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Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD

ConditionsUrea Cycle Disorders, InbornUrea Cycle DisordersOrnithine Transcarbamylase (OTC) DeficiencyArginase 1 DeficiencyNAGS DeficiencyCarbamylphosphate Synthetase IOrnithine Transporter Mitochondrial 1Argininosuccinate SynthaseArgininosuccinate Lyase DeficiencyN-acetyl Glutamate - SynthetaseCitrin Deficiency
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorKorro Bio, Inc.

About this trial

Phase 1/2 Study of KRRO-121 is a randomized, placebo-controlled study in Healthy Volunteers (Phase 1 SAD and MAD) and Study Participants with Urea Cycle Disorders (Phase 2). The primary objective of the study is to evaluate the safety of KRRO-121.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Adult male or female participants, 18 to 65 years of age

Healthy as determined by medical history, physical examination, vital signs, 12-lead electrocardiogram (ECG), and screening laboratory assessments, in the opinion of the Investigator.

Female participant of childbearing potential or male participant that is willing to use an approved, reliable means of contraception

Willing and able to provide signed written informed consent and to comply with all study requirements

Disqualifiers

Any medical history that is clinically significant in the opinion of the Principal Investigator and Sponsor Medical Monitor

Any laboratory value at screening or predose on Day -1 outside the local laboratory reference range that is clinically significant in the opinion of the Principal Investigator and Sponsor Medical Monitor

Evidence of active systemic infection at Screening or Day 1 requiring antimicrobial, antiviral, or antifungal therapy

Use of any investigational drug or device within 3 months or 5 half-lives (whichever is longer) prior to Day 1

Trial design

Design model

Sequential

Treatments tested in this trial

  • KRRO-121

    Drug

    KRRO-121, subcutaneous injection

  • Placebo

    Drug

    Placebo, subcutaneous injection

Treatment groups

30 Participants
are divided into 2 treatment groups
Group A: KRRO-121Experimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of dose limiting toxicities (DLT) (Safety and Tolerability)

General toxicity: Any Grade 3 or higher adverse event (AE) per Common Terminology Criteria for Adverse Events (CTCAE); Serious adverse events: Any serious adverse event (SAE) assessed as related or possibly related to KRRO-121.

Time frame
Day 1 through 14 days post-dose
2

Frequency and severity of treatment-emergent adverse events (TEAEs; per Common Terminology Criteria for Adverse Events [CTCAE]) (Safety and Tolerability)

Any adverse event (AE) per Common Terminology Criteria for Adverse Events (CTCAE),

Time frame
Day 1 through Day 50

Secondary outcomes

1

Maximum blood concentration (Cmax)

Maximum blood concentration (Cmax) of KRRO-121

Time frame
Day 1 to Day 50

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Korro Bio, Inc.

Lead sponsor

Source ClinicalTrials.gov
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