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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03976349
Recruitment Status : Recruiting
First Posted : June 6, 2019
Last Update Posted : April 13, 2023
Sponsor:
Collaborator:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Biogen

Study Description
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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.

Condition or disease Intervention/treatment Phase
Parkinson's Disease Drug: BIIB094 Drug: Placebo Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 82 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : August 12, 2019
Estimated Primary Completion Date : December 2, 2023
Estimated Study Completion Date : December 2, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Part A (SAD): BIIB094 Dose 1
Participants will receive a single IT injection of BIIB094 during Part A [Single Ascending Dose (SAD)].
 Drug: BIIB094
Administered as specified in the treatment arm.
Experimental: Part A (SAD): BIIB094 Dose 2
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
 Drug: BIIB094
Administered as specified in the treatment arm.
Experimental: Part A (SAD): BIIB094 Dose 3
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
 Drug: BIIB094
Administered as specified in the treatment arm.
Experimental: Part A (SAD): BIIB094 Dose 4
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
 Drug: BIIB094
Administered as specified in the treatment arm.
Experimental: Part A (SAD): BIIB094 Dose 5
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
 Drug: BIIB094
Administered as specified in the treatment arm.
Experimental: Part A (SAD): BIIB094 Dose 6
Participants will receive a single IT injection of BIIB094 during Part A (SAD).
 Drug: BIIB094
Administered as specified in the treatment arm.
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)].
 Drug: BIIB094
Administered as specified in the treatment arm.
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).
 Drug: BIIB094
Administered as specified in the treatment arm.
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).
 Drug: BIIB094
Administered as specified in the treatment arm.
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).
 Drug: BIIB094
Administered as specified in the treatment arm.
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).
 Drug: BIIB094
Administered as specified in the treatment arm.
Placebo Comparator: Part A (SAD): Matching Placebo
Participants will receive matching placebo during Part A [Single Ascending Dose (SAD)].
 Drug: Placebo
Administered as specified in the treatment arm.
Placebo Comparator: Part B (MAD): Matching Placebo
Participants will receive matching placebo on multiple days during Part B (MAD).
 Drug: Placebo
Administered as specified in the treatment arm.


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Serum Concentrations of BIIB094 [ Time Frame: Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 ]
  2. 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 ]
  3. 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 ]
  4. Maximum Concentration (Cmax) of BIIB094 [ Time Frame: Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 ]
  5. Time to Reach Maximum Concentration (Tmax) of BIIB094 [ Time Frame: Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 ]
  6. Terminal Elimination Half-Life (t1/2) of BIIB094 [ Time Frame: Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 ]

Eligibility Criteria
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Ages Eligible for Study:   35 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03976349


Contacts
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Contact: US Biogen Clinical Trial Center 866-633-4636 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com

Locations
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Sponsors and Collaborators
Biogen
Ionis Pharmaceuticals, Inc.
Investigators
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Study Director: Medical Director Biogen
More Information
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Additional Information:

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Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT03976349    
Other Study ID Numbers: 254PD101
2018-002995-42 ( EudraCT Number )
First Posted: June 6, 2019    Key Record Dates
Last Update Posted: April 13, 2023
Last Verified: April 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
URL: https://vivli.org/

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Movement Disorders
Synucleinopathies
Neurodegenerative Diseases