CTX340: 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.
Placebo: One of the arms in Phase 2 will be placebo.
Study summary
A Study of CTX340 in Participants with Uncontrolled Hypertension.
Eligibility
Sex
ALL
Min age
18 Years
Max age
75 Years
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 should also agree to use an effective method of contraception.
Exclusion Criteria:
1. Serum aldosterone and direct renin concentration (or plasma renin activity \[PRA\]) suggestive of primary aldosteronism
2. Mean diastolic blood pressure (DBP) ≤65 mm Hg on screening ABPM.
3. Participants with vascular cause of hypertension which may be amendable to revascularization
4. Participants with treatable/reversible causes of uncontrolled hypertension
5. History of renal artery denervation within past 12 months.
6. Orthostatic hypotension
7. Complete blood count (CBC) outside the specified ranges per protocol.
8. Evidence of liver disease
9. History of a significant coagulation disorder.
10. Uncontrolled or untreated thyroid disease
Primary outcome measure(s)
Phase 1: Incidence of dose-limiting toxicities (DLTs) — Up to 12 months To evaluate the safety and tolerability of a single ascending dose of CTX340 in participants with hypertension to determine the recommended Phase 2 dose (RP2D).
Phase 2: Percentage change in circulating angiotensinogen (AGT) concentration from baseline — Through 6 months of follow-up. To evaluate the pharmacodynamics (PD) effect of CTX340 at the recommended Phase 2 dose (RP2D)
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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