Niemann-Pick disease, GM2 gangliosidosis and ataxia telangiectasia Nervous System Diseases
Conditions
Interventions
This is a two-part study. Part 1 is a two-period, open-label, non-randomised study. Part 2 is an open-label, part-randomised, two-way crossover study. It is planned to enrol 16 and 14 healthy male and
Sponsors
IntraBio Inc.
Eligibility
Sex/Gender
All
Age
18 Years to 55 Years
Inclusion criteria
Inclusion criteria: 1. Must provide written informed consent 2. Must be willing and able to communicate and participate in the whole study 3. Subject is willing to consume the entirety of a high-fat breakfast including bacon, dairy and eggs (Part 2 only) 4. Aged 18 to 55 years inclusive at the time of signing informed consent 5. Must agree to adhere to the contraception requirements defined in the clinical protocol 6. Healthy male or non-pregnant, non-lactating healthy females according to the assessment of the investigator, as based on a complete medical history including a physical examination, vital signs, 12-lead ECG, and laboratory safety tests without any clinically significant abnormalities. Safety bloods, urinalysis, ECGs and vital signs to be re-checked at admission (Part 1 only) 7. Body mass index (BMI) of 18.0 to 32.0 kg/m2 as measured at screening 8. Weight =50 kg at screening
Exclusion criteria
Exclusion criteria: 1. Serious adverse reaction or serious hypersensitivity to any drug or formulation excipients 2. Presence or history of clinically significant allergy requiring treatment, as judged by the investigator. Hay fever is allowed unless it is active 3. History of clinically significant cardiovascular, renal, hepatic, dermatological, chronic respiratory or gastrointestinal disease, neurological or psychiatric disorder, as judged by the investigator 4. Subjects with a history of cholecystectomy or gallstones 5. Subjects who do not have suitable veins for multiple venepunctures/cannulations as assessed by the investigator or delegate at screening 6. Clinically significant abnormal clinical chemistry (including CK >1.5 × ULN), haematology or urinalysis as judged by the investigator (detailed in the clinical protocol) 7. Positive hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCV Ab) or human immunodeficiency virus (HIV) 1 and 2 antibody results 8. Evidence of renal impairment at screening, as indicated by an estimated eGFR of 21 units per week and in females >14 units per week (1 unit = ½ pint beer, or a 25 mL shot of 40% spirit, 1.5 to 2 units = 125 mL glass of wine, depending on type) 17. A confirmed positive alcohol breath test at screening or admission 18. Current smokers and those who have smoked within the last 12 months 19. Current users of e-cigarettes and nicotine replacement products and those who have used these products within the last 12 months 20. A confirmed breath carbon monoxide reading of greater than 10 ppm at screening or admission 21. Confirmed positive drugs of abuse test result at screening or admission 22. Male subjects with pregnant or lactating partners or partners planning to become pregnant 23. Subjects who are, or are immediate family members of, a study site or sponsor employee 24. Failure to satisfy the investigator of fitness to participate for any other reason
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part 1: 1. PK parameters including but not limited to Cmax, AUC(0-last), and AUC(0-inf), for digoxin alone and in combination with IB1001, measured using blood samples taken from Period 1 Day 1 to Period 2 Day 6 2. PK parameters including but not limited to Cmax, AUC(0-last) and AUC(0-inf) for rosuvastatin alone and in combination with IB1001, measured using blood samples taken from Period 1 Day 1 to Period 2 Day 6 Part 2: 1. Results of the formal statistical analysis of PK parameters Cmax, AUC(0-last) and AUC(0 inf) for IB1001 in the fed vs fasted states, measured using blood samples taken from Period 1 Day 1 to Period 2 Day 2 | — |
Secondary
| Measure | Time frame |
|---|---|
| Parts 1 and 2: Safety and tolerability measured using the incidence of treatment-emergent adverse events, physical examinations and change from baseline in vital signs, electrocardiograms and laboratory safety tests from Day -1 of Period 1 until the follow-up phone call in each study part. | — |
Countries
England, United Kingdom
Outcome results
None listed