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

NCT07491172

RecruitingPhase 1

Sponsor: CRISPR Therapeutics AG

Conditions: Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia

Interventions: CTX310

Countries: United States, Australia, New Zealand, United Kingdom

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.

Eligibility overview

Sex: ALL

Age: 18 Years to 75 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
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.
Locations (17)
  • Hialeah, Florida, United States
  • Jacksonville, Florida, United States
  • Orlando, Florida, United States
  • Port Orange, Florida, United States
  • Rochester, New York, United States
  • Durham, North Carolina, United States
  • High Point, North Carolina, United States
  • Portland, Oregon, United States
  • Adelaide, Australia
  • Brisbane, Australia
  • Camperdown, Australia
  • Heidelberg, Australia
  • Melbourne, Australia
  • Auckland, New Zealand
  • Christchurch, New Zealand
  • Cambridge, United Kingdom
  • London, United Kingdom