A Phase 1, Subject- and Investigator-Blinded, Sponsor-Unblinded, Placebo-Controlled, Randomized, Sequential Cohort Study to Assess the Safety and Pharmacokinetics of Multiple Ascending Subcutaneous Doses of DS-2325a in Healthy Subjects
Netherton syndrome (NS) is a rare autosomal recessive disease and no systemic treatment or standard of care currently exists for patients with NS. DS-2325a, a specific and potent inhibitor of kallikrein 5, is expected to treat NS by replacing a defective gene.
• Must be able to understand a written informed consent, which must be obtained prior to initiation of study procedures.
• Must be willing and able to comply with all study requirements.
• Healthy males or non-pregnant, non-lactating healthy females.
• Aged 18 to 50 years of age (inclusive) at the time of signing informed consent.
• BMI of 18.0 kg/m\^2 to 30.0 kg/m\^2 (inclusive) as measured at Screening.
• Women of childbearing potential who are sexually active with a male partner must practice effective contraception during the study treatment period and for 90 days after last IMP administration. They must agree to use 2 different means of nonhormonal contraceptive methods.
• Women of non-childbearing potential must be either surgically sterile or in menopausal state confirmed as follows: 1 year of spontaneous amenorrhea without an alternative medical cause and a serum follicle stimulating hormone (FSH) level ≥40 mIU/mL.
• Male participants who are sexually active with a female partner of childbearing potential must use, with their partner, a condom plus an approved method of highly effective contraception from the time of informed consent until 90 days after last IMP administration.
• Women should not donate eggs and men should not donate sperm during the study treatment period and for at least 90 days after last IMP administration.
• All female participants must have a negative serum pregnancy test at Screening and Admission (Day -2).