A Study to Evaluate Isavuconazonium Sulfate for the Treatment of Invasive Aspergillosis (IA) or Invasive Mucormycosis (IM) in Pediatric Participants
Condition: Invasive Mucormycosis; Invasive Aspergillosis
MeSH terms: Aspergillosis; Mucormycosis; Zygomycosis
Intervention: Isavuconazonium sulfate; Isavuconazonium sulfate
Intervention description: Intravenous (IV) infusion; Oral capsule
Intervention MeSH terms: Isavuconazole
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 31.0
Sex: All
Minimum age: 1 Year
Maximum age: 17 Years
Healthy volunteers: No
Keywords: ASP9766; Cresemba; BAL8557; isavuconazonium sulfate

Summary:

      The purpose of this study was to evaluate the safety, tolerability, and efficacy of
      isavuconazonium sulfate in pediatric participants.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Subject diagnosed with IA or IM. A positive diagnosis is defined as follows:

               -  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 familial short QT syndrome, is receiving medications that are known to
             shorten the QT interval, or has a clinically significant abnormal ECG

          -  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