A study to examine the comparative exposure of N-Acetyl-D-Mannosamine Monohydrate (ManNAc) when administered orally as a solution and two novel tablet formulations in healthy adult males
- Conditions
- GNE MyopathyHereditary Inclusion Body MyopathyMusculoskeletal - Other muscular and skeletal disorders
- Registration Number
- ACTRN12623000609651
- Lead Sponsor
- niversity of South Australia
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Male
- Target Recruitment
- 6
1. Male and aged 18-59 years, inclusive.
2. Body mass index (BMI) is between 18.0 – 30.0 kg/m^2 with a body weight between 45.0 – 120.0 kg.
3. Medically healthy without any clinically significant abnormalities. Health status will be determined by the participant's medical history with specific attention to: (i) drug history, identifying any known drug allergies or drug abuse, (ii) any chronic use of medication, and (iii) a thorough review of body systems (vital signs, electrocardiogram (ECG), physical examination and clinical laboratory tests).
4. Adequate venous access on their left or right arm to allow for collection of multiple blood samples.
5. Aware of the study procedures and the risks involved, and voluntarily agrees to participate by providing written informed consent.
6. Willing and able to understand the study procedures and communicate effectively with study personnel.
1. History or presence of any medical condition that, in the opinion of the Medical Officer, may pose an unacceptable level of risk to participants or study staff, may interfere with the interpretation of safety data obtained in the trial, or may interfere with the absorption, distribution, metabolism, or excretion of ManNAc.
2. History of hypersensitivity to ManNAc or ingredients within the formulations or, in the judgment of the Medical Officer, has a condition that places the participant at increased risk for adverse effects.
3. Ingestion of an investigational medication or a new chemical entity within 30 days or 5 half-lives (whichever is longer) prior to dosing in Period 1.
4. Treated with ManNAc, sialic acid, intravenous immunoglobulin (IVIg), and/or other supplement containing sialic acid (e.g. St. John’s Wort, sialyllactose) within 120 days prior to dosing in Period 1.
5. Unable or unwilling to refrain from the use of medications, including complementary therapies and supplements, within 5 half-lives of, or for 24 h prior to dosing (whichever is longer) until completion of all study procedures within each study period.
6. Major surgery within 4 weeks prior to the screening evaluation, or planned surgery prior to completion of all study procedures.
7. Donation of blood or blood products of 470 mL or greater within 12 weeks prior to dosing in Period 1, and/or unable or unwilling to refrain from donation from the screening evaluation until completion of all study procedures.
8. History or current evidence of alcohol abuse and/or unable or unwilling to refrain from alcohol consumption for 24 h prior to dosing until completion of all study procedures in each study period.
9. History or current evidence of drug abuse, positive urine drug screen during screening, and/or unable or unwilling to refrain ingestion of drugs of abuse from the screening evaluation until completion of all study procedures.
10. Unable or unwilling to refrain from consuming food and/or beverages that contain caffeine or other xanthines (e.g. coffee, tea, cola, energy drinks and chocolate) for 24 h prior to dosing until completion of all study procedures in each study period.
11. Unable or unwilling to refrain from the use of tobacco products for 24 h prior to dosing until completion of all study procedures in each study period.
12. Unable or unwilling to refrain from food intake from 10 h prior to dosing until the 4 h timepoint in each study period.
13. Unable or unwilling to refrain from fluid intake, aside from that given as part of study procedures, from 1 h prior to dosing until the 4 h timepoint in each study period.
14. Unable or unwilling to remain seated in an upright position from immediately prior to dosing until the 4 h timepoint in each study period.
15. Unable or unwilling to be confined to the UniSA Clinical Trial Facility for 12 hours on the specified study days and/or attend the other study visits.
16. Dietary requirements that prevent consumption of the standardised study meals.
17. Poor complier or unlikely to attend specified study days.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method ManNAc pharmacokinetic parameters including:<br>- Area under the concentration versus time profile from 0 to the last quantifiable concentration (AUClast)<br>- Maximum observed plasma concentration (Cmax)<br>- Time to maximum plasma concentration (Tmax)<br>In addition, where the terminal phase of the concentration-time profile can be determined, the following pharmacokinetic parameters will also be calculated:<br>- Terminal rate constant<br>- Area under the concentration versus time profile extrapolated to infinite time (AUCinf)<br>- Terminal half-life (T1/2)<br>- Clearance (CL)<br>- Volume of distribution (Vd)<br><br>Pharmacokinetic analysis will be based on baseline-corrected plasma ManNAc concentrations,[ Prior to dosing (0 h) and then at 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 7, 8, 10, 12 and 24 h.<br>]
- Secondary Outcome Measures
Name Time Method