A Phase 2, Open-Label, Non-Comparative, Multicenter Study to Evaluate the Safety and Tolerability, Efficacy and Pharmacokinetics of Isavuconazonium Sulfate for the Treatment of Invasive Aspergillosis (IA) or Invasive Mucormycosis (IM) in Pediatric Subjects
Open to recruitment
This study invites children and young adults aged 1 year to 18years with invasive aspergillosis (IA) or invasive mucormycosis (IM), to see if they could benefit from treatment with an antifungal medication, isavuconazonium sulfate. The purpose of the study is to evaluate how safe and effective isavuconazonium sulfate is for paediatric patients. The study will also look at the way the body takes up, uses, breaks down and removes the study medicine. This is important because it will help determine how the study medicine may be used safely in children and adolescents with as few side effects as possible.
Inclusion and exclusion criteria:
- Institutional Review Board (IRB)-approved written informed consent and privacy language as per national regulations (e.g., Health Insurance Portability and Accountability Act Authorization) must be obtained from the subject’s parent or legal guardian and, if required, subject’s assent, prior to any study-related procedures (including withdrawal of prohibited medication, if applicable).
- Male or female subject 1 year to < 18 years of age 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.
- 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 she is 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 the contraceptive guidance throughout the treatment period and for at least 30 days after the final study drug administration.
- Female subject who is of childbearing potential must agree not to breastfeed starting at screening and throughout the study, and for 30 days after the final study drug administration.
- Female subject who is of childbearing potential must not donate ova starting at screening and throughout the study, and for 30 days after the final study drug administration.
- A male subject with female partner(s) of childbearing potential must agree to use contraception during the treatment period and for at least 30 days after the final study drug administration.
- Male subject must not donate sperm starting at screening and throughout the study and for 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.
Subject will be excluded from participation if any of the following apply:
- 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 4 days during the 7 days preceding the first dose.
- 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, in the investigator’s opinion, makes the subject unsuitable for study participation.
- Subject is unlikely to survive 30 days in the investigator’s opinion.
- 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 5 half-lives, whichever is longer, prior to screening.
What participating in the study involves?:
The study includes the following Study Periods:
- Screening Period (5 days before the start of the study up to day 1)
- Treatment Period (day 1 up to day 84 for participants with IA and day 180 for participants with IM)
- Follow-up Period (30 and 60 days from the last dose)