Back to resultsA Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease (REASON)
Primary Purpose
Parkinson's Disease
Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
BIIB094
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Parkinson's Disease
Eligibility Criteria
Key Inclusion Criteria:
- Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulations.
- Diagnosed with PD within 7 years at the time of initial enrollment (i.e., at time of SAD enrollment for rollover participants), without major motor fluctuations or dyskinesia that may interfere with study treatment and assessments in the opinion of the investigator after consultation with the Sponsor.
- Modified Hoehn and Yahr Stage ≤ 3.
Key Exclusion Criteria:
- Montreal Cognitive Assessment (MoCA) score less than (<) 23, dementia, or other significant cognitive impairment that, in the opinion of the Investigator, would interfere with study evaluation.
- History of any brain surgery for PD or a history of focused ultrasound treatment at any time; or history of neuromodulation procedures.
- Transient ischemic attack or stroke or any unexplained loss of consciousness within 1 year before Screening.
- History of unstable angina, myocardial infarction, chronic heart failure, or clinically significant conduction abnormalities within 1 year before Screening.
- Poorly controlled diabetes mellitus, as defined by having dosage adjustment of diabetic medication within 3 months before dosing (Day 1) or glycosylated hemoglobin value greater than or equal to (≥) 8 percent (%) at Screening.
- History or positive test result at Screening for human immunodeficiency virus.
- History or positive test result at Screening for hepatitis C virus antibody.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Sites / Locations
- Northwestern University PD and Movement Disorders CenterRecruiting
- Quest Research InstituteRecruiting
- The Cleveland Clinic FoundationRecruiting
- Oregon Health and Science UniversityRecruiting
- University of PennsylvaniaRecruiting
- Alliance for Multispecialty ResearchRecruiting
- Inland Northwest ResearchRecruiting
- Research Site
- Montreal Neurological Institute and HospitalRecruiting
- Sourasky Medical CenterRecruiting
- Neuro-SysMed CenterRecruiting
- St. Olav University Hospital
- Laboratorios de Investigación Biocruces 3., Hospital de CrucesRecruiting
- Hospital General de CatalunyaRecruiting
- Hospital Clinic de BarcelonaRecruiting
- Hospital General Universitario Gregorio MarañónRecruiting
- Research SiteRecruiting
- Queen Square (Neurology) CRF Site Institute of Neurology & the National Hospital for Neurology and Neurosurgery UCLHRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Arm 7
Arm 8
Arm 9
Arm 10
Arm 11
Arm 12
Arm 13
Arm Type
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Placebo Comparator
Placebo Comparator
Arm Label
Part A (SAD): BIIB094 Dose 1
Part A (SAD): BIIB094 Dose 2
Part A (SAD): BIIB094 Dose 3
Part A (SAD): BIIB094 Dose 4
Part A (SAD): BIIB094 Dose 5
Part A (SAD): BIIB094 Dose 6
Part B (MAD): BIIB094 Dose 1
Part B (MAD): BIIB094 (Non LRRK2) Dose 2
Part B (MAD): BIIB094 (LRRK2) Dose 2
Part B (MAD): BIIB094 (Non LRRK2) Dose 3
Part B (MAD): BIIB094 (LRRK2) Dose 3
Part A (SAD): Matching Placebo
Part B (MAD): Matching Placebo
Arm Description
Participants will receive a single IT injection of BIIB094 during Part A [Single Ascending Dose (SAD)].
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
Participants will receive a single IT injection of BIIB094 on multiple days during Part B [Multiple Ascending Dose (MAD)].
Participants [Non leucine-rich repeat kinase 2 (Non LRRK2)] will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Participants (Non LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Participants will receive matching placebo during Part A [Single Ascending Dose (SAD)].
Participants will receive matching placebo on multiple days during Part B (MAD).
Outcomes
Primary Outcome Measures
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); however, this does not include an event that, had it occurred in a more severe form, might have caused death; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; is a medically important event.
Secondary Outcome Measures
Serum Concentrations of BIIB094
Area Under the Concentration-Time Curve From Time Zero Extrapolated to Infinity (AUCinf) of BIIB094
Area Under the Concentration-Time Curve From Time Zero to Last Quantifiable Concentration (AUClast) of BIIB094
Maximum Concentration (Cmax) of BIIB094
Time to Reach Maximum Concentration (Tmax) of BIIB094
Terminal Elimination Half-Life (t1/2) of BIIB094
Full Information
NCT ID
NCT03976349
First Posted
June 4, 2019
Last Updated
April 11, 2023
Sponsor
Biogen
Collaborators
Ionis Pharmaceuticals, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03976349
Brief Title
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
Acronym
REASON
Official Title
A Phase 1 Single- and Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB094 Administered Intrathecally to Adults With Parkinson's Disease
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 12, 2019 (Actual)
Primary Completion Date
December 2, 2023 (Anticipated)
Study Completion Date
December 2, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Biogen
Collaborators
Ionis Pharmaceuticals, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The primary objective of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 administered via intrathecal (IT) injection to participants with Parkinson's Disease (PD). The secondary objective of this study is to evaluate the pharmacokinetic (PK) profile of BIIB094.The study is open for PD patients with verified presence or absence of variations in the leucine-rich repeated kinase 2 (LRRK2) gene, but also for patients without any verified PD-related genetic variant.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson's Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
ParticipantInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
82 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Part A (SAD): BIIB094 Dose 1
Arm Type
Experimental
Arm Description
Participants will receive a single IT injection of BIIB094 during Part A [Single Ascending Dose (SAD)].
Arm Title
Part A (SAD): BIIB094 Dose 2
Arm Type
Experimental
Arm Description
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
Arm Title
Part A (SAD): BIIB094 Dose 3
Arm Type
Experimental
Arm Description
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
Arm Title
Part A (SAD): BIIB094 Dose 4
Arm Type
Experimental
Arm Description
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
Arm Title
Part A (SAD): BIIB094 Dose 5
Arm Type
Experimental
Arm Description
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
Arm Title
Part A (SAD): BIIB094 Dose 6
Arm Type
Experimental
Arm Description
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
Arm Title
Part B (MAD): BIIB094 Dose 1
Arm Type
Experimental
Arm Description
Participants will receive a single IT injection of BIIB094 on multiple days during Part B [Multiple Ascending Dose (MAD)].
Arm Title
Part B (MAD): BIIB094 (Non LRRK2) Dose 2
Arm Type
Experimental
Arm Description
Participants [Non leucine-rich repeat kinase 2 (Non LRRK2)] will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Arm Title
Part B (MAD): BIIB094 (LRRK2) Dose 2
Arm Type
Experimental
Arm Description
Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Arm Title
Part B (MAD): BIIB094 (Non LRRK2) Dose 3
Arm Type
Experimental
Arm Description
Participants (Non LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Arm Title
Part B (MAD): BIIB094 (LRRK2) Dose 3
Arm Type
Experimental
Arm Description
Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Arm Title
Part A (SAD): Matching Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will receive matching placebo during Part A [Single Ascending Dose (SAD)].
Arm Title
Part B (MAD): Matching Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will receive matching placebo on multiple days during Part B (MAD).
Intervention Type
Drug
Intervention Name(s)
BIIB094
Intervention Description
Administered as specified in the treatment arm.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Administered as specified in the treatment arm.
Primary Outcome Measure Information:
Title
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Description
An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); however, this does not include an event that, had it occurred in a more severe form, might have caused death; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; is a medically important event.
Time Frame
Part A: Screening (Day -42) up to Day 85, Part B: Screening (Day -77) up to Day 253
Secondary Outcome Measure Information:
Title
Serum Concentrations of BIIB094
Time Frame
Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
Title
Area Under the Concentration-Time Curve From Time Zero Extrapolated to Infinity (AUCinf) of BIIB094
Time Frame
Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
Title
Area Under the Concentration-Time Curve From Time Zero to Last Quantifiable Concentration (AUClast) of BIIB094
Time Frame
Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
Title
Maximum Concentration (Cmax) of BIIB094
Time Frame
Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
Title
Time to Reach Maximum Concentration (Tmax) of BIIB094
Time Frame
Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
Title
Terminal Elimination Half-Life (t1/2) of BIIB094
Time Frame
Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
10. Eligibility
Sex
All
Minimum Age & Unit of Time
35 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulations.
Diagnosed with PD within 7 years at the time of initial enrollment (i.e., at time of SAD enrollment for rollover participants), without major motor fluctuations or dyskinesia that may interfere with study treatment and assessments in the opinion of the investigator after consultation with the Sponsor.
Modified Hoehn and Yahr Stage ≤ 3.
Key Exclusion Criteria:
Montreal Cognitive Assessment (MoCA) score less than (<) 23, dementia, or other significant cognitive impairment that, in the opinion of the Investigator, would interfere with study evaluation.
History of any brain surgery for PD or a history of focused ultrasound treatment at any time; or history of neuromodulation procedures.
Transient ischemic attack or stroke or any unexplained loss of consciousness within 1 year before Screening.
History of unstable angina, myocardial infarction, chronic heart failure, or clinically significant conduction abnormalities within 1 year before Screening.
Poorly controlled diabetes mellitus, as defined by having dosage adjustment of diabetic medication within 3 months before dosing (Day 1) or glycosylated hemoglobin value greater than or equal to (≥) 8 percent (%) at Screening.
History or positive test result at Screening for human immunodeficiency virus.
History or positive test result at Screening for hepatitis C virus antibody.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
US Biogen Clinical Trial Center
Phone
866-633-4636
Email
clinicaltrials@biogen.com
First Name & Middle Initial & Last Name or Official Title & Degree
Global Biogen Clinical Trial Center
Email
clinicaltrials@biogen.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Biogen
Official's Role
Study Director
Facility Information:
Facility Name
Northwestern University PD and Movement Disorders Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Monika Szela
Phone
312-503-2593
Email
monika.szela@northwestern.edu
Facility Name
Quest Research Institute
City
Farmington Hills
State/Province
Michigan
ZIP/Postal Code
48334
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Denise Morgott
Phone
248-957-8940
Email
denise.morgott@questri.com
Facility Name
The Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jennifer Mule
Phone
216-444-1134
Email
mulej@ccf.org
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Emily Leonard
Phone
503-494-8881
Email
leonarde@ohsu.edu
First Name & Middle Initial & Last Name & Degree
Morgan Willhelmi
Phone
503-494-8881
Email
wilhelmo@ohsu.edu
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Debbra Grier
Phone
215-662-4079
Email
grier@pennmedicine.upenn.edu
Facility Name
Alliance for Multispecialty Research
City
Knoxville
State/Province
Tennessee
ZIP/Postal Code
37920
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Colleen Hayzen, RN
Phone
865-305-9100
Ext
277
Email
colleen.hayzen@amrllc.com
Facility Name
Inland Northwest Research
City
Spokane
State/Province
Washington
ZIP/Postal Code
99202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Melissa Bixby
Phone
509-960-2818
Ext
709
Email
mbixby@inwresearch.com
Facility Name
Research Site
City
Toronto
State/Province
Ontario
Country
Canada
Individual Site Status
Withdrawn
Facility Name
Montreal Neurological Institute and Hospital
City
Montreal
State/Province
Quebec
ZIP/Postal Code
QC H3A 2B4
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Christina Caccese
Phone
514-398-1409
Email
christina.caccese@mcgill.ca
Facility Name
Sourasky Medical Center
City
Tel-Aviv
ZIP/Postal Code
64239
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Angel Migirov
Email
angelm@tlvmc.gov.il
Facility Name
Neuro-SysMed Center
City
Bergen
ZIP/Postal Code
5053
Country
Norway
Individual Site Status
Recruiting
Facility Name
St. Olav University Hospital
City
Trondheim
ZIP/Postal Code
7030
Country
Norway
Individual Site Status
Withdrawn
Facility Name
Laboratorios de Investigación Biocruces 3., Hospital de Cruces
City
Barakaldo
State/Province
Bizkaia
ZIP/Postal Code
48903
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Juan Carlos Gomez Esteban
Phone
+34946006363
Email
juancarlos.gomezesteban@osakidetza.net
Facility Name
Hospital General de Catalunya
City
Barcelona
State/Province
Vizcaya
ZIP/Postal Code
08195
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ernest Balaguer Martinez
Phone
+3493 5656000
Ext
ext 1756
Email
s.andrade@udic.es
Facility Name
Hospital Clinic de Barcelona
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pilar Santacruz
Phone
+34 93-2275785
Email
trastornsmoviment@clinic.cat
Facility Name
Hospital General Universitario Gregorio Marañón
City
Madrid
ZIP/Postal Code
28007
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Francisco Grandas
Phone
+34 91 5868339
Email
francisco.grandas@salud.madrid.org
Facility Name
Research Site
City
Sevilla
Country
Spain
Individual Site Status
Recruiting
Facility Name
Queen Square (Neurology) CRF Site Institute of Neurology & the National Hospital for Neurology and Neurosurgery UCLH
City
London
ZIP/Postal Code
WC1N 3BG
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alexandra Lameirinhas
Phone
+44 0203 44 84531
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
IPD Sharing URL
https://vivli.org/
Links:
URL
https://thereasonstudy.com/
Description
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Learn more about this trial
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
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