NCT05388903

A Study to Assess the Safety and Pharmacokinetics of Single Ascending Subcutaneous and Intravenous Doses of DS-2325a in Healthy Subjects

A Phase 1, Subjects- and Investigator-Blinded, Sponsor-Unblinded, Placebo-Controlled, Randomized, Sequential Cohort Study to Assess the Safety and Pharmacokinetics of Single Ascending Subcutaneous and Intravenous Doses of DS-2325a in Healthy Subjects

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 64 (actual)
• Sponsor: Daiichi Sankyo · Industry
• Sex: All
• Age: 18 Years – 45 Years
• Healthy volunteers: Accepted

Summary

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.

Detailed description

This first-in-human study for DS-2325a will evaluate the safety, tolerability, and pharmacokinetics of single ascending doses of DS-2325a in healthy participants. DS-2325a will be evaluated after both subcutaneous (SC) injections and intravenous (IV) infusions, as it may have to be given intravenously to young patients and as loading dose.

Conditions

• Netherton Syndrome

Interventions

Type	Name	Description
DRUG	DS-2325a	Subcutaneous injection (starting dose 30 mg)
DRUG	DS-2325a	Intravenous infusion (starting dose 100 mg)
DRUG	Placebo	Subcutaneous injection
DRUG	Placebo	Intravenous infusion

Timeline

• Start date: 2022-06-20
• Primary completion: 2023-01-26
• Completion: 2023-01-26
• First posted: 2022-05-24
• Last updated: 2023-03-06

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study