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NCT Number: NCT07758634

A Trial Evaluating Safety, Tolerability and Efficacy of CTX340 in Participants With Hypertension

A Study of CTX340 in Participants with Uncontrolled Hypertension.

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Key information

Conditions

Hypertension Cardiovascular Diseases Vascular Diseases

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Research Site 2, Melbourne, Australia

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About this study

This is a dose ascending study of CTX340 in participants with hypertension. Subjects will receive CTX340 or placebo via intravenous (IV) infusion.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Age: ≥18 and ≤75 years.
  • Body mass index ≤40 kg/m2.
  • 24-hour mean ambulatory blood pressure monitoring (ABPM) systolic blood pressure (SBP) measurement of ≥130 mm Hg but ≤160 mm Hg despite treatment
  • Be on treatment with ≥4 antihypertensive therapies at effective doses, of which ≥1 must be a diuretic.
  • Participants who at any point had childbearing potential must currently be postmenopausal
  • 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

  • Serum aldosterone and direct renin concentration (or plasma renin activity [PRA]) suggestive of primary aldosteronism
  • Mean diastolic blood pressure (DBP) ≤65 mm Hg on screening ABPM.
  • Participants with vascular cause of hypertension which may be amendable to revascularization
  • Participants with treatable/reversible causes of uncontrolled hypertension
  • History of renal artery denervation within past 12 months.
  • Orthostatic hypotension
  • Complete blood count (CBC) outside the specified ranges per protocol.
  • Evidence of liver disease
  • History of a significant coagulation disorder.
  • Uncontrolled or untreated thyroid disease

Treatment and study plan

CTX340

Drug

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

Drug

One of the arms in Phase 2 will be placebo.

Primary outcomes

  1. Phase 1: Incidence of dose-limiting toxicities (DLTs)

    Time frame: 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).

  2. Phase 2: Percentage change in circulating angiotensinogen (AGT) concentration from baseline

    Time frame: Through 6 months of follow-up.

    To evaluate the pharmacodynamics (PD) effect of CTX340 at the recommended Phase 2 dose (RP2D)

Secondary outcomes

  1. To assess the safety of CTX340

    Time frame: From CTX340 infusion up to 12 months

    Incidence of adverse events (AEs), including treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs), clinically significant laboratory abnormalities, and clinically significant abnormal vital signs during 12 months of follow-up.

  2. To assess the effect of CTX340 on systolic blood pressure (SBP) by ambulatory blood pressure monitoring (ABPM)

    Time frame: From CTX340 infusion up to 12 months

    Change from baseline in systolic blood pressure (SBP) assessed by 24-hour ambulatory blood pressure monitoring (ABPM)

  3. To assess the pharmacodynamics (PD) effect of CTX340

    Time frame: Over 12 months, compared to baseline

    Percentage change in circulating angiotensinogen (AGT) concentrations over time compared to baseline.

  4. To characterize the pharmacokinetics (PK) of CTX340

    Time frame: From CTX340 infusion up to 12 months

    Plasma levels of LNP (ionizable lipid and PEG lipid)

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Trials

CONTACT

[email protected]

877-214-4634

Sponsors and collaborators

Lead sponsor

CRISPR Therapeutics AG

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2028
Study completion
2029
First posted
Aug 11, 2026
Registry last updated
Aug 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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