To Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AZ-3102 (single and multiple ascending doses) in Healthy Volunteers
- Conditions
- ysosomal storage disordersLysosomal storage disordersHuman Genetics and Inherited Disorders - Other human genetics and inherited disordersMetabolic and Endocrine - Other metabolic disorders
- Registration Number
- ACTRN12621000576820
- Lead Sponsor
- Azafaros
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 35
1. Subjects must be able and willing to give written informed consent and are willing to comply with the requirements and restrictions of the study.
2. Subject must be at least 18 years of age at Screening and maximum 55 years of age on date of first dose.
3. A male participant with a female partner of childbearing potential is eligible if he agrees to follow the contraceptive guidance.
4. Female subject is eligible if she is not a woman of childbearing potential (WOCBP) OR, if she is a WOCBP, she agrees to follow the contraceptive guidance.
5. Body mass index (BMI) of greater than or equal to 18.0 kg/m2 and less than or equal to 30.0 kg/m2 at Screening.
6. Healthy as determined by the Investigator, based upon a medical evaluation including medical history, physical examination, clinical laboratory tests, and 12-lead ECG performed at Screening. Out of range values can be repeated once.
7. Subjects must be willing to use adequate contraception, and to refrain from sperm donation, from the time of dosing until 90 days after the last dose of study drug.
8. Subjects that undergo CSF collection: subjects must be willing and able to undergo CSF collections using a lumbar puncture.
Subjects will be excluded if they meet any of the following criteria:
1. Prior or ongoing medical condition, medical history, physical findings, ECG findings, laboratory, or vital signs abnormality that, in the Investigator’s opinion, could adversely affect the safety of the subject.
2. History of clinically significant drug allergies.
3. Alkaline phosphatase, aspartate aminotransferase (AST), and/or alanine aminotransferase (ALT) level greater than 1.5 x upper limit of normal (ULN) at Screening.
4.Creatinine clearance less than 90 mL/min (according to Cockcroft-Gault formula).
5.Total bilirubin greater than 1.5 x ULN (isolated bilirubin greater than 1.5 x ULN is acceptable if bilirubin is fractionated and direct bilirubin is less than 35%).
6.Platelet count less than 100 x 10^9/L
7.Use of prescription or non-prescription drugs, including vitamins, herbal and dietary supplements within 21 days or five half-lives (whichever is longer) before the first dose of study drug, unless in the opinion of the Investigator and Sponsor’s Medical Monitor the medication will not interfere with the study procedures or compromise subject safety.
8.ECG with an average of triplicate QTcF interval greater than 450 msec.
9.A female subject who has a positive pregnancy test at screening or Day -1, or is breastfeeding.
10.Positive urine drug screen or positive alcohol breath test at Screening or Day -1.
11.History of alcohol abuse within 6 months prior to Screening, defined as an average weekly intake of greater than 10 units.
12.Positive urine cotinine at screening or Day -1.
13.Positive result at screening for any of the following infectious disease tests: hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCV Ab), human immunodeficiency virus antigen and antibody (HIV Ag, HIV Ab)
14.History of seizure, head trauma, loss of consciousness, symptomatic orthostatic hypotension (e.g. postural syncope).
15.Donation of 500 mL or more blood within 3 months prior to the first dose of study drug; or any amount of plasma in the 7 days prior to Screening; or any amount of platelets in the 42 days prior to Screening.
16.Receipt of an investigational product within 90 days prior to the first dose of study drug or exposure to more than four new chemical entities within 12 months prior to the first dose of study drug.
17.CSF subjects: Any condition or anatomic abnormality that would preclude spinal CSF collection by lumbar puncture or contraindications to lumbar puncture such as papilledema/raised intracranial pressure, infection near lumbar puncture site.
18.Evidence of suicidal ideation with intent (Type 4-5) on the Columbia Suicide Severity Rating Scale (C-SSRS) at Screening (Part 2 MAD only).
19.Attempted suicide attempt in the 6 months before Screening.
20.Employee or immediate family member (eg, spouse, parent, child, sibling) of clinical research unit (CRU) or of the Sponsor.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method