None listed
Conditions
Brief 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.
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 0.1 mg/kg, 0.3 mg/kg, 0.6 mg/kg, 0.8 mg/kg. Participants will receive only one dose.
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 dose 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 in each phase. The study will consist of 2 Phases: Phase 1a and Phase 1b. Phase 1a (15 participants) will be the dose escalation portion and Phase 1b (up to 69 participants) will consist of four disease-specific cohor
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 dose 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 in each phase. The study will consist of 2 Phases: Phase 1a and Phase 1b. Phase 1a (15 participants) will be the dose escalation portion and Phase 1b (up to 69 participants) will consist of four disease-specific cohort expansions.
Sponsors
CRISPR Therapeutics AG
Study design
Allocation
Non-randomised trial
Primary purpose
Treatment
Eligibility
Sex/Gender
All
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Subjects diagnosed with persistent dyslipidemia 2. Subjects must be refractory to the maximum tolerated doses of standard of care lines of treatment 3. Female subjects must be postmenopausal 4. Nonsterile male subjects and their female partners should agree to use an effective method of contraception through at least 12 months after CTX310 infusion 5. Adequate renal, liver and cardiac function 6. Willing to participate in a long-term follow-up study after completion of this study
Exclusion criteria
1. No participants with Familial Chylomicronemia Syndrome (FCS) 2. Evidence of liver disease 3. History of alcohol or drug abuse 4. History of a significant coagulation disorder 5. Uncontrolled or untreated thyroid disease 6. Prior treatment with gene therapy/editing product 7. Active HIV, hepatitis B virus or hepatitis C virus infection 8. Any prior or current malignancy or myeloproliferative disorder or a significant immunodeficiency disorder
Outcome results
None listed