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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06677307




Registration number
NCT06677307
Ethics application status
Date submitted
4/11/2024
Date registered
6/11/2024

Titles & IDs
Public title
A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110
Scientific title
A Phase 1/2a, Single- and Multiple-dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of KRRO 110 in Healthy Adult Volunteers and in Adult Participants with Alpha-1 Antitrypsin Deficiency (AATD) (REWRITE)
Secondary ID [1] 0 0
KRRO-110-001
Universal Trial Number (UTN)
Trial acronym
REWRITE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Alpha-1 Anti-trypsin Deficiency 0 0
AATD 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Respiratory 0 0 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - KRRO-110

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

Placebo comparator: Arm 2: Placebo (Part A only) - Placebo, IV administration


Treatment: Drugs: KRRO-110
KRRO-110 drug product, IV
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and Tolerability
Timepoint [1] 0 0
From Day 1 to Day 43 in the SAD and from Day 1 to Day 85 in the MAD
Secondary outcome [1] 0 0
Pharmacokinetics (PK) of KRRO-110
Timepoint [1] 0 0
Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD)
Secondary outcome [2] 0 0
Pharmacodynamics (PD) of KRRO-110
Timepoint [2] 0 0
Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD)
Secondary outcome [3] 0 0
Pharmacokinetics (PK) of KRRO-110
Timepoint [3] 0 0
Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD)

Eligibility
Key inclusion criteria
Part A SAD cohort Inclusion Criteria (Healthy Volunteers)

* 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

PiZZ Genotype (Part A SAD and Part B MAD cohorts) Inclusion Criteria (PiZZ Genotype)

* Adult male or female participants 18 to 70 years of age (inclusive)
* Documented PiZZ genotype
* Baseline blood total AAT level < 11 µM/L
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Exclusion Criteria (Healthy Volunteers)

* 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

Exclusion Criteria (PiZZ Genotype)

* 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 < 50%
* 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
* Serology result consistent with exposure to HIV, or serology consistent with active hepatitis B or hepatitis C infection

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/01/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2026
Actual
Sample size
Target
64
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Nucleus Network Pty Ltd - Melbourne
Recruitment postcode(s) [1] 0 0
3004 - Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Korro Bio, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Mathias Leinders, PhD
Address 0 0
Korro Bio, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Study Director
Address 0 0
Country 0 0
Phone 0 0
857-529-5757
Fax 0 0
Email 0 0
clinicaltrials@korrobio.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06677307