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

NCT ID: NCT05583669

Last Updated: 2023-05-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-11-08
• Study Completion Date: 2023-05-11

Brief 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 study for DS-2325a will evaluate the safety, tolerability, and pharmacokinetics of multiple ascending doses of DS-2325a in healthy participants. DS-2325a will be evaluated after subcutaneous (SC) injections.

Conditions

Netherton Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

DS-2325a SC

Participants who will be randomized to receive DS-2325a as a fixed dose subcutaneous (SC) injection (starting dose 300 mg).

Group Type: EXPERIMENTAL

DS-2325a

Intervention Type: DRUG

Subcutaneous injection (starting dose 300 mg)

Placebo SC

Participants who will be randomized to receive placebo as a subcutaneous (SC) injection.

Group Type: EXPERIMENTAL

Placebo

Intervention Type: DRUG

Subcutaneous injection

Interventions

DS-2325a

Subcutaneous injection (starting dose 300 mg)

Intervention Type: DRUG

Placebo

Subcutaneous injection

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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).

Exclusion Criteria

• History or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, metabolic, endocrine, immunologic, infectious, dermatologic, neurologic, oncologic, psychological, psychiatric, ophthalmologic, or gastrointestinal disease (except cholecystectomy), as judged by the Investigator.
• History or presence of chronic lung or respiratory disease, including clinically significant asthma (as judged by the Investigator) and chronic obstructive pulmonary disease (COPD).
• History, or presence in the average of triplicate ECGs at Screening and Admission (Day -2).
• Laboratory results (serum chemistry, hematology, coagulation, and urinalysis) outside the normal range, if considered clinically significant by the Investigator at Screening or Admission (Day -2).
• Creatinine clearance (CrCl) <80 mL/mina t Screening.
• History or presence of any other clinically significant condition, including laboratory abnormality, that in the opinion of the Investigator, would jeopardize the safety of the participant, obtaining informed consent, compliance to the study procedures, or the validity of the study results.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Daiichi Sankyo

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Director

Role: STUDY_DIRECTOR

Daiichi Sankyo

Locations

Quotient Sciences -Miami

Miami, Florida, United States

Countries

United States

Other Identifiers

DS2325-106

Identifier Type: -

Identifier Source: org_study_id