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

Kira Pharmacenticals

Status and phase: Completed; Phase 1

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Treatments: Drug: KP104; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT05490017

KP104-101

Details and patient eligibility

About

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).

Enrollment: 80 patients

Sex: All

Ages: 18 to 55 years old

Volunteers: Accepts Healthy Volunteers

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.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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80 participants in 12 patient groups, including a placebo group

Part 1: Single Ascending Dose Cohort 1

Experimental group

Treatment:

Drug: KP104

Part 1: Single Ascending Dose Cohort 2

Experimental group

Treatment:

Drug: KP104

Part 1: Single Ascending Dose Cohort 3

Experimental group

Treatment:

Drug: KP104

Part 1: Single Ascending Dose Cohort 4

Experimental group

Treatment:

Drug: KP104

Part 1: Single Ascending Dose Cohort 5

Experimental group

Treatment:

Drug: KP104

Part 1: Single Ascending Dose Cohort 6

Experimental group

Treatment:

Drug: KP104

Part 1: Single Ascending Dose Cohort 7

Experimental group

Treatment:

Drug: KP104

Part 2: Multiple Ascending Dose Cohort 1

Experimental group

Treatment:

Drug: KP104

Part 2: Multiple Ascending Dose Cohort 2

Experimental group

Treatment:

Drug: KP104

Part 2: Multiple Ascending Dose Cohort 3

Experimental group

Treatment:

Drug: KP104

Part 1: Placebo

Placebo Comparator group

Treatment:

Drug: Placebo

Part 2: Placebo

Placebo Comparator group

Treatment:

Drug: Placebo