Phase 3
N=598
Safety and Efficacy of TRx0237 in Subjects With Alzheimer's Disease Followed by Open-Label Treatment
Alzheimer Disease
Bottom Line
View on ClinicalTrials.gov: NCT03446001 ↗Enrolled (actual)
598
Serious AEs
6.5%
Results posted
Sep 2025
Primary outcome: Primary: Change From Baseline on Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-cog11) (16 mg/Day vs Control) — 1.71; 1.34 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- TRx0237 16 mg/day (Drug); Control (Drug); TRx0237 8 mg/day (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- TauRx Therapeutics Ltd
- Primary completion
- Mar 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline on Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-cog11) (16 mg/Day vs Control) |
1.71; 1.34 | — |
| PRIMARY Change From Baseline on Alzheimer's Disease Cooperative Study - Activities of Daily Living (ADCS-ADL23) (16 mg/Day vs Control) |
-0.77; -0.62 | — |
| PRIMARY Number of Study Participants With Serious and Non-serious Adverse Events (16 mg/Day vs Control) |
17; 18; 146; 131 | — |
| SECONDARY Change in Annualized Rate of Whole Brain Atrophy (16 mg/Day vs Control) |
-11137.44; -11162.70 | — |
| SECONDARY Change in Standardized Uptake Value Ratio (SUVR) Based on Temporal Lobe 18F-fluorodeoxyglucose Positron Emission Tomography (18F-FDG-PET) (16 mg/Day vs Control) |
-0.025; -0.026 | — |
| SECONDARY Change in Annualized Rate of Temporoparietal Lobe Atrophy (16 mg/Day vs Control) |
-740.79; -711.14 | — |
| SECONDARY Change From Baseline on Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-cog11) (8 mg/Day vs Control) |
1.78; 1.06 | — |
| SECONDARY Change From Baseline on Alzheimer's Disease Cooperative Study - Activities of Daily Living (ADCS-ADL23) (8 mg/Day vs Control) |
-0.82; -1.41 | — |
| SECONDARY Change in Standardized Uptake Value Ratio (SUVR) Based on Temporal Lobe 18F-fluorodeoxyglucose Positron Emission Tomography (18F-FDG-PET) (8 mg/Day vs Control) |
-0.027; -0.022 | — |
| SECONDARY Change in Annualized Rate of Temporoparietal Lobe Atrophy (8 mg/Day vs Control) |
-729.55; -651.28 | — |
| SECONDARY Change From Open-Label Baseline on Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-cog11) |
2.98; 3.58 | — |
| SECONDARY Number of Study Participants With Serious and Non-serious Adverse Events (8 mg/Day vs Control) |
17; 6; 146; 47 | — |
| SECONDARY Number of Study Participants With Serious and Non-serious Adverse Events (Open-label) |
14; 13; 88; 101 | — |
Summary
The purpose of this study is to determine the safety and efficacy of TRx0237 16 mg/day and 8 mg/day in the treatment of subjects with Alzheimer's Disease compared to placebo. In addition, an open-label, delayed-start phase is included to demonstrate a disease-modifying effect of TRx0237.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of Alzheimer's Disease (AD), encompassing probable AD and mild cognitive impairment due to AD (MCI-AD) based on the 2011 National Institute on Aging and Alzheimer's Association (NIA/AA) criteria
- Documented PET scan that is positive for amyloid
- Mini-Mental State Examination (MMSE) score of 16-27 (inclusive), subject to stratification requirements
- Global Clinical Dementia Rating (CDR) of 0.5 to 2 (if 0.5, including a score of >0 in one of the functional domains: Community Affairs, Home and Hobbies, or Personal Care)
- Age 6 months prior to Screening is considered acceptable)
- Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition criteria met for major depressive disorder; schizophrenia; other psychotic disorders, bipolar disorder; substance (including alcohol) related disorders
- Metal implants in the head, pacemaker, cochlear implants, or any other non-removable items that are contraindications to MRI
- Resides in hospital or moderate to high dependency continuous care facility
- Any physical disability that would prevent completion of study procedures or assessments
- History of swallowing difficulties
- Pregnant or breastfeeding
- Glucose-6-phosphate dehydrogenase (G6PD) deficiency
- History of significant hematological abnormality or current acute or chronic clinically significant abnormality
- Abnormal serum chemistry laboratory value at Screening deemed to be clinically significant by the Investigator
- Clinically significant cardiovascular disease or abnormal electrocardiogram assessments
- Pre-existing or current signs or symptoms of respiratory failure
- Concurrent acute or chronic clinically significant immunologic, hepatobiliary, or endocrine disease and/or other unstable or major disease other than probable AD or MCI-AD
- Diagnosis of cancer (excluding basal cell carcinoma, squamous cell carcinoma, or prostate carcinoma in situ [Stage 1]) within the past 2 years or a previous (>2 years) diagnosis of cancer that has required any form of intervention or treatment within the past 2 years
- Prior intolerance or hypersensitivity to methylthioninium (MT)-containing drug or methemoglobinemia induced by MT-containing drug, similar organic dyes, or any of the excipients
- Treatment currently or within 90 days before Baseline with Souvenaid®, clozapine, carbamazepine, primidone, valproate, or drugs for which there is a warning or precaution in the labeling about methemoglobinemia at approved doses
- Current or prior participation in any clinical trial of TRx0237; a clinical trial of a product for cognition prior to Baseline in which the last dose was received within 90 days prior to Baseline unless confirmed to have been randomized to placebo; or a clinical trial of any other investigational drug, biologic, device, or medical food in which the last dose was received within 28 days prior to Baseline
Data sourced from ClinicalTrials.gov (NCT03446001). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.