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AZD3241

Phase 2

Multiple System Atrophy, MSA | Small molecule | Neurology |AstraZeneca PLC|Last Updated: Sep 25, 2017

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment59

FDA Designations

No designations recorded

Clinical trial landscape

AZD3241 · 5 trials · 4 indications

Phase 2 3Phase 1 2
NCT02388295AZD3241 PET MSA Trial, Phase 2, Randomized,12 Week Safety and Tolerability Trial With PET in MSA PatientsMultiple System Atrophy, MSA
COMPLETED59 Analytics
NCT01603069A Study to Assess Safety and Tolerability of Oral AZD3241 in Patients With Parkinson's DiseaseParkinson's Disease
COMPLETED51 Analytics
NCT01527695PET Study in Parkinson's Disease PatientsParkinson's Disease
COMPLETED24 Analytics
PHASE2COMPLETED
AZD3241 PET MSA Trial, Phase 2, Randomized,12 Week Safety and Tolerability Trial With PET in MSA Patients
Multiple System Atrophy, MSAUnlock trial analytics
PHASE2COMPLETED
A Study to Assess Safety and Tolerability of Oral AZD3241 in Patients With Parkinson's Disease
Parkinson's DiseaseUnlock trial analytics
PHASE2COMPLETED
PET Study in Parkinson's Disease Patients
Parkinson's DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Striatum Brain Region: Change From Baseline in Microglia Activation Via Positron Emission Tomography(PET)
Baseline (pre randomization) and Week 12

Striatum Brain region: Change from baseline in microglia activation via PET By \[11C\]PBR28 binding to translocator protein

Change from baseline in vital signs.
Screening (baseline), randomization, after 2, 4, 8 and 12 weeks of treatment and 2 weeks after termination of treatment (week 14)

Vital signs: systolic and diastolic blood pressure and pulse including orthostatic challenge will be assessed. Change from baseline at each visit will be calculated as the visit value minus the baseline value for each vital sign: Blood Pressure, pulse rate (supine and standing), weight and oral temperature.

Change from baseline in Physical Exam results.
Baseline and 2 weeks after termination of treatment (week 14)

Assessment of general appearance, skin, head and neck, lymph nodes, thyroid, abdomen, cardiovascular, respiratory, and neurological systems, including full palpation of thyroid gland.

Change from baseline in Suicidality as assessed by the Columbia-Suicide Severity Rating Scale (CSSRS).
screening (baseline), randomization, after 2, 4, 8 and 12 weeks of treatment and 2 weeks after termination of treatment (week 14)

Suicidality as assessed by the Columbia-Suicide Severity Rating Scale (CSSRS) The CSSRS assesses the suicidal behavior and suicidal ideation in patients. Occurrence of suicidal behavior is defined as having answered "yes" to a least 1 of the 4 suicidal behavior sub categories (actual attempt, interrupted attempt, aborted attempt, and preparatory acts or behavior) at any post randomization evaluation. Occurrence of suicidal ideation after randomization is defined as having answered "yes" to at least one of the suicidal ideation sub-categories (wish to be dead, non-specific active suicidal thoughts, active suicidal ideation with any methods \[not plan\] without intent to act, active suicidal ideation with some intent to act \[without specific plan\], and active suicidal ideation with specific plan and intent) at any post randomization evaluation.

Adverse events (AEs) including frequency and severity.
Screening, randomization, week 1, 2, 4, 8, 12, 14
Change from baseline in laboratory safety assessments.
Screening (baseline), randomization, after 2, 4, 8 and 12 weeks of treatment and 2 weeks after termination of treatment (week 14)

Change from baseline at each visit will be calculated as the visit value minus the baseline value for each continuous clinical chemistry, hematology and urinalysis measurements. Abnormal or out-of-range values will be flagged.

Change from baseline in 12-lead ECG.
Screening (baseline), randomization, after 2, 4, 8 and 12 weeks of treatment and 2 weeks after termination of treatment (week 14)

Change from baseline at each visit will be calculated as the visit value minus the baseline value for each ECG parameter: heart rate, QRS duration, PR interval, RR interval, QT and calculated QTcF interval. Abnormal or out-of-range values will be flagged.

Change of binding [11C]PBR28 to translocator protein (TSPO) measured by Positron Emission Tomography (PET).
baseline, 2-4 weeks
Change of binding of [11C]PBR28 to TSPO measured by PET.
baseline, 7-8 weeks
Safety variables (adverse events, vital signs, ECG, safety lab)
Assessments performed at frequent timepoints during a 4-8 week period
General tolerability and safety variables
2 weeks

Secondary Endpoints

Myeloperoxidase (MPO) Inhibition in Plasma (Change From Baseline), Specific Activity
Baseline (Day -1) and week 12
Pharmacokinetics (PK) of AZD3241 in the terms of Cmax, Cmin, and AUC0-t.
Randomization and after week 1, 2, 4, 8, and 12 weeks of treatment
Pharmacodynamic effect of AZD3241 in the terms of Myeloperoxidase (MPO) activity in plasma.
Screening (baseline), randomization, after, 4, 8 and 12 weeks of treatment and 2 weeks after termination of treatment (week 14)
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AZD3241EXPERIMENTALSubjects will be randomized to one of the two doses of AZD3241 or placebo in a 1:1:1 ratio.
Placebo to match AZD3241PLACEBO_COMPARATORSubjects will be randomized to one of the two doses of AZD3241 or placebo in a 1:1:1 ratio.
AZD3241, 300 mgACTIVE_COMPARATORAZD3241 300 mg BID
AZD3241, 600 mgACTIVE_COMPARATORAZD3241 600 mg BID
PlaceboPLACEBO_COMPARATORPlacebo to AZD3241
1EXPERIMENTAL -
2PLACEBO_COMPARATOR -

Interventions

NameTypeDescription
AZD3241DRUGDrug: AZD3241 administered for 12 weeks orally as a tablet.
PlaceboDRUGPlacebo to match AZD3241 administered for 12 weeks orally as a tablet.
AZD3241 300 mg BIDDRUGThe following dose escalation schedule will be used for 300 mg BID: 100 mg BID from Day 1 through Day 7. On Day 8, the patients will start maintenance treatment of 300 mg BID for the duration of the treatment period.
AZD3241 600 mg BIDDRUGThe following dose escalation schedule will be used for 600 mg BID: 100 mg BID from Day 1 through Day 7 and 300 mg BID from Day 8 through Day 14. On Day 15, the patients will start maintenance treatment of 600 mg BID for the duration of the treatment period.
ER tablet 25 mg AZD3241DRUG2 tablets twice daily for Day 1
ER tablet 100 mg AZD3241DRUG1-6 tablets twice daily from Day 2 until Day 56±3 days
Placebo for AZD3241 25 mgDRUG2 tablets twice daily for Day 1
Placebo for AZD3241 100 mgDRUG1-6 tablets twice daily from Day 2 until Day 56±3 days
Placebo TabletDRUGOral Tablet. Repeated Administration
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Eligibility Criteria

Age Range30 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites17

Inclusion Criteria: 1. Male or female, age 30-80 years, inclusive, at screen. 2. Meet criteria for diagnosis of probable or possible MSA according to the consensus criteria (Gilman et al. 2008 ). 3. "High-affinity binder" or "mixed-affinity binder" for TSPO, as confirmed by prospective genotyping o...

Countries:United StatesAustriaFinlandFranceItalySwedenUnited Kingdom
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Frequently asked questions about AZD3241

What is AZD3241 used for?

AZD3241 is an investigational small molecule being studied in Parkinson's Disease, Multiple System Atrophy (MSA), and in healthy volunteers. It has been evaluated in clinical trials for these conditions, though it remains in clinical development and is not approved.

Who makes AZD3241?

AZD3241 is being developed by AstraZeneca PLC, which trades under the ticker AZN. The company has sponsored clinical trials of the drug in Parkinson's Disease and Multiple System Atrophy.

What phase is AZD3241 in?

AZD3241 has completed Phase 1 and Phase 2 clinical trials. It is an investigational drug still in clinical development, with no approved status. Trials have been completed in healthy volunteers, Parkinson's Disease, and Multiple System Atrophy.

What clinical trials is AZD3241 in?

AZD3241 has been studied in several completed trials, including NCT00729443 in healthy volunteers, NCT01527695 and NCT01603069 in Parkinson's Disease, and NCT02388295 in Multiple System Atrophy. All trials are completed, with no active trials ongoing.

Is AZD3241 the same as other names?

AZD3241 is the primary name used in clinical trials and publications. No alternative names have been reported for this drug in the available data.