Single and multiple ascending dose study of oral LAD765 (formerly ZKN-0013) or placebo given randomly to healthy volunteers to evaluate the safety, Tolerability and the amount of drug in the body after drug administration.

A Combined Single and Multiple Ascending Dose (SAD-MAD) Phase 1, Randomized, Double-Blinded, Placebo-Controlled, Multiple Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Oral LAD765 (formerly ZKN-0013) in Healthy Subjects. Prior to May 2025, 16 subjects were enrolled.

Status

Recruiting

Phases

Phase 1

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12624000632594

Enrollment

Registered

2024-05-16

Start date

2024-07-04

Completion date

2026-10-01

Last updated

2026-05-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

Almirall S.A. has acquired a new product which works on mutated gene and allows for production of the correct protein instead of the defective protein typical to a number of hereditary diseases. The product is called LAD765 and it is taken orally. The purpose of this research is to determine whether LAD765 is safe and well tolerated in humans. In addition, the study purpose is to learn about the study drug properties. The participants will take one or multiple doses of LAD765 during their participation based on the group they will be assigned to.

Interventions

ZKN-0013 is a modified macrolide SAD cohorts: • Cohort 1: ZKN-0013 15 mg or placebo orally once • Cohort 2: ZKN-0013 50 mg or placebo orally once • Cohorts 3 up to 7: dose levels to be determined based on Human PK modeling after SAD Cohorts 1 and 2 MAD cohorts • Up to 7 cohorts: ZKN-0013 or placebo orally once daily for 15 days. Dose levels to be determined based on Human PK modeling after SAD Cohorts 1 and 2 Both ZKN-0013 and placebo will be administered as oral capsules. Subjects will be randomized to receive single and then multiple doses of ZKN-0013 or placebo at a ratio of 3:1 in each cohort: Six subjects will receive ZKN-0013 and two will receive placebo. Study drug administration will be recorded in each subject file to ensure compliance.

LAD765 is a modified macrolide SAD cohorts: • Cohort 1: LAD765 15 mg or placebo orally once • Cohort 2: LAD765 50 mg or placebo orally once • Cohorts 3 up to 7: dose levels to be determined based on Human PK modeling after each cohort. The dose levels planned for this study range from 15 to 1,500 mg MAD cohorts • Up to 7 cohorts: LAD765 or placebo orally once daily for 15 days. Dose levels to be determined based on Human PK modeling after SAD Cohorts Both LAD765 and placebo will be administered as oral capsules. Subjects will be randomized to receive single and then multiple doses of LAD765 or placebo at a ratio of 3:1 in each cohort: Six subjects will receive LAD765 and two will receive placebo. Study drug administration will be recorded in each subject file to ensure compliance. Prior to May 2025, 16 subjects were enrolled.

Study design

Allocation

Randomised controlled trial

Primary purpose

Treatment

Eligibility

Sex/Gender

All

Age

18 Years to 55 Years

Healthy volunteers

Yes

Inclusion criteria

- Healthy females and males ages 18-55 inclusive - Contraception requirement for woman of child bearing potential and males with female partners of child bearing potential - Not using prescription medication 14 days prior to admission; and 7 days prior to admission for over-the-counter (OTC) medications/vitamins/supplements (Exceptions contraception, Acetaminophen equal to 2g/day, stand dose of vitamins) - Non-smoking and no use of any tobacco or nicotine products (by declaration) for a period of at least 1 months prior to screening visit - Be on no medication with potential to impair hepatic and renal function at the time of screening - Normal hepatic enzymes and bilirubin - Normal renal function (glomerular filtration rate greater than 60 mL/min/1.73m2) - Negative human immunodeficiency virus (HIV) or Hepatitis B surface antigen (HBsAg) or Hepatitis C (HCV) Ab by serology - No history of alcohol or other drugs of abuse - Body Mass Index (BMI) of 18.0 to 32.0 kg/m2 (inclusive) - No vaccines given within 14 days of dosing

Exclusion criteria

- Dosed in another clinical trial within at least 10 tissue half-lives prior to dosing - Evidence or history of clinically relevant hepatic, hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease - History of regular alcohol consumption exceeding 14 drinks/week for females or 21 drinks/week for males within 6 months of screening - Screening supine BP equal to 140 mm Hg (systolic) or equal to 90 mm Hg (diastolic) - Screening supine (for 5 minutes) 12-lead electrocardiogram (ECG) demonstrating QTc greater than 450 millisecond for men and greater than 470 millisecond for women, or a QRS interval >120 millisecond - Subjects with any abnormalities in clinical laboratory tests at screening, considered by the study physician as clinically significant - Pregnant or breastfeeding female subjects - Subjects who donated blood or received blood or plasma derivatives in 30 days preceding study drug administration - Subjects with any acute medical situation (e.g., acute infection) within 48 hours (h) of - Screening or start of dosing - Major surgery within last 3 months, or minor surgery in the last 1 month, or any planned surgery during the trial. - Any other condition or prior therapy that in the opinion of the study physician or designee would make the participant unsuitable for this study

Outcome results