An Open-label Phase 1b Cross-over Pharmacokinetic Relative Bioavailability Study Comparing 2 Formulations of STG-001 in Healthy Subjects
- Conditions
- Stargardt's DiseaseEye - Diseases / disorders of the eyeHuman Genetics and Inherited Disorders - Other human genetics and inherited disorders
- Registration Number
- ACTRN12620000766910
- Lead Sponsor
- Stargazer Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 10
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 signing an approved informed consent form for the study.
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 greater than or equal to 50 kg.
4.Male subjects must abstain from heterosexual activities or agree to use a highly effective double barrier method of contraception from admission to the clinical unit through 90 days after the last dose of study drug (except if female partner is documented to be post-menopausal) and will not donate sperm during this period. Heterosexual women of child-bearing potential (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 greater than or equal to 25 mIU/mL at screening, and urine pregnancy test with sensitivity greater than or equal to 25 mIU/mL on Day 1 prior to dosing), be willing to use a highly effective 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 AND one of the following highly effective methods;
oBirth control pills (The Pill)
oBirth Control Patch (e.g. Ortho Evra)
oNuvaRing®
oDepot or injectable birth control
oIUD
oContraceptive implant (e.g. Implanon)
oDocumented 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.Pre-study ocular exam with no clinically significant abnormalities, including no significant macular abnormalities, DA testing within normal limits and BCVA greater than or equal to 20/50 visual acuity (or equivalent)
6.Willing and able to comply with the protocol, including attending assessment visits.
7.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.
Any subject who meets any of the following criteria will not qualify for entry into the study:
1.The subject has a known hypersensitivity or contraindication to any component of STG 001.
2.The subject has any history of an anaphylaxis event.
3.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 tov1 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 Principal Investigator and the Sponsor.
4.The subject has a history of vitamin A deficiency 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).
5.Has taken non-approved items (supplement containing vitamin A or beta-carotene, liver-based products (foods, such as liverwurst, are not an exclusion), or prescription oral retinoid medications) within 30 days prior to admission at the clinical site.
6.Use of medications that may interact with Vitamin A metabolism within 60 days of screening (e.g. Accutane [isotretinoin], doxycycline).
7.Participation in an interventional study of a Vitamin A derivative less than or equal to 3 months prior to screening
8.Presence of significant cardiovascular or cerebrovascular disease, including stroke.
9.Has a clinically significant abnormal electrocardiogram (ECG), or has a corrected QT interval (QTc) that is 450 ms or greater
10.Resting heart rate outside specified limits (less than 40/minute, greater than 100/min upon repeated measurement).
11.History of diabetes, hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or hypervitaminosis A.
12.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.
13.Women who are pregnant or nursing.
14.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.
15.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
16.Actively participating in an experimental therapy study or have received experimental therapy within 60 days or 5 half-lives, whichever is longer, of screening.
17.Any history of significant eye disorders (including retinal disorders) or visual disturbances.
18.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 o
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method