Trial Outcomes & Findings for Open Label Study of ATH-1017 for Treatment of Mild to Moderate Alzheimer's Disease (NCT NCT04886063)
NCT ID: NCT04886063
Last Updated: 2025-04-01
Results Overview
Description - To determine the safety and tolerability of ATH-1017 in subjects with mild to moderate Alzheimer's disease (AD) who completed the 26-week randomized treatment in Study ATH-1017-AD-0201 or Study ATH-1017-AD-0202
Recruitment status
TERMINATED
Study phase
PHASE2/PHASE3
Target enrollment
423 participants
Primary outcome timeframe
Up to 173 weeks (study termination)
Results posted on
2025-04-01
Participant Flow
This study was conducted in the United States and Australia.
This was a multicenter, open label extension (OLEX) study of ATH-1017 treatment in participants with mild to moderate Alzheimer's disease (AD) who completed 26 weeks of treatment in the randomized, placebo-controlled, double-blind studies (ATH-1017-AD-0201; LIFT-AD or ATH-1017-AD-0202; ACT-AD).
Participant milestones
| Measure |
ATH-1017 40 Milligrams (mg)
Participants administered ATH-1017 40 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD).
Participants entering ATH-1017-AD-0203 on protocol versions prior to V4 were initially administered ATH-1017 70 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD). Data are reported for the overall safety population, including those who were previously treated with ATH-1017 70 mg.
|
|---|---|
|
Overall Study
STARTED
|
423
|
|
Overall Study
COMPLETED
|
35
|
|
Overall Study
NOT COMPLETED
|
388
|
Reasons for withdrawal
| Measure |
ATH-1017 40 Milligrams (mg)
Participants administered ATH-1017 40 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD).
Participants entering ATH-1017-AD-0203 on protocol versions prior to V4 were initially administered ATH-1017 70 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD). Data are reported for the overall safety population, including those who were previously treated with ATH-1017 70 mg.
|
|---|---|
|
Overall Study
Adverse Event
|
50
|
|
Overall Study
Lack of Efficacy
|
31
|
|
Overall Study
Withdrawal by Subject
|
45
|
|
Overall Study
Physician Decision
|
3
|
|
Overall Study
Protocol Violation
|
3
|
|
Overall Study
Site closure
|
4
|
|
Overall Study
Nursing home replacement
|
3
|
|
Overall Study
Lost to Follow-up
|
5
|
|
Overall Study
Early study termination or other undetermined reasons
|
224
|
|
Overall Study
>1 reason
|
20
|
Baseline Characteristics
Open Label Study of ATH-1017 for Treatment of Mild to Moderate Alzheimer's Disease
Baseline characteristics by cohort
| Measure |
ATH-1017 40 Milligrams (mg)
n=423 Participants
Participants administered ATH-1017 40 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD).
Participants entering ATH-1017-AD-0203 on protocol versions prior to V4 were initially administered ATH-1017 70 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD). Data are reported for the overall safety population, including those who were previously treated with ATH-1017 70 mg.
|
|---|---|
|
Age, Continuous
|
72.5 years
STANDARD_DEVIATION 7.34 • n=5 Participants
|
|
Sex: Female, Male
Female
|
218 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
205 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
14 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
17 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
384 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
7 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 173 weeks (study termination)Population: Safety population: all participants who received at least one dose of study medication
Description - To determine the safety and tolerability of ATH-1017 in subjects with mild to moderate Alzheimer's disease (AD) who completed the 26-week randomized treatment in Study ATH-1017-AD-0201 or Study ATH-1017-AD-0202
Outcome measures
| Measure |
ATH-1017 40 Milligrams (mg)
n=423 Participants
Participants administered ATH-1017 40 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD).
Participants entering ATH-1017-AD-0203 on protocol versions prior to V4 were initially administered ATH-1017 70 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD). Data are reported for the overall safety population, including those who were previously treated with ATH-1017 70 mg.
|
|---|---|
|
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
At least 1 TEAE
|
323 Participants
|
|
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
No TEAEs
|
100 Participants
|
Adverse Events
ATH-1017 40 Milligrams (mg)
Serious events: 54 serious events
Other events: 348 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
ATH-1017 40 Milligrams (mg)
n=423 participants at risk
Participants administered ATH-1017 40 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD).
Participants entering ATH-1017-AD-0203 on protocol versions prior to V4 were initially administered ATH-1017 70 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD). Data are reported for the overall safety population, including those who were previously treated with ATH-1017 70 mg.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Blood and lymphatic system disorders
Monoclonal B-cell lymphocytosis
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Cardiac disorders
Atrial fibrillation
|
0.47%
2/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Cardiac disorders
Aortic valve incompetence
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Cardiac disorders
Atrioventricular block second degree
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Cardiac disorders
Bradycardia
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Cardiac disorders
Coronary artery stenosis
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Cardiac disorders
Myocardial infarction
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Congenital, familial and genetic disorders
Early onset familial Alzheimer's disease
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Gastrointestinal disorders
Faecaloma
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Gastrointestinal disorders
Oesophageal food impaction
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
General disorders
Chest pain
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Hepatobiliary disorders
Cholecystitis
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Infections and infestations
COVID-19
|
0.95%
4/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Infections and infestations
Pneumonia
|
0.95%
4/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Infections and infestations
Urinary tract infection
|
0.95%
4/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
0.47%
2/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Infections and infestations
Appendicitis
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Infections and infestations
COVID-19 pneumonia
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Infections and infestations
Cellulitis
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Injury, poisoning and procedural complications
Fall
|
0.71%
3/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Injury, poisoning and procedural complications
Cervical vertebral fracture
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Injury, poisoning and procedural complications
Hip fracture
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Injury, poisoning and procedural complications
Multiple fractures
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Metabolism and nutrition disorders
Failure to thrive
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.47%
2/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma of colon
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Clear cell renal cell carcinoma
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lung adenocarcinoma
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Prostate cancer
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Seizure
|
0.71%
3/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Metabolic encephalopathy
|
0.47%
2/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Altered state of consciousness
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Cerebellar infarction
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Cerebrovascular accident
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Dementia Alzheimer's type
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Generalised tonic-clonic seizure
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Ischaemic stroke
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Syncope
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Transient ischaemic attack
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Nervous system disorders
Tremor
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Psychiatric disorders
Acute psychosis
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Psychiatric disorders
Psychotic disorder
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Renal and urinary disorders
Ureterolithiasis
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Skin and subcutaneous tissue disorders
Angioedema
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Vascular disorders
Aortic stenosis
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Vascular disorders
Deep vein thrombosis
|
0.24%
1/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
Other adverse events
| Measure |
ATH-1017 40 Milligrams (mg)
n=423 participants at risk
Participants administered ATH-1017 40 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD).
Participants entering ATH-1017-AD-0203 on protocol versions prior to V4 were initially administered ATH-1017 70 mg via subcutaneous (SC) injection (1 mL pre-filled syringe) once-daily (QD). Data are reported for the overall safety population, including those who were previously treated with ATH-1017 70 mg.
|
|---|---|
|
General disorders
Injection site reactions
|
50.4%
213/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Infections and infestations
COVID-19
|
10.4%
44/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Infections and infestations
Upper respiratory tract infection
|
4.5%
19/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Infections and infestations
Urinary tract infection
|
6.6%
28/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Injury, poisoning and procedural complications
Fall
|
5.7%
24/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
|
Blood and lymphatic system disorders
Eosinophilia
|
4.7%
20/423 • Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER