A Study to Evaluate Isavuconazonium Sulfate for the Treatment of Invasive Aspergillosis (IA) or Invasive Mucormycosis (IM) in Pediatric Participants
NCT ID: NCT03816176
Last Updated: 2024-12-05
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
31 participants
INTERVENTIONAL
2019-08-22
2022-12-14
Brief Summary
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Detailed Description
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Participants received a loading regimen of isavuconazonium sulfate (via intravenous or oral administration at the investigator's discretion), which consisted of a dose every 8 hours (± 2 hours) on Days 1 and 2 (for a total of 6 doses), followed by once daily maintenance dosing for up to 84 days (IA) or 180 days (IM) of dosing. The first maintenance dose started 12 to 24 hours after the administration of the last loading dose. Subsequent maintenance doses were administered once daily (24 hours ± 2 hours from the previous maintenance dose). The oral formulation could only be given to participants 6 years to \< 18 years of age and with a body weight of at least 12 kg. Participants who were discharged from the hospital with oral capsules for at-home administration had to return weekly for study drug accountability and to receive new oral dosing supplies. Participants who began oral administration were to complete the oral dosing acceptability assessment after ingesting their first oral dose.
Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Isavuconazonium sulfate
Participants received 10 milligrams/killograms (mg/kg) dose of isavuconazonium sulfate every 8 hours (± 2 hours) on days 1 and 2 for a total of 6 doses (via intravenous or oral administration at the investigator's discretion) followed by once-daily maintenance dose of 10 mg/kg for up to 84 days IA or 180 days IM or until the participant had a successful outcome as judged by the investigator, whichever occured first. The route of administration could have been changed per the investigator's discretion.
Isavuconazonium sulfate
Intravenous (IV) infusion
Isavuconazonium sulfate
Oral capsule
Interventions
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Isavuconazonium sulfate
Intravenous (IV) infusion
Isavuconazonium sulfate
Oral capsule
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Proven, probable or possible IFI per the European Organisation for Research and Treatment of Cancer/Mycoses Study Group \[EORTC/MSG\], 2008 criteria Note: Subjects with "possible" IFI will be eligible for enrollment; however, diagnostic tests to confirm the invasive fungal disease as "probable" or "proven" according to the EORTC/MSG criteria should be completed within 10 calendar days after the first dose of study drug
* Note: In addition to the criteria set for mycological criteria by the EORTC/MSG in 2008, and only for subjects with an underlying hematologic malignancy or recipients of hematopoietic stem cell transplant (HSCT) who also have clinical and radiologic features consistent with invasive fungal infection, the following are acceptable:
* Galactomannan (GM) levels (optical density index) meeting the below criteria are acceptable mycological evidence for enrollment or upgrading the diagnosis to probable IA:
* 1\. A single value for serum or bronchoalveolar lavage (BAL) fluid of ≥ 1.0 or
* 2\. Two serum GM values of ≥ 0.5 from two separate samples
* Subject has sufficient venous access to permit intravenous administration of study drug or the ability to swallow oral capsules
* A female subject is eligible to participate if not pregnant and at least one of the following conditions applies:
* Not a subject who is of childbearing potential, OR
* Subject who is of childbearing potential who agrees to follow a contraceptive guidance throughout the treatment period and for at least 30 days after the final study drug administration
* Subject and subject's parent(s) or legal guardian agree that the subject will not participate in another interventional study while on treatment with the exception of oncology trials
Exclusion Criteria
* Subject has evidence of hepatic dysfunction defined as any of the following:
* Total bilirubin (TBL) ≥ 3 times the upper limit of normal (ULN)
* Alanine transaminase (ALT) or aspartate transaminase (AST) ≥ 5 times the ULN
* Known cirrhosis or chronic hepatic failure
* Subject has used strong cytochrome P450 (CYP3A4) inhibitors or inducers such as ketoconazole, high dose ritonavir, rifampin/rifampicin, long acting barbiturates (e.g., phenytoin), carbamazepine and St. John's Wort in the 5 days prior to the first dose of study drug
* Subject has another IFI other than possible, probably or proven IA or IM
* Subject has chronic aspergillosis, aspergilloma or allergic bronchopulmonary aspergillosis
* Subject has received mould active systemic antifungal therapy, effective against the primary IMI, for more than four days during the seven days preceding the first dose
* Note: Prior use of prophylactic antifungal therapy is acceptable. In case of breakthrough IA while on prophylactic mould-active azole class drugs, additional documentation will be required to be submitted to the sponsor medical monitor or designee to approve subject enrollment
* Subject has known history of allergy, hypersensitivity or any serious reaction to any of the azole class antifungals, or any components of the study drug formulation
* Subject has any condition which makes the subject unsuitable for study participation
* Subject is unlikely to survive 30 days
* Subject has received investigational drug, with the exception of oncology drug trials, or trials with investigational drugs treating graft versus host disease, within 28 days or five half-lives, whichever is longer, prior to screening
1 Year
17 Years
ALL
No
Sponsors
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Astellas Pharma Global Development, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Executive Director
Role: STUDY_DIRECTOR
Astellas Pharma Global Development, Inc.
Locations
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Children's Hospital, Los Angeles
Los Angeles, California, United States
University of California - Los Angeles
Los Angeles, California, United States
Children's Hospital of Orange County
Orange, California, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Site BE32001
Ghent, , Belgium
Site BE32002
Leuven, , Belgium
Site ES34002
Barcelona, , Spain
Site ES34003
Madrid, , Spain
Site ES34001
Madrid, , Spain
Countries
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References
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Segers H, Deville JG, Muller WJ, Manzanares A, Desai A, Neely M, Bordon V, Hanisch B, Lassaletta A, Fisher BT, Autmizguine J, Groll AH, Sinnar S, Croos-Dabrera R, Engelhardt M, Jones M, Kovanda LL, Arrieta AC. Safety, outcomes, and pharmacokinetics of isavuconazole as a treatment for invasive fungal diseases in pediatric patients: a non-comparative phase 2 trial. Antimicrob Agents Chemother. 2024 Dec 5;68(12):e0048424. doi: 10.1128/aac.00484-24. Epub 2024 Nov 14.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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Link to results and other applicable study documents on the Astellas Clinical Trials website
Link to plain language summary of the study on the Trial Results Summaries website
Other Identifiers
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2018-003975-36
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
9766-CL-0107
Identifier Type: -
Identifier Source: org_study_id