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NCT07491172PHASE1Recruiting

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

CRISPR Therapeutics AG

Start Date

6/21/2024

Completion Date

6/1/2028

Summary

This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.

Detailed Description

This is a phase 1, open-label, multi-center study of CTX310 in participants with refractory dyslipidemias. Subjects will receive a dose of CTX310 via intravenous (IV) infusion.

Eligibility Criteria

Age Range: 18 years to 75 years

Key Inclusion Criteria: 1. Age of ≥18 and ≤75 years at the time of signing the informed consent. 2. Able to provide written informed consent. 3. Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL in participants with or without ASCVD respectively, or TG ≥500 mg/dL. 4. Refractory to the maximal intensity or MTD of standard of care lines of lipid-lowering therapies available through routine clinical care, for at least 12 weeks prior to screening 5. Female participants must be postmenopausal or surgically sterile. 6. All male participants and their female partners must agree to the use of an acceptable method of effective contraception for the duration of the study. Exclusion Criteria: 1. Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply. 2. Evidence of liver disease, defined as but not limited to: LFTS \>2 × upper limit of normal (ULN), or total bilirubin \>2 × ULN, or INR \>1.5 × ULN, or liver stiffness measured by liver elastography 3. Abnormal or compromised function of kidney, heart, blood or liver. 4. Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1. 5. Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran). 6. Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial. 7. Any prior malignancy within the past 5 years, or current malignancy (exceptions for resected or removed basal cell carcinoma, squamous cell carcinoma in situ and carcinoma in situ of the cervix or breast). 8. Women of childbearing potential. Note: Other protocol defined Inclusion/Exclusion criteria may apply. Note: The inclusion and exclusion criteria listed represent the global protocol. Additional or modified eligibility criteria may apply in certain countries in accordance with local regulatory and ethics committee requirements and the approved country-specific protocol.

Interventions

DRUG

CTX310

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Conditions

CardiovascularMetabolic DiseaseDyslipidemiasLipid DisorderHypertriglyceridemiaHeterozygous Familial Hypercholesterolemia (HeFH)Homozygous Familial Hypercholesterolemia (HoFH)Severe Hypertriglyceridemia (sHTG)Mixed HyperlipemiaHypercholesterolaemia

Locations

Integrity Clinical Research

Hialeah, Florida 33015

United States

Jacksonville Center for Clinical Research

Jacksonville, Florida 32216

United States

Flourish Orlando

Orlando, Florida 32789

United States

United Medical Research

Port Orange, Florida 32127

United States

University of Rochester

Rochester, New York 14642

United States

Duke Early Phase Clinical Research

Durham, North Carolina 27710

United States

Peters Medical Research

High Point, North Carolina 27260

United States

Cleveland Clinic

Cleveland, Ohio 44195

United States

Oregon Health & Science University (OHSU)

Portland, Oregon 97239

United States

Royal Adelaide

Adelaide, 5000

Australia

CORE Research

Brisbane, 4064

Australia

Royal Prince Alfred Hospital

Camperdown, 2050

Australia

Austin Health

Heidelberg, 3084

Australia

Monash Medical Center

Melbourne, 3168

Australia

Aotearoa

Auckland, 2025

New Zealand

NZCR

Christchurch, 8011

New Zealand

Royal Papworth Hospital

Cambridge,

United Kingdom

Barts

London, E1 1BB

United Kingdom

Richmond Pharmacology

London, SE1 1YR

United Kingdom