None listed
Conditions
Brief summary
This is a randomized, parallel arm, double-blind, placebo-controlled study. Subjects will be randomized to one of 5 arms, 4 dose levels of BTD-001 (25mg, 50mg, 100mg or 200mg) or placebo. Each arm consists of 6 subjects for a total of 30 subjects. Subjects will complete a Screening period of up to 28 days, and will be admitted to the Clinical Research Unit (CRU) on Day -1. First dose will be administered on the morning of Day 1. Plasma samples for PK analyses, vital signs and ECGs will be collected at multiple time points on Days 1 and 2. To allow full PK characterization of the first dose, no evening dose will be administered on Day 1, though dosing will be BID subsequently. Subjects will be discharged on Day 2 with their evening dose of medication and a diary card. Subjects will report to clinic each morning on Days 3-6 for the AM dose of medication. On Days 3, 4 and 5, they will be issued with the evening dose and a diary card. Subjects will be readmitted to the research unit of Day 6 and will receive the evening dose of study drug in the unit, approximately 12 +/- 1 hours prior to the scheduled AM dose for Day 7. Following the AM dose on Day 7, subjects will remain in the CRU for at least 8 hours, during which PK and safety measures will be collected. Subjects will then be discharged home. A final Safety Follow Up visit will take place on Day 14, one week after the last dose of study drug.
Interventions
BTD-001 will be supplied as the active drug substance in powder form, for the Pharmacist to re-constitute as a concentrated stock solution.
To improve palatability and to preserve the double blind nature of the proposed trial, Balance has established that grape Kool-AID and orange TANG (provided by Balance) mask the bitterness of the drug substance. Subjects will be allowed to choose either Kool-AID or TANG and maintain that choice throughout the study. BTD-001 stock solution is diluted with
BTD-001 will be supplied as the active drug substance in powder form, for the Pharmacist to re-constitute as a concentrated stock solution. To improve palatability and to preserve the double blind nature of the proposed trial, Balance has established that grape Kool-AID and orange TANG (provided by Balance) mask the bitterness of the drug substance. Subjects will be allowed to choose either Kool-AID or TANG and maintain that choice throughout the study. BTD-001 stock solution is diluted with 200mL of the fruit flavored drink by the pharmacist and administered at 4 dose levels: 25 mg 50 mg 100 mg 200 mg Placebo will be Sterile, non pyrogenic Water for Irrigation, BP. One milliliter (1 mL) of placebo will be diluted in 200 mL of a fruit flavored drink (e.g., orange TANG or grape Kool AID) and consumed orally by the subject. The subject will remain on the same dose throughout the intervention period. The pharmacist will prepare the BTD-001 stock solutions daily for the final drug product and placebo. The dosing period is a total of 7 consecutive days. On day 1 subjects receive a single dose of study medication in the AM. On Days 2-5, the pharmacist will dispense two doses for each subject, one to be consumed in the AM at the unit, and one to be consumed at home in the evening. Morning dose on Days 1, 2 and 7 is consumed at least 1 hour prior to breakfast; on Days 3-6 may be at least 1 hour prior to, or at least 2 hours after breakfast. The PM dose for days 2-6 is to be taken at least 2 hours after the evening meal. On day 6 the subject will receive both the AM and PM dose in the unit. On day 7 a single AM dose is administered in the unit. Compliance for AM doses will be monitored and documented by site staff. compliance for PM doses not consumed in the unit will be determined by examining returned dosing containers and questioning the volunteer. Compliance will be documented in a binary form only - volunteer compliant yes or no.
Sponsors
Balance Therapeutics Pty Limited
Study design
Allocation
Randomised controlled trial
Intervention model
Parallel
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
18 Years to 35 Years
Healthy volunteers
Yes
Inclusion criteria
1. Healthy males and females as determined by medical history and physical examination between 18 and 35 years of age; 2. Written informed consent from the subject; 3. Able to comply with requirements for concomitant medications and consumption of caffeine, alcohol, tobacco and nicotine; 4. For females of child bearing potential, start date of their last menstrual period must be within 28 days of Day 1, first dose of study drug; 5. Sexually active females of childbearing potential must be willing to use a highly effective method of birth control from start of Screening until at least 3 months after last dose of study drug [defined as one that results in a low failure rate (i.e., less than 1% per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, or some intrauterine contraceptive devices (IUDs); -female partners of male study participants must use one of the following methods: Barrier contraceptive (e.g., female condom, cervical cap, diaphragm); Hormonal (oral, injectable, transdermal patch); or Intrauterine device; AND -male participants must use condoms.
Exclusion criteria
1. Weight <50 kg or > 110 kg; 2. BMI <18 kg/m2 or >35 kg/m2; 3. History of epilepsy, generalized convulsion or focal seizure (with exception of a single febrile seizure prior to 5 years of age); 4. Clinically significant (i.e., requiring medication or psychotherapy) psychiatric condition within the 2 years prior to Day 1, including but not limited to depression, bipolar disorder, generalized anxiety disorder, phobic disorders, panic disorder; 5. History of suicide attempt at any time, or of suicidal thinking within the last 6 months or behavior within the last 5 years reported on Screening Columbia Suicide Severity Rating Scale; 6. Personal or family history of congenital long QT syndrome or sudden death; 7. History of or current clinically significant or unstable neurological, psychiatric, cardiovascular, pulmonary, peripheral vascular, gastrointestinal, hepatic, endocrinological, hematological, or immunological conditions; 8. Any condition possibly affecting drug absorption (e.g., gastrectomy, vagotomy, or bowel resection); 9. Estimated glomerular filtration rate (eGFR) <90 mL/min by Cockroft Gault) at Screening or Baseline; 10. Screening or Baseline systolic blood pressure > 160 mm Hg or a diastolic blood pressure > 90 mm Hg (sitting or supine). One repeat measure is allowed to confirm eligibility; 11. Clinically significant abnormal electrocardiogram (ECG) at Screening or Baseline; 12. Clinically significant abnormalities in laboratory test results at Screening or Baseline; 13. Breastfeeding or pregnancy (females only); 14. Treatment with an investigational drug or biologic within 60 days preceding Day 1 (the first dose of study medication) or plans to take another investigational drug or biologic within 30 days of study completion; 15. Flu vaccination within 14 days of Day 1; 16. Blood donation or significant blood loss within 60 days prior to Day 1; 17. Plasma donation within 7 days of Day 1; 18. Other severe, acute, or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judgment of the Investigator, would make the subject inappropriate for entry into this trial.
Outcome results
None listed