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CompletedNCT04842552EHSANUpdated Sep 9, 2026

Effect of Hydralazine on Alzheimer's Disease

A Phase 3 interventional study of Hydralazine hydrochloride 25mg tablets and Placebo in Alzheimer Disease, sponsored by Shahid Sadoughi University of Medical Sciences and Health Services. Completed at 1 site in Iran. Open to participants aged 50 Years and older. Per ClinicalTrials.gov, last updated 2026-09-09.

Sponsored by Shahid Sadoughi University of Medical Sciences and Health Services · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
228
Allocation
Randomized
Ages
50 Years and older
Sex
All
01

Study summary

It has been recently discovered that the FDA-approved drug, hydralazine, has anti-neurodegenerative efficacy based on three intriguing observations. hydralazine; 1) activates the Nrf2 pathway that controls more than 200 antioxidant proteins, 2) rejuvenates mitochondria and increases their respiration capacity and adenosine triphosphate production, 3) activates autophagy which has pathophysiological roles such as intracellular aggregate clearance. There is an emerging agreement that autophagy-lysosome defects occur early in the pathogenesis of Alzheimer's disease (AD). Nrf2 is another pathway known to be impaired in the hippocampus of AD patients who need antioxidant protection the most. Rejuvenation of mitochondria is crucial for fighting AD, as neuronal cells need more energy to afford activation of pathways such as autophagy and Nrf2. The prime objective of this application is to conduct a randomized clinical trial to assess the efficacy of hydralazine in early-stage AD patients who take one of the acetylcholinesterase inhibitor (AChEI) donepezil, rivastigmine, or galantamine.

Read the detailed description

Study aim:

  1. Determination and comparison of the effect of 75mg (25mg TDS) hydralazine vs. placebo in patients with mild to moderate Alzheimer's disease.
  2. Development of an electronic Case Report Form (CRF) and push notification system to remind patients (and/or caregivers) of drug intake to improve drug intake adherence and reduce follow-up losses.
  3. Evaluation of the prognostic accuracy of olfactory tests to predict the changes in cognition and performance of patients with mild to moderate Alzheimer's disease.

Design:

This is a phase III, triple-blind, parallel double-armed randomized clinical trial with an allocation ratio of 1-1 to the intervention and placebo arms. This trial will be conducted on 424 randomly selected patients using random permuted blocks.

Settings and conduct:

All patients who are identified as potentially eligible by the supporting neurologists and psychiatrists will be referred to Adineh Clinic to evaluate their cognitive function, assess for inclusion and exclusion criteria and obtain informed consent. The two arms of the study are hydralazine 75mg (25mg three times per day) or hydralazine placebo. A follow-up evaluation will continue for one year after drug administration. The participants, outcome assessors, researchers, and data analyzers will be blinded to the study arms.

Participants/Inclusion and exclusion criteria:

patients aged 50 and over who are diagnosed with mild to moderate AD will be included in this study; dementia patients with etiologies other than AD (i.e. vascular dementia) will not be included.

Intervention groups:

The two arms of the study are Hydralazine 75mg (25mg three times per day) or Hydralazine placebo.

Main outcome variables:

Various cognitive and function tests for patients and caregivers, olfactory tests, biochemistry as well as drug side effects will be assessed regularly over the period of follow-up.

Treatment was initiated at 37.5 mg/day (12.5 mg three times daily) and titrated to 75 mg/day (25 mg three times daily) over two weeks. Randomization was stratified by age (under 65 versus 65 and older), sex, and baseline MMSE score (12-18 versus 19-26), using permuted blocks of four generated via Sealed Envelope and integrated into the study eCRF. Independent oversight was provided by a Data Monitoring Committee and a Data and Safety Monitoring Committee, and by the Clinical Trial Centres of Kermanshah and Yazd Universities of Medical Sciences, under the NIMAD ethics committee. Serum hydralazine concentrations were measured at months 6 and 12 to assess adherence. Adherence was supported by an electronic Case Report Form with SMS reminders and participant logbooks. Screening included the Schedule for Affective Disorders and Schizophrenia (SADS) and electrocardiography (ECG).

02

Conditions studied

  • Alzheimer Disease

Keywords

  • Alzheimer Disease
  • Hydralazine
  • Early stage
  • Olfactory sense
03

Who can participate

Ages eligible
50 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Diagnoses of Alzheimer's disease according to the National Institute of Neurological and Communicative Disorders and Stroke and the Alzheimer's Disease and Related Disorders Association (NINCDS-ADRDA) criteria.
  • Presence of a caregiver (friend or relative) who can assume responsibility for medication administrations, accompany the patient to all visits, and rate patient's condition.
  • Written informed consent form from both the patient (or surrogate) and caregiver.
  • A Mini-Mental State Examination score between 12 and 26 inclusive.
  • Prescription of donepezil (5-10mg/d), memantine (5-21mg/d), rivastigmine (3-6mg/d), galantamine or galantamine ER (8-16mg/d) for a minimum of 4 weeks prior to randomization.
  • Agreement not to take hydralazine.
  • Age 50 and over.

Exclusion criteria

Exclusion Criteria:

  • Non-Alzheimer primary dementia diagnosis (e.g., vascular dementia, Lewy body dementia, frontotemporal dementia, vitamin B-12 deficiency, hypothyroidism).
  • Diagnosis of any of the following conditions; major depression, delirium, alcohol or psychoactive substance abuse or dependency, schizophrenia, or delusional disorder as defined by Diagnostic and Statistical Manual (DSM)-5.
  • Diagnosis of systemic illnesses that would interfere with participation in the study or decrease the life expectancy to less than one year.
  • Currently being treated with hydralazine or a history of intolerance to oral therapy with hydralazine
  • Any intravenous treatment for heart failure, except IV furosemide (e.g. IV inotropes, pressors, nitrates or nesiritide) at the time of screening.
  • Systolic blood pressure \<100 mmHg, reversible etiology of acute heart failure such as myocarditis, acute myocardial infarction-over the past 4 weeks, arrhythmia and existence of pacing device (Acute myocardial infarction is defined as symptoms and major electrocardiogram (ECG) changes (i.e., ST segment elevations), and arrhythmia includes unstable heart rates above 120/min or below 50/min).
  • Existence of severe congenital heart disease (such as uncorrected tetralogy of fallot or transposition of the aorta) and severe aortic or mitral stenosis or severe rheumatic mitral regurgitation.
  • Concurrent use of phosphodiesterase type 5 (PDE5) inhibitors (e.g. Viagra, Etc.)
  • Cardiac revascularization within the last 3 months or likelihood of requiring coronary revascularization within the study period. eGFR (Glomerular Filtration Rate) \< 15ml/min/1.73m2, or on regular dialysis, or planned dialysis within the study period.
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Investigator, Outcomes assessor)
Enrollment
228 participants (actual)

Study arms

  • Active comparator
    Hydralazine hydrochloride 25mg

    Hydralazine hydrochloride (25mg tablets) every eight hours (TDS)

    Drug: Hydralazine hydrochloride 25mg tablets

  • Placebo comparator
    Placebo

    Placebo tablets (identical in shape to the active comparator) every eight hours (TDS)

    Drug: Placebo

Interventions

  • DrugHydralazine hydrochloride 25mg tablets

    Hydralazine hydrochloride 25mg tablets three time daily for 365 days (one year)

  • DrugPlacebo

    Placebo

05

What researchers measure

Primary outcomes

  1. Change in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) Score from Baseline

    Change from baseline to Month 12 in the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) total score, hydralazine compared with placebo. The ADAS-Cog total score ranges from 0 to 70; higher scores indicate greater cognitive impairment. The primary endpoint is analyzed using a mixed-effects model for repeated measures (MMRM) across assessments at 3, 6, 9, and 12 months.

    Time frame: Baseline, and Months 3, 6, 9, and 12

Secondary outcomes

  1. Change in Lawton Instrumental Activities of Daily Living (IADL) Scale Score from Baseline

    Changes in the function of patients with Alzheimer's disease in the hydralazine-treated group compared to placebo- treated using Lawton Activity of Daily Living Scale. Scores range from 0 to 8; higher scores indicate greater independence.

    Time frame: Baseline, and Months 3, 6, 9, and 12

  2. Change in Neuropsychiatric Inventory (NPI) Score from Baseline

    Changes in the behaviour of the patients with Alzheimer's disease in the hydralazine-treated group compared to placebo-treated using Neuropsychiatric Inventory

    Time frame: Baseline, and Months 3, 6, 9, and 12

  3. Change in Caregiver Activity Scale Score from Baseline

    Changes in caregiver's spent time for the patients in the hydralazine-treated group compare to placebo using Caregiver Activity Scale. Measures caregiver time (hours) over the prior 24 hours across six activities; higher values indicate greater caregiver time.

    Time frame: Baseline, and Months 3, 6, 9, and 12

06

Study locations

1 site
  • Adineh Health Centre
    Yazd, Yazd Province 8916713151, Iran
07

References and documents

Publications

  • Mirzaei M, Ahmadi N, Bagheri Fahraji B, Ardekani AM, Rahimdel A, Soltani MH, Ardekani SMY, Bidaki R, Kasnavie FH, Dastjerdi G, Aboutorabi M, Mirzaei H. A randomized clinical trial evaluating Hydralazine's efficacy in early-stage Alzheimer's disease: The EHSAN Study. Sci Rep. 2024 Nov 21;14(1):28837. doi: 10.1038/s41598-024-79616-4. PubMed 39572624 ↗

Study documents

  • Study protocol · Nov 20, 2024

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Individual patient data (IPD) sharing plan will be decided according to the upcoming requests and the ethics committee approval.

Supporting information: Study protocol, Sap, Icf, Csr

08

Registry details

Key details

Study ID
NCT04842552
Lead sponsor
Shahid Sadoughi University of Medical Sciences and Health Services
Collaborators
National Institute for Medical Research and Development
Responsible party
Masoud Mirzaei (Professor, Shahid Sadoughi University of Medical Sciences and Health Services) — Principal investigator
First posted
Apr 13, 2021
Start date
Aug 2, 2021
Primary completion
Mar 5, 2026
Completion
Mar 5, 2026
Last update
Sep 9, 2026

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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