Phase 1, Double-blind, Randomized, Placebo-controlled, Single Ascending Dose Study of Subcutaneous APL-9 in Healthy Volunteers
- Conditions
- Paroxysmal nocturnal hemoglobinuria (PNH)Blood - Haematological diseases
- Registration Number
- ACTRN12616000862448
- Lead Sponsor
- Clinical Network Services Pty Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Stopped early
- Sex
- All
- Target Recruitment
- 6
Medically healthy adults
Weigh more than 50 kg and less than 95 kg and have a BMI higher than 18.0 kg/m2 and lower than 32.0 kg/m2.
Have been vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenza within two years or willing to receive vaccinations.
Mentally or legally incapacitated or has significant emotional problems or has a history of a significant medical or psychiatric condition or a history of hypersensitivity to compounds related to APL-9 or a history of chronic infections or a recent active infection or recent surgery.
Use of any prescription or non-prescription medications, herbal remedies, or vitamin supplements within the last 14 days
Blood donation or significant blood loss within previous 56 days or plasma donation within previous 7 days
Participation in another clinical trial within the previous 60 days or participation in any previous clinical trial with APL-9.
Female subjects who are pregnant or lactating.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The safety and tolerability of single subcutaneous doses of APL-9 when administered to healthy adults.[Throughout the study, routine clinical tests will be conducted, including vital signs, ECGs, and blood and urine tests.<br>Vital signs and ECGs will be recorded at screening, upon check-in to the clinic on the day before dosing, before dosing; at 1 and 4 hours post-dosing on Day 1, and then once per day on Day 2, 3, 4, 5, 6, 7, 8, 15, 22, 29, and 43.<br>Blood and urine samples will be collected for testing at screening, upon check-in to the clinic on the day before dosing. then on Day 2, 4, 8, 15, 29, and 43.<br>]
- Secondary Outcome Measures
Name Time Method Serum pharmacokinetics of single subcutaneous doses of APL-9 when administered to healthy adults. AUC, Cmax, tmax, Kel and t1/2 will be determined.[Blood will be collected for serum APL-9 measurements before dosing; at 15 and 30 minutes and 1, 4, 8 and 12 hours post-dosing on Day 1, and then once per day on Day 2, 3, 4, 5, 6, 7, 8, 15, 22, 29, and 43.];Serum pharmacodynamics of single subcutaneous doses of APL-9 when administered to healthy adults, AH50, CH50, C3 and C3a will be measured.[Blood will be collected for serum complement activation marker measurements before dosing; at 1, 4 and 12 hours post-dosing on Day 1, and then once per day on Day 2, 3, 4, 5, 6, 7, 8, 15, 22, 29, and 43.]