A Phase 1/2 Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety, Tolerability, and Efficacy of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX340) for In Vivo Editing of the Angiotensinogen (AGT) Gene in Participants With Hypertension
CRISPR Therapeutics AG
Summary
A Study of CTX340 in Participants with Uncontrolled Hypertension.
Description
This is a dose ascending study of CTX340 in participants with hypertension. Subjects will receive CTX340 or placebo via intravenous (IV) infusion.
Eligibility
- Age range
- 18–75 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Age: ≥18 and ≤75 years. 2. Body mass index ≤40 kg/m2. 3. 24-hour mean ambulatory blood pressure monitoring (ABPM) systolic blood pressure (SBP) measurement of ≥130 mm Hg but ≤160 mm Hg despite treatment 4. Be on treatment with ≥4 antihypertensive therapies at effective doses, of which ≥1 must be a diuretic. 5. Participants who at any point had childbearing potential must currently be postmenopausal 6. All participants capable of producing sperm must agree to the use of an acceptable method of effective contraception and their partners with childbearing potential sho…
Interventions
- DrugCTX340
CTX340 is an in vivo gene editing therapy designed to utilize clustered regularly interspaced short palindromic repeats-CRISPR-associated protein 9 (CRISPR-Cas9) to target and disrupt human angiotensinogen (AGT) gene in liver.
- DrugPlacebo
One of the arms in Phase 2 will be placebo.
Locations (2)
- Research Site 1Port Orange, Florida
- Research Site 2Melbourne