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RecruitingPhase 1ACTRN12623000809639

A Safety and Tolerability Study Evaluating CTX310 in Subjects With Refractory Dyslipidemias

A Phase 1 Open-label, Multicenter, First-in-human, Ascending Single-dose Study Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR–Guide RNA–Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Subjects With Refractory Dyslipidemias

Sponsor

CRISPR Therapeutics AG

Enrollment

24 participants

Start Date

Sep 15, 2023

Study Type

Interventional

Conditions

Refractory Dyslipidemias

Summary

This study aims to evaluate the safety and tolerability of a single ascending dose of CTX310 in patients with refractory dyslipidemias and to determine the recommended Phase 2 dose.

Eligibility

Sex: Both males and femalesMin Age: 18 YearssMax Age: 70 Yearss

Inclusion Criteria6

  • Subjects diagnosed with persistent dyslipidemia
  • Subjects must be refractory to the maximum tolerated doses of standard of care lines of treatment
  • Female subjects must be postmenopausal
  • Nonsterile male subjects and their female partners should agree to use an effective method of contraception through at least 12 months after CTX310 infusion
  • Adequate renal, liver and cardiac function
  • Willing to participate in a long-term follow-up study after completion of this study

Exclusion Criteria8

  • No participants with Familial Chylomicronemia Syndrome (FCS)
  • Evidence of liver disease
  • History of alcohol or drug abuse
  • History of a significant coagulation disorder
  • Uncontrolled or untreated thyroid disease
  • Prior treatment with gene therapy/editing product
  • Active HIV, hepatitis B virus or hepatitis C virus infection
  • Any prior or current malignancy or myeloproliferative disorder or a significant immunodeficiency disorder

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Interventions

This is a single-arm, open-label, multicenter, ascending single-dose Phase 1 study. Subjects will receive a single dose of CTX310 via intravenous (IV) infusion. Planned ascending doses levels will be

This is a single-arm, open-label, multicenter, ascending single-dose Phase 1 study. Subjects will receive a single dose of CTX310 via intravenous (IV) infusion. Planned ascending doses levels will be 0.1 mg/kg, 0.3 mg/kg, 0.6 mg/kg, 0.8 mg/kg. Participants will receive only one dose.

Locations(1)

Monash Medical Centre - Clayton campus - Clayton

SA,WA, Australia

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ACTRN12623000809639