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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CTX310 | Experimental | Subjects will receive an intravenous (IV) infusion. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CTX310 | Drug | CTX310 is a lipid nanoparticle (LNP) formulation of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) components for in vivo editing of the target gene angiopoietin-like 3 (ANGPTL3). |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments | Incidence of dose-limiting toxicities and frequency of adverse events | From CTX310 infusion up to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments | Percentage change from baseline in lipid values (LDL-C, non-HDL-C, Trigs, apoB and HDL-C) | Over 12 months, compared to baseline |
| To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments |
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Key Inclusion Criteria:
Exclusion Criteria:
Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.
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
Abnormal or compromised function of kidney, heart, blood or liver.
Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.
Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).
Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial.
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).
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.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trials | Contact | 877-214-4634 | medicalaffairs@crisprtx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site 10 | Not yet recruiting | Jacksonville | Florida | 32216 | United States | |
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Frequency and severity of adverse events (AE), including treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs), clinically significant laboratory abnormalities, and clinically significant abnormal vital signs. |
| From CTX310 infusion up to 12 months |
| To assess the pharmacokinetics (PK) of CTX310 in adult participants with dyslipidemias that are refractory to available treatments | Levels of CTX310 in blood over time | From CTX310 infusion up to 12 months |
| To assess the pharmacodynamic (PD) response of CTX310 in adult participants with dyslipidemias that are refractory to available treatments | Percentage change from baseline of ANGPTL3 | Over 12 months, compared to baseline |
| Research Site 17 |
| Recruiting |
| Orlando |
| Florida |
| 32789 |
| United States |
| Research Site 11 | Recruiting | Port Orange | Florida | 32127 | United States |
| Research Site 14 | Not yet recruiting | Durham | North Carolina | 27710 | United States |
| Research Site 13 | Recruiting | High Point | North Carolina | 27260 | United States |
| Research Site 9 | Not yet recruiting | Cleveland | Ohio | 44195 | United States |
| Research Site 12 | Not yet recruiting | Portland | Oregon | 97239 | United States |
| Research Site 1 | Recruiting | Adelaide | 5000 | Australia |
| Research Site 15 | Not yet recruiting | Brisbane | 4064 | Australia |
| Research Site 16 | Not yet recruiting | Brisbane | Australia |
| Research Site 7 | Not yet recruiting | Camperdown | 2050 | Australia |
| Research Site 3 | Recruiting | Heidelberg | 3084 | Australia |
| Research Site 2 | Recruiting | Melbourne | 3168 | Australia |
| Research Site 5 | Recruiting | Auckland | 2025 | New Zealand |
| Research Site 4 | Recruiting | Christchurch | 8011 | New Zealand |
| Research Site 18 | Not yet recruiting | Cambridge | United Kingdom |
| Research Site 8 | Not yet recruiting | London | E1 1BB | United Kingdom |
| Research Site 6 | Recruiting | London | SE1 1YR | United Kingdom |
| ID | Term |
|---|---|
| D008659 | Metabolic Diseases |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D015228 | Hypertriglyceridemia |
| D000090542 | Homozygous Familial Hypercholesterolemia |
| D006954 | Hyperlipoproteinemia Type V |
| D006937 | Hypercholesterolemia |
| ID | Term |
|---|---|
| D009750 | Nutritional and Metabolic Diseases |
| D006949 | Hyperlipidemias |
| D006938 | Hyperlipoproteinemia Type II |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006951 | Hyperlipoproteinemias |
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