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

NCT ID: NCT05388903

Last Updated: 2023-03-06

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 64 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-06-20
• Study Completion Date: 2023-01-26

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 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

Study Design

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

Study Groups

DS-2325a SC

Participants who will be randomized to receive DS-2325a as a fixed dose subcutaneous (SC) injection (starting dose 30 mg).

Group Type: EXPERIMENTAL

DS-2325a

Intervention Type: DRUG

Subcutaneous injection (starting dose 30 mg)

Placebo SC

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Subcutaneous injection

DS-2325a IV

Participants who will be randomized to receive DS-2325a as a fixed dose intravenous (IV) infusion (starting dose 100 mg).

Group Type: EXPERIMENTAL

DS-2325a

Intervention Type: DRUG

Intravenous infusion (starting dose 100 mg)

Placebo IV

Participants who will be randomized to receive placebo as an intravenous infusion.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Intravenous infusion

Interventions

DS-2325a

Subcutaneous injection (starting dose 30 mg)

Intervention Type: DRUG

DS-2325a

Intravenous infusion (starting dose 100 mg)

Intervention Type: DRUG

Placebo

Subcutaneous injection

Intervention Type: DRUG

Placebo

Intravenous infusion

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Participants must give written informed consent to participation in the study prior to Screening
• Healthy men and women 18 to 45 years of age (inclusive), with a BMI of 18 kg/m2 to 30 kg/m^2 (inclusive) at Screening
• All women must have a negative serum pregnancy test at Screening and all women of childbearing potential must have a negative urine pregnancy test on Day -1
• Women must not be lactating during the study treatment period and for 3 months after the last dose of study treatment
• Women of childbearing potential must practice effective contraception during the study treatment period and for 3 months after the last dose of study treatment. They must agree to use 2 different means of nonhormonal contraceptive methods
• Women of non-childbearing potential must be either surgically sterile (ie, bilateral tubal ligation at least 3 months prior to dosing) or in menopausal state confirmed as follows: 1 year of spontaneous amenorrhea without an alternative medical cause and a serum FSH level ≥40 mIU/mL
• Men must agree to use contraception (condom with spermicide) during the study treatment period and for at least 3 months after the last dose of study treatment or be surgically sterile (vasectomy at least 3 months prior to dosing)
• Women should not donate eggs and men should not donate sperm during the study treatment period and for at least 3 months after the last dose of study treatment
• Participants must be in good health as determined by Screening medical history, physical examination, vital signs, ECGs, serum chemistry, hematology, virology, and urinalysis performed at Screening and on Day -1

Exclusion Criteria

• History or current chronic lung disease including asthma, chronic obstructive pulmonary disease (COPD), or heavy smoking of >10 pack years
• Previous or current treatment with systemic corticosteroids or any immunosuppressive agents
• Participants who have received a transfusion or any blood products within the last year prior to dosing
• Participants who have made any blood donation or have had a loss of blood of ≥500 mL within 56 days prior to the dose of study drug
• Participants who consume more than 21 units of alcohol per week (1 unit of alcohol equals 1/2 pint of beer, 4 ounces of wine, or 1 ounce of spirits) or those participants who have a significant history of alcoholism or drug/chemical abuse within the last 2 years.
• Participants with positive results on tests for drugs of abuse, cotinine, or alcohol at Screening and/or Day -1.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 45 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

Worldwide Clinical Trials

San Antonio, Texas, United States

Countries

United States

Other Identifiers

DS2325-104

Identifier Type: -

Identifier Source: org_study_id