ClinicalTrials.gov ID
- NCT02057289
- Acronym / sponsor ID
- Micafungin / 2013-4868
- Conditions
- Immunocompromised, Bone Marrow Transplant, Cancer
- Keywords
- pediatric, antifungal, bone marrow, AML or relapsed ALL
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Astellas Pharma Inc
- Study start
- Jan 2014
- Primary completion
- Nov 2014 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 7 Feb 2014 (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 research study is to examine the pharmacokinetics (the process by which a drug is absorbed, distributed, metabolized, and eliminated by the body) of micafungin when it is given at 5mg/kg dose to immunocompromised children as anti-fungal prophylaxis. These children are at high risk for developing invasive fungal disease due to their compromised immunity and associated variable degree and duration of neutropenia. Currently, children who receive micafungin are given daily or alternate day dosing. The investigators will give a ONE TIME dose of micafungin and draw PK levels up to 96 hours post-infusion. The investigators goal is to obtain comparable micafungin drug concentrations at the end of 96 hours (4 days) as compared to lower dose at every 24 hour dosing. The investigators dosing proposal is likely to be effective prophylaxis for immunocompromised patients and would broaden its applicability to larger populations.
Detailed description
Disseminated fungal infection is a major cause of morbidity and mortality in immunocompromised children. Many of the drugs used for fungal prophylaxis have been associated with renal and hepatic toxicity. Also, breakthrough infections have been reported with the use of some of the oral agents due to poor oral absorption. An alternative approach is the use of intravenous micafungin for fungal prophylaxis. Micafungin has a distinct advantage due to its better safety profile, specifically in terms of hepatic and renal toxicity. Currently, children who receive micafungin are given daily or alternate day dosing (based on their last Pk study, Mehta et al 2010). The investigators objective is to examine the pharmacokinetics of micafungin when it is given on a less frequent schedule. The investigators hypothesize that a single dose of micafungin (at 5mg/kg) every 4 days will provide drug exposure equivalent to daily dosing (at 1mg/kg) while reducing administration costs and improving patient convenience (with essentially twice a week dosing regimen). Both animal and adult data support the use of this approach. Fifteen patients will be enrolled on this study and will be given a SINGLE DOSE of micafungin antifungal prophylaxis (5 mg/kg). Blood samples will be drawn for pharmacokinetic measurements after administration of micafungin. Plasma concentration data will be analyzed by compartmental and non-compartmental pharmacokinetic analysis. The data will also be analyzed by a population pharmacokinetic approach.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Micafungin | Experimental | Drug: MicafunginSubjects will be administered 5 mg/kg of micafungin intravenously as a ONE TIME dose. For patients undergoing Hematopoietic Stem Cell Transplant (HSCT), Micafungin will be given on during rest days (i.e. days when no chemotherapy is administered) and blood for pharmacokinetic measurements will be drawn over next 96 hours. Following this, further anti-fungal coverage will be at the discretion of the patient's attending physician. (I.e. other antifungal agent(s) or Micafungin at a standard clinical dose; repeat doses of 5mg/kg will NOT be administered.) |
Who may qualify
- Ages
- 6 months to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Patients who are at risk for fungal infection and require prophylaxis. Example: patients undergoing blood and marrow transplant, immunodeficiency patients, patients with aplastic anemia.
- Age >= 6 months to <= 10 years (at time of enrollment).
- Patients with adequate organ function (documented within 2 weeks prior to start of micafungin):
Key exclusion criteria (6 listed)
- Patients who have history of past or evidence of active fungal disease (by either radiological studies or biopsy proven) or are being treated for presumed fungal infection.
- Patients who have history of allergy to micafungin or other echinocandin preparations, such as Caspofungin or Anidulafungin.
- Patients who have received micafungin or other echinocandin preparations in the previous two weeks.
- Patients receiving antifungal prophylaxis other than Fluconazole at the time of enrollment. This is due to the fact that during transplant, Fluconazole is usually switched to agents with better coverage. This will avoid the possibility of reducing effective antifungal coverage for the purpose of the study.
- Failure to sign informed consent, or inability to undergo informed consent process.
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 |
|---|---|---|
| MicafunginAlso: MycamineMicafungin (trade name Mycamine) is an echinocandin antifungal drug developed by Astellas Pharma. It inhibits the production of beta-1,3-glucan, an essential component of fungal cell walls. Micafungin is administered intravenously. It received final approval from the U.S. Food and Drug Administration on March 16, 2005, and gained approval in the European Union on April 25, 2008. | Drug | Micafungin |
Primary outcome
Pharmacokinetics to measure the levels of micafungin
Time frame: Prior to the micafungin infusion at hour 0, at the end of the infusion (60 min), then at 11/2, 2, 4, 6, 8, 10, 24, 36, 48, 60, 72, 84 and 96 hours after the start of the micafungin infusion.
Blood samples from the above time points will be analyzed to study the bodily absorption, distribution, metabolism, and excretion of micafungin
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2018.
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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.
Parinda Mehta, MD principal investigator
CCHMC
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Micafungin in people with Immunocompromised and Bone Marrow Transplant, run by Children's Hospital Medical Center, Cincinnati 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 6 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: stopped early, as of the sponsor's last update on 23 May 2018. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 was stopped early. Results are not available in the source record.
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.
What does the primary outcome measure?
Pharmacokinetics to measure the levels of micafungin (time frame: Prior to the micafungin infusion at hour 0, at the end of the infusion (60 min), then at 11/2, 2, 4, 6, 8, 10, 24, 36, 48, 60, 72, 84 and 96 hours after the start of the micafungin infusion.). It is what the study was designed to measure, not a finding.
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 23 May 2018.