To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104

NCT ID: NCT05490017

Last Updated: 2023-03-01

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 80 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-12-30
• Study Completion Date: 2022-09-02

Brief Summary

The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in healthy volunteers. The study will be conducted in 2 parts: Part 1, the single ascending dose (SAD) is the first in human (FIH) study of KP104 and Part 2, multiple ascending dose (MAD).

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Study Design

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

Study Groups

Part 1: Single Ascending Dose Cohort 1

Group Type: EXPERIMENTAL

KP104

Intervention Type: DRUG

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Part 1: Single Ascending Dose Cohort 2

Group Type: EXPERIMENTAL

KP104

Intervention Type: DRUG

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Part 1: Single Ascending Dose Cohort 3

Group Type: EXPERIMENTAL

KP104

Intervention Type: DRUG

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Part 1: Single Ascending Dose Cohort 4

Group Type: EXPERIMENTAL

KP104

Intervention Type: DRUG

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Part 1: Single Ascending Dose Cohort 5

Group Type: EXPERIMENTAL

KP104

Intervention Type: DRUG

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Part 1: Single Ascending Dose Cohort 6

Group Type: EXPERIMENTAL

KP104

Intervention Type: DRUG

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Part 1: Single Ascending Dose Cohort 7

Group Type: EXPERIMENTAL

KP104

Intervention Type: DRUG

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Part 2: Multiple Ascending Dose Cohort 1

Group Type: EXPERIMENTAL

KP104

Intervention Type: DRUG

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Part 2: Multiple Ascending Dose Cohort 2

Group Type: EXPERIMENTAL

KP104

Intervention Type: DRUG

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Part 2: Multiple Ascending Dose Cohort 3

Group Type: EXPERIMENTAL

KP104

Intervention Type: DRUG

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Part 1: Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Participants will receive matching placebo which is KP104 vehicle containing sodium phosphate, sodium chloride, and L-Lysine Hydrochloride (L-Lys-HCL).

Part 2: Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Participants will receive matching placebo which is KP104 vehicle containing sodium phosphate, sodium chloride, and L-Lysine Hydrochloride (L-Lys-HCL).

Interventions

KP104

Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.

Intervention Type: DRUG

Placebo

Participants will receive matching placebo which is KP104 vehicle containing sodium phosphate, sodium chloride, and L-Lysine Hydrochloride (L-Lys-HCL).

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Weight of > 40 kilograms (kg) and < 120 kg at Screening.
• In good general health, determined by no clinically significant findings in the opinion of the Investigator from medical history, physical examination, 12-lead ECG, clinical laboratory findings, and vital signs at Screening and Check-in.
• Hemoglobin, hematocrit, white blood cell count, absolute neutrophil count, and platelet count results within the normal range at the Screening Visit; participants with Gilbert's disease with associated abnormalities of liver function tests are eligible for enrollment. Tests may be repeated at the discretion of the Investigator to confirm abnormalities.
• Creatinine clearance based on the Cockcroft-Gault equation of >= 80 milliliters per minute (ml/min).
• Females of childbearing potential and males must practice effective contraception from Screening until 28 days after the end of study (EOS) visit.
• Females of childbearing potential must have a negative pregnancy test at Screening and within 24 hours prior to dosing of study drug; for post-menopausal subjects, a blood sample will also be tested for follicle stimulating hormone to confirm post-menopausal status.

Exclusion Criteria

• Any clinically significant underlying illness in the opinion of the Investigator.
• Any history or sign of significant chronic active or recurrent infection, or screening laboratory evidence consistent with a significant chronic active or recurrent infection requiring treatment with antibacterials, antivirals, or antifungals.
• Treatment of any infection with IV (within 30 days of Screening) or oral (within 14 days of Screening) antibacterials, antivirals, or antifungals.
• History of clinically significant hematologic or bone marrow disease or blood dyscrasias.
• History of meningococcal infection.
• History of tuberculosis.
• History of asplenia (functional or anatomical).
• Prior exposure to KP104.
• Known allergy to penicillin antibiotics or history of allergy or contraindication to required prophylactic antibiotic therapy to be used during the study.
• Known or suspected complement deficiency during screening.
• Positive serology for Hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) at Screening.
• History of drug or alcohol abuse within 1 year of Screening in the opinion of the investigator, or a positive test for drugs of abuse or alcohol at Screening or Check-in.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Kira Pharmacenticals (US), LLC.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

CMAX Clinical Research

Adelaide, , Australia

Countries

Australia

Other Identifiers

KP104-101

Identifier Type: -

Identifier Source: org_study_id