A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110

NCT06677307 | Terminated Phase 1 DMC

• 1 other identifier: KRRO-110-001
• Study Type: interventional
• Target: 42
• Locations: 2 countries
• Sites: 5

Brief Summary

The purpose of this first-in human (FIH) study is to assess the safety, tolerability, and pharmacokinetics (PK) of single and multiple doses of KRRO-110 in both healthy adult participants and in clinically stable patients with Alpha-1 antitrypsin deficiency (AATD).

Conditions

Alpha-1 Anti-trypsin Deficiency; AATD

Outcome Measures

Primary Outcomes (1)

• Safety and Tolerability

  Type, frequency, and severity of treatment-emergent adverse events (TEAEs)

  From Day 1 to Day 43 in the SAD and from Day 1 to Day 85 in the MAD

Secondary Outcomes (3)

• Pharmacokinetics (PK) of KRRO-110

  Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD)

• Pharmacodynamics (PD) of KRRO-110

  Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD)

• Pharmacokinetics (PK) of KRRO-110

  Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD)

Study Arms (2)

Arm 1: KRRO-110 (Part A and Part B)

EXPERIMENTAL

KRRO-110 is an RNA editing oligonucleotide encapsulated in a lipid nanoparticle (LNP) administered by intravenous (IV) infusion as a single dose in Part A (SAD), multi-dose in Part B (MAD).

Drug: KRRO-110

Arm 2: Placebo (Part A only)

PLACEBO COMPARATOR

Placebo, IV administration

Drug: KRRO-110

Interventions

KRRO-110 DRUG

KRRO-110 drug product, IV

Arm 1: KRRO-110 (Part A and Part B); Arm 2: Placebo (Part A only)

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult male or female participants, 18 to 65 years of age
• Documented PiMM genotype
• Participants who are willing to and able to provide signed written informed consent
• Adult male or female participants 18 to 70 years of age (inclusive)
• Documented PiZZ genotype
• Baseline blood total AAT level \< 11 µM/L

You may not qualify if:

• Female participant of childbearing potential or male participant that is unable or unwilling to use an approved, reliable means of contraception
• Body mass index (BMI) \> 32 or \< 18.5 kg/m2
• History or current clinical evidence of hepatic disease
• Evidence of active infection
• History of medical condition(s), eg, advance cardiac disease, current or recent malignancy, organ transplantation, or other illness
• Serology result indicative of any exposure (past or active) to hepatitis C, hepatitis B, or human immunodeficiency virus (HIV)
• Respiratory or other acute illness within 8 weeks
• Tobacco use of any kind within 6 months
• Female participant of childbearing potential or male participant that is unable or unwilling to use an approved, reliable means of contraception
• BMI \> 32 or \< 18.5 kg/m2 or weight \> 90 kg
• History of FEV1 \< 35%
• History or current clinical evidence of advanced hepatic disease and/or pulmonary disease
• Use of an experimental therapy except KRRO-110 within 6 months or 5 half-lives for the experimental therapy, whichever is greater
• Tobacco use of any kind within 6 months
• Use of conventionally dosed AAT augmentation therapy within 5 half-lives

Sponsors & Collaborators

• Korro Bio, Inc.: lead

Study Sites (5)

The Prince Charles Hospital

Brisbane, Queensland, 4032, Australia

Location

Nucleus Network Pty Ltd

Melbourne, Victoria, 3004, Australia

Location

St. Vincent's Hospital Melbourne

Melbourne, Victoria, 3065, Australia

Location

New Zealand Clinical Research

Auckland, New Zealand

Location

New Zealand Clinical Research

Christchurch, New Zealand

Location

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Study Director

  Korro Bio, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 4, 2024
• First Posted: November 6, 2024
• Study Start: January 13, 2025
• Primary Completion: October 31, 2025
• Study Completion: December 15, 2025
• Last Updated: April 17, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3); NZ (2)