NCT02388295

Brief Summary

AZD3241 myeloperoxidase (MPO) inhibitor trial is assessing safety and tolerability, randomized trial, in patients with Multiple System Atrophy.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
59

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Apr 2015

Shorter than P25 for phase_2

Geographic Reach
7 countries

17 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 9, 2015

Completed
8 days until next milestone

First Posted

Study publicly available on registry

March 17, 2015

Completed
1 month until next milestone

Study Start

First participant enrolled

April 27, 2015

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 19, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 19, 2016

Completed
1 year until next milestone

Results Posted

Study results publicly available

September 25, 2017

Completed
Last Updated

September 25, 2017

Status Verified

July 1, 2017

Enrollment Period

1.4 years

First QC Date

March 9, 2015

Results QC Date

June 23, 2017

Last Update Submit

August 18, 2017

Conditions

Multiple System Atrophy, MSA

Keywords

Multiple System Atrophy (MSA), PET imaging, myeloperoxidase (MPO) inhibitor

Outcome Measures

Primary Outcomes (1)

  • Striatum Brain Region: Change From Baseline in Microglia Activation Via Positron Emission Tomography(PET)

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

    Baseline (pre randomization) and Week 12

Secondary Outcomes (1)

  • Myeloperoxidase (MPO) Inhibition in Plasma (Change From Baseline), Specific Activity

    Baseline (Day -1) and week 12

Other Outcomes (1)

  • Exploratory Efficacy: Unified Multiple System Atropy Rating Scale, Change From Baseline (Total Score, Part 1 + Part 2)

    Baseline to final treatment visit

Study Arms (2)

AZD3241

EXPERIMENTAL

Subjects will be randomized to one of the two doses of AZD3241 or placebo in a 1:1:1 ratio.

Drug: AZD3241

Placebo to match AZD3241

PLACEBO COMPARATOR

Subjects will be randomized to one of the two doses of AZD3241 or placebo in a 1:1:1 ratio.

Drug: Placebo

Interventions

AZD3241DRUG

Drug: AZD3241 administered for 12 weeks orally as a tablet.

Also known as: AZD3241 to match placebo administered for 12 weeks.
AZD3241
PlaceboDRUG

Placebo to match AZD3241 administered for 12 weeks orally as a tablet.

Also known as: Placebo to match AZD3241 administered for 12 weeks.
Placebo to match AZD3241

Eligibility Criteria

Age30 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male or female, age 30-80 years, inclusive, at screen.
  • Meet criteria for diagnosis of probable or possible MSA according to the consensus criteria (Gilman et al. 2008 ).
  • "High-affinity binder" or "mixed-affinity binder" for TSPO, as confirmed by prospective genotyping of TSPO polymorphism during screen.
  • Subjects must understand the nature of the study and must provide signed and dated written informed consent in accordance with local regulations before the conduct of any study-related procedures. The informed consent should reflect the protocol stipulations concerning the use of contraception.
  • Medical treatment of MSA and co-morbid medical conditions must be stable for at least 30 days prior to screen and between screen and baseline.
  • Written and oral fluency in the local language.
  • Able and willing to participate in all scheduled evaluations, abide by all study restrictions, and complete all required tests and procedures.
  • In the opinion of the investigator, the subject must be considered likely to comply with the study protocol and to have a high probability of completing the study.
  • Able to swallow tablets whole.

You may not qualify if:

  • Prior participation in any AZD3241 study.
  • Magnetic resonance imaging (MRI) performed during screen not consistent with diagnosis of MSA.
  • Received a PET scan within the last 12 months.
  • Negative Allen test in both hands, unless the brachial artery is used for arterial cannulation.
  • Subjects determined to be "low affinity binders" by TSPO genotyping.
  • Claustrophobia that would contraindicate a brain MRI scan or brain PET scan.
  • Pregnancy, lactation, or, if female of childbearing potential, positive serum β-hCG at screen or positive urine β-hCG at baseline (Day -1).
  • Initiation or change in pharmacologic therapy for symptoms of MSA within 30 days prior to screen or between screen and baseline (Day -1).
  • Significant neurological disease affecting the central nervous system (CNS), other than MSA
  • History of brain surgery for parkinsonism.
  • History of stem cell treatment.
  • Seizure disorder, unless well controlled and for which treatment has been stable for at least 30 days prior to screen and between screen and baseline (Day -1).
  • Presence of any clinically significant medical condition
  • History or presence of thyroid disease.
  • Any abnormal TSH or Free T4 test result at screen or baseline (Day -1).
  • +9 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

Research Site

Stanford, California, 94305, United States

Location

Research Site

New Haven, Connecticut, 06520-8048, United States

Location

Research Site

Tampa, Florida, 33613, United States

Location

Research Site

Boston, Massachusetts, 02215, United States

Location

Research Site

Ann Arbor, Michigan, 48105-2945, United States

Location

Research Site

Rochester, Minnesota, 55905, United States

Location

Research Site

New York, New York, 10016, United States

Location

Research Site

New York, New York, 10032, United States

Location

Research Site

Innsbruck, 6020, Austria

Location

Research Site

Turku, 20520, Finland

Location

Research Site

Bordeaux, 33076, France

Location

Research Site

Toulouse, 31059, France

Location

Research Site

Salerno, 84131, Italy

Location

Research Site

Stockholm, 141 86, Sweden

Location

Research Site

London, SE5 9RJ, United Kingdom

Location

Research Site

London, W12 0NN, United Kingdom

Location

Research Site

Oxford, OX3 9DU, United Kingdom

Location

MeSH Terms

Conditions

Multiple System AtrophyThyroid Dyshormonogenesis 2A

Interventions

AZD3241

Condition Hierarchy (Ancestors)

Primary DysautonomiasAutonomic Nervous System DiseasesNervous System DiseasesBasal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesMovement DisordersSynucleinopathiesNeurodegenerative Diseases

Limitations and Caveats

Biomarker samples were included if testing done within 6 months of collection, recommendation changed to 3 months of collection during study. Included based on 6 months as per protocol to include as many samples as possible.

Results Point of Contact

Title
Jamie Mullen MD
Organization
AstraZeneca Pharmaceuticals LP
Clinicaltrialtransparency@astrazeneca.net

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 9, 2015

First Posted

March 17, 2015

Study Start

April 27, 2015

Primary Completion

September 19, 2016

Study Completion

September 19, 2016

Last Updated

September 25, 2017

Results First Posted

September 25, 2017

Record last verified: 2017-07

Locations

FR(2)US(8)IT(1)FI(1)GB(3)SE(1)AU(1)