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A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease (REASON)

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ClinicalTrials.gov Identifier: NCT03976349
Recruitment Status : Recruiting
First Posted : June 6, 2019
Last Update Posted : January 14, 2021
Sponsor:
Collaborator:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Biogen

Tracking Information
First Submitted Date  ICMJE June 4, 2019
First Posted Date  ICMJE June 6, 2019
Last Update Posted Date January 14, 2021
Actual Study Start Date  ICMJE August 12, 2019
Estimated Primary Completion Date September 29, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 28, 2020)
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Part A: Screening (Day -42) up to Day 85, Part B: Screening (Day -77) up to Day 253 ]
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.
Original Primary Outcome Measures  ICMJE
 (submitted: June 4, 2019)
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Screening (Day -42) up to Day 253 ]
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.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 4, 2019)
  • Serum Concentrations of BIIB094 [ Time Frame: Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 ]
  • 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 ]
  • 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 ]
  • Maximum Concentration (Cmax) of BIIB094 [ Time Frame: Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 ]
  • Time to Reach Maximum Concentration (Tmax) of BIIB094 [ Time Frame: Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 ]
  • Terminal Elimination Half-Life (t1/2) of BIIB094 [ Time Frame: Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Parkinson's Disease
Intervention  ICMJE
  • Drug: BIIB094
    Administered as specified in the treatment arm.
  • Drug: Placebo
    Administered as specified in the treatment arm.
Study Arms  ICMJE
  • Experimental: Part A (SAD): BIIB094 Dose 1
    Participants will receive a single IT injection of BIIB094 during Part A [Single Ascending Dose (SAD)].
    Intervention: Drug: BIIB094
  • Experimental: Part A (SAD): BIIB094 Dose 2
    Participants will receive a single IT injection of BIIB094 during Part A (SAD).
    Intervention: Drug: BIIB094
  • Experimental: Part A (SAD): BIIB094 Dose 3
    Participants will receive a single IT injection of BIIB094 during Part A (SAD).
    Intervention: Drug: BIIB094
  • Experimental: Part A (SAD): BIIB094 Dose 4
    Participants will receive a single IT injection of BIIB094 during Part A (SAD).
    Intervention: Drug: BIIB094
  • Experimental: Part A (SAD): BIIB094 Dose 5
    Participants will receive a single IT injection of BIIB094 during Part A (SAD).
    Intervention: Drug: BIIB094
  • Experimental: Part A (SAD): BIIB094 Dose 6
    Participants will receive a single IT injection of BIIB094 during Part A (SAD).
    Intervention: Drug: BIIB094
  • Experimental: Part B (MAD): BIIB094 Dose 1
    Participants will receive a single IT injection of BIIB094 on multiple days during Part B [Multiple Ascending Dose (MAD)].
    Intervention: Drug: BIIB094
  • Experimental: Part B (MAD): BIIB094 (Non LRRK2) Dose 2
    Participants [Non leucine-rich repeat kinase 2 (Non LRRK2)] will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
    Intervention: Drug: BIIB094
  • Experimental: Part B (MAD): BIIB094 (LRRK2) Dose 2
    Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
    Intervention: Drug: BIIB094
  • Experimental: Part B (MAD): BIIB094 (Non LRRK2) Dose 3
    Participants (Non LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
    Intervention: Drug: BIIB094
  • Experimental: Part B (MAD): BIIB094 (LRRK2) Dose 3
    Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
    Intervention: Drug: BIIB094
  • Placebo Comparator: Part A (SAD): Matching Placebo
    Participants will receive matching placebo during Part A [Single Ascending Dose (SAD)].
    Intervention: Drug: Placebo
  • Placebo Comparator: Part B (MAD): Matching Placebo
    Participants will receive matching placebo on multiple days during Part B (MAD).
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 28, 2020)
82
Original Estimated Enrollment  ICMJE
 (submitted: June 4, 2019)
62
Estimated Study Completion Date  ICMJE September 29, 2023
Estimated Primary Completion Date September 29, 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 in the last 7 years, 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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 35 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: US Biogen Clinical Trial Center 866-633-4636 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com
Listed Location Countries  ICMJE Canada,   Israel,   Norway,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03976349
Other Study ID Numbers  ICMJE 254PD101
2018-002995-42 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
URL: http://www.biogenclinicaldatarequest.com/
Responsible Party Biogen
Study Sponsor  ICMJE Biogen
Collaborators  ICMJE Ionis Pharmaceuticals, Inc.
Investigators  ICMJE
Study Director: Medical Director Biogen
PRS Account Biogen
Verification Date January 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP