None listed
Conditions
Brief summary
Stargazer Pharmaceuticals. is developing STG-001 a potential oral therapy for patients with Stargardt's disease. This study will be conducted in up to 84 healthy volunteers who meet all of the inclusion criteria and none of the exclusion criteria. The study is to assess the safety and tolerability of STG-001 in normal healthy volunteers. This includes vital signs, safety labs, ECGs, and ocular and non-ocular examinations. The drug will be given in single ascending then multiple ascending doses. The study will also evaluate the PK and PD of the drug after dose administration. Participants will be entered into standard study cohorts
Interventions
Intervention: STG-001
Administration: Oral capsule
Part 1: Single Ascending Dose (SAD) study
Cohort 1 Fasted: STG-001 6 active (1 at 0.2 and 5 at 1 mg) and 2 placebo
Cohort 2 Fasted: STG-001 5mg (6 active, 2 placebo)
Cohort 3 Fasted and Fed : STG-001 10mg (6 active, 2 placebo)
Cohort Fasted 4: STG-001 20mg (6 active, 2 placebo)
Cohort Fasted 5: STG-001 40mg (6 active, 2 placebo)
Optional Cohort Fasted 9: STG-001 80mg (6active, 2 placebo)
The Fed period will be conducted in Cohort 3 after a washo
Intervention: STG-001 Administration: Oral capsule Part 1: Single Ascending Dose (SAD) study Cohort 1 Fasted: STG-001 6 active (1 at 0.2 and 5 at 1 mg) and 2 placebo Cohort 2 Fasted: STG-001 5mg (6 active, 2 placebo) Cohort 3 Fasted and Fed : STG-001 10mg (6 active, 2 placebo) Cohort Fasted 4: STG-001 20mg (6 active, 2 placebo) Cohort Fasted 5: STG-001 40mg (6 active, 2 placebo) Optional Cohort Fasted 9: STG-001 80mg (6active, 2 placebo) The Fed period will be conducted in Cohort 3 after a washout period of >=28 days and, based on prior SAD data, dose of STG-001 will be determined to be studied under fed conditions. Part 2: (Multiple Ascending Dose) MAD Study (STG-001 starting dose to be confirmed based on SAD results and subsequent ascending dose to be determined by study investigator based on safety and PK data,). IP will be administered once daily for 7 days. Cohort 6 Fasted: STG-001 'X' mg (7 active and 2 placebo) Cohort 7 Fasted: STG-001 'Y' mg (7 active and 2 placebo) cohort 8 Fasted: STG-001 'Z' mg (7 active and 2 placebo) Optional cohort 10 Fasted: STG-001 'A' mg (7 active and 2 placebo) New participants will be enrolled in each cohort. In cohort 3, participants will return after >=28 days for Fed dosing during which subjects will all receive a dose of STG-001 within 30 minutes after completing a high-fat meal (- 2 normal fried eggs, 2 slices white toast, 250 mL whole milk, 2 full rashes middle bacon, 2 serves butter for cooking, 2 serves butter for toast, 2 hash browns) The cohorts will run sequentially such that the study periods for Cohort 2 occur after Cohort 1. MAD cohort will commence after completion and safety review of SAD cohort 4. Participants in SAD and MAD cohorts will not be the same. At the discretion of Sponsor, and with approval of the Review Committee, Sponsor may enroll an additional cohort in the SAD portion of study (Cohort 9) and an additional cohort in the MAD portion of study (Cohort 10). The dose of the MAD cohort will be determined by the Review committee.
Sponsors
Stargazer Pharmaceuticals
Study design
Allocation
Randomised controlled trial
Intervention model
Parallel
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver)
Eligibility
Sex/Gender
All
Age
18 Years to 55 Years
Healthy volunteers
Yes
Inclusion criteria
The subject must understand the study procedures and agree to participate by providing written informed consent. The subject must be willing and able to comply with all study procedures and restrictions. To be eligible for participation in this study, the subject must: 1. Understand the study procedures and agree to participate by providing written informed consent. 2. Healthy male and female subjects. 3. Subjects aged 18 to 55 years, inclusive, with BMI of 18 to 32 kg/m2, inclusive, and body weight >=50 kg. 4. Male subjects must abstain from heterosexual activities or agree to use a double barrier method of contraception from admission to the clinical unit through 90 days after the last dose of study drug and will not donate sperm during this period. Heterosexual WOCBP who are sexually active must not be pregnant upon entering the study that is confirmed by testing (i.e., serum pregnancy test with sensitivity of >=25 mIU/mL at screening, and urine pregnancy test with sensitivity >=50 mIU/mL on Day 1 prior to dosing), be willing to use double-barrier contraception from screening through 60 days after the last dose of study drug, and will not donate eggs during this period. Highly effective double barrier contraception is defined as a condom or diaphragm AND one of the following; o Birth control pills (The Pill) o Birth Control Patch (e.g. Ortho Evra) o NuvaRing® o Depot or injectable birth control o IUD o Documented evidence of surgical sterilization at least 6 months prior to screening visit, i.e., tubal ligation or hysterectomy for women or vasectomy for men. 5. Sign an approved informed consent form for the study. 6. Pre-study ocular exam with no clinically significant abnormalities, including no significant macular abnormalities, DA testing within normal limits and BCVA >= 20/50 visual acuity (or equivalent) 7. Willing and able to comply with the protocol, including attending assessment visits. 8. Be judged to be in good health by the Investigator, based on clinical evaluations including laboratory safety tests, medical history, physical examination, ECG, and vital sign measurements performed at the screening visit and before administration of the initial dose of study drug.
Exclusion criteria
1. The subject is an employee of the sponsor or study site or immediate family member (e.g., spouse, parent, child, sibling) of the sponsor or study site. 2. The subject is a citizen or resident of an EU member State. 3. The subject has a known hypersensitivity or contraindication to any component of STG 001. 4. The subjects has any history of an anaphylaxis event. 5. Use of prescription or nonprescription drugs and dietary supplements within 7 days or 5 half-lives (whichever is longer) prior to Check-in (Day -1). Herbal supplements and HRT must be discontinued 28 days prior to Check-in (Day -1). As an exception, paracetamol may be used at doses of less than or equal to 1 g/day. Limited use of nonprescription medications that are not believed to affect subject safety or the overall results of the study may be permitted on a case-by-case basis following approval by the Sponsor. 6. The subject has vitamin A deficiency as defined based upon serum values less than 20 mcg/dl (0.7 µmol/L) or clinical signs during slit lamp examination (conjunctival or corneal xerosis; Bitot’s spots; corneal ulcers of scarring not due to trauma or other secondary causes). 7. Has taken non-approved items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days prior to admission at the clinical site. 8. Use of medications that may interact with Vitamin A metabolism within 60 days of screening (e.g. Accutane [isotretinoin], doxycycline). 9. Participation in an interventional study of a Vitamin A derivative less than or equal to months prior to screening 10. Presence of significant cardiovascular or cerebrovascular disease, including stroke. 11. Has a clinically significant abnormal electrocardiogram (ECG), or has a corrected QT interval (QTc) that is 450 ms or greater 12. Resting heart rate outside specified limits (less than 40/minute, greater than 100/min upon repeated measurement). 13. History of diabetes, hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or hypervitaminosis A. 14. Any surgical procedure within three month of trial entry or anticipated during the trial. If such surgical procedure was within 3 months of trial entry and there was a full recovery with no expected impact on study procedures, data or safety, such a subject would be eligible at the discretion of the PI. 15. Women who are pregnant or nursing. 16. Abnormal blood pressure outside specified limits (90 mm Hg > Systolic > 140 mm Hg and/or 40 mm Hg > Diastolic > 90 mm Hg) upon repeated measurement. 17. Clinically significant abnormal lab results at screening, including liver function test (aspartate transaminase, alanine transaminase, bilirubin and alkaline phosphatase) greater than 1.5 x the ULN 18. Actively participating in an experimental therapy study or have received experimental therapy within 60 days of screening or 5 half-lives, whichever is longer. 19. Any history of significant eye disorders (including retinal disorders) or visual disturbances. 20. In the PI’s opinion, any acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality that may increase the risk associated with study participation or administration of study treatment or interfere with the interpretation of study results. Minor medical conditions that are not currently being treated with medication and that are not expected to interfere with study procedures, data or participant safety may be considered for inclusion at the discretion of the PI. 21. Participants could be at increased risk of harm or put other people at increased risk of harm (e.g. if they are taxi or Uber drivers), if they were to experience visual impairment like impaired night time vision. Participants must agree to comply with advice of study investigators or study ophthalmologist around modifying activity and taking precautions if visual impairment occurs until such time as the visual impairment resolves, or the participant will be excluded from the study.
Outcome results
None listed