A trial to evaluate the safety and efficacy of treatment with azacitidine in patients with symptomatic non-obstructive hypertrophic cardiomyopathy

AZacitidine Treatment Effect on hypertrophic Cardiomyopathy: an open-label dose escalation Phase IIa trial followed by a randomised, controlled, open-label Phase IIb efficacy trial

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

ISRCTN

Registry ID

ISRCTN38776134

Enrollment

Registered

2026-02-03

Start date

2026-06-30

Completion date

Unknown

Last updated

2026-05-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM) Circulatory System

Interventions

The investigational medicinal product (IMP) will be azacitidine. Azacitidine is an epigenetic-modifying therapy that irreversibly binds to DNA methyltransferases, thus inhibiting their enzymatic activ

Eligibility

Sex/Gender

All

Age

18 Years to 75 Years

Inclusion criteria

Inclusion criteria: 1. Age 18 to 75 years who have a diagnosis of phenotype-positive HCM consistent with current American and European guidelines; Left ventricle (LV) wall thickness =15 mm (or =13 mm with a family history of HCM). 2. Symptomatic of HCM at screening (defined as being NYHA functional class II/III). 3. Elevated N-terminal pro-hormone of Brain Natriuretic Peptide (NT-proBNP) level =125 pg/ml as measured within 6 months of screening. 4. Left ventricular ejection fraction (LVEF) =55%. 5. Left Ventricular Outflow Tract (LVOT) peak gradient at rest and during Valsalva 30 ml/min/1.73m2 at screening.

Exclusion criteria

Exclusion criteria: 1. Patient has a history of malignancy in the last 5 years (with the exception of non-melanoma skin cancer). 2. Patient is known to be pregnant or lactating. 3. Absolute contraindication to study drug. 4. Patients with reduced baseline blood counts (i.e. White Blood Cells (WBC) 75mmol/mol. 12. Patient has not recovered from an infection that required systemic treatment. 13. Consent to participate is declined.

Countries

Northern Ireland, United Kingdom

Contacts

Public ContactMegan Campbell

AZTEC@nictu.hscni.net+44 (0)28961 51447

Outcome results

None listed

Source: ISRCTN (via WHO ICTRP) · Data processed: May 16, 2026