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出境医 / 临床实验 / A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease (REASON)
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease (REASON)
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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Parkinson's Disease | Drug: BIIB094 Drug: Placebo | Phase 1 |
Study Design
| 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 : | September 29, 2023 |
| Estimated Study Completion Date : | September 29, 2023 |
Arms and Interventions
| 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
Primary Outcome Measures :
- 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 :
- 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 ]
Eligibility Criteria
| 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 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.
Contacts and Locations
Contacts
| Contact: US Biogen Clinical Trial Center | 866-633-4636 | clinicaltrials@biogen.com | |
| Contact: Global Biogen Clinical Trial Center | clinicaltrials@biogen.com |
Locations
| United States, Illinois | |
| Northwestern University Feinberg School of Medicine | Recruiting |
| Chicago, Illinois, United States, 60611 | |
| Contact: Monika Szela 312-503-2593 Monika.szela@northwestern.edu | |
| United States, Michigan | |
| Quest Research Institute | Recruiting |
| Farmington Hills, Michigan, United States, 48334 | |
| Contact: Ashley Murphy 248-957-8940 ashley@questri.com | |
| United States, Ohio | |
| The Cleveland Clinic Foundation | Recruiting |
| Cleveland, Ohio, United States, 44195 | |
| Contact: Jennifer Mule 216-444-1134 mulej@ccf.org | |
| United States, Oregon | |
| Oregon Health and Science University | Recruiting |
| Portland, Oregon, United States, 97239 | |
| Contact: Jean Allen 503-494-9531 allenjea@ohsu.edu | |
| United States, Tennessee | |
| Alliance for Multispecialty Research NOCCR/VRG | Recruiting |
| Knoxville, Tennessee, United States, 37920 | |
| Contact: Colleen Hayzen, RN 865-305-9100 colleen.hayzen@amrllc.com | |
| United States, Washington | |
| Inland Northwest Research | Recruiting |
| Spokane, Washington, United States, 99202 | |
| Contact: Melissa Bixby 509-960-2818 mbixby@inwresearch.com | |
| Canada, Ontario | |
| Research Site | Recruiting |
| Toronto, Ontario, Canada | |
| Canada, Quebec | |
| Montreal Neurological Institute and Hospital | Recruiting |
| Montreal, Quebec, Canada, QC H3A 2B4 | |
| Contact: Salma Khalil 514-398-2733 salma.khalil@mcgill.ca | |
| Israel | |
| Sourasky Medical Center | Recruiting |
| Tel-Aviv, Israel, 64239 | |
| Contact: Angel Migirov angelm@tlvmc.gov.il | |
| Norway | |
| St. Olav University Hospital | Withdrawn |
| Trondheim, Norway, 7030 | |
| Spain | |
| Laboratorios de Investigación Biocruces 3., Hospital de Cruces | Recruiting |
| Barakaldo, Bizkaia, Spain, 48903 | |
| Contact: Juan Carlos Gomez Esteban +34946006363 juancarlos.gomezesteban@osakidetza.net | |
| Hospital General de Catalunya | Recruiting |
| Barcelona, Vizcaya, Spain, 08195 | |
| Contact: Ernest Balaguer Martinez +3493 5656000 ext ext 1756 ebalaguer@quironsalud.es | |
| Hospital Clinic de Barcelona | Recruiting |
| Barcelona, Spain, 08036 | |
| Contact: Pilar Santacruz +34 93-2275785 trastornsmoviment@clinic.cat | |
| Hospital General Universitario Gregorio Marañón | Recruiting |
| Madrid, Spain, 28007 | |
| Contact: Francisco Grandas +34 91 5868339 francisco.grandas@salud.madrid.org | |
| Research Site | Recruiting |
| Sevilla, Spain | |
| United Kingdom | |
| Queen Square (Neurology) CRF Site Institute of Neurology & the National Hospital for Neurology and Neurosurgery UCLH | Recruiting |
| London, United Kingdom, WC1N 3BG | |
| Contact +44 0203 44 84531 | |
Sponsors and Collaborators
Biogen
Ionis Pharmaceuticals, Inc.
Investigators
| Study Director: | Medical Director | Biogen |
| Tracking Information | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| First Submitted Date ICMJE | June 4, 2019 | ||||||||
| First Posted Date ICMJE | June 6, 2019 | ||||||||
| Last Update Posted Date | May 28, 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 |
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.
|
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| Original Primary Outcome Measures ICMJE |
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.
|
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| Change History | |||||||||
| Current Secondary Outcome Measures ICMJE |
|
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| 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 |
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| Condition ICMJE | Parkinson's Disease | ||||||||
| Intervention ICMJE |
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| Study Arms ICMJE |
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| Publications * | Not Provided | ||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status ICMJE | Recruiting | ||||||||
| Estimated Enrollment ICMJE |
82 | ||||||||
| Original Estimated Enrollment ICMJE |
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:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply. |
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| Sex/Gender ICMJE |
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| Ages ICMJE | 35 Years to 80 Years (Adult, Older Adult) | ||||||||
| Accepts Healthy Volunteers ICMJE | No | ||||||||
| Contacts ICMJE |
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| 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 ) |
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| Has Data Monitoring Committee | No | ||||||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement ICMJE |
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| Responsible Party | Biogen | ||||||||
| Study Sponsor ICMJE | Biogen | ||||||||
| Collaborators ICMJE | Ionis Pharmaceuticals, Inc. | ||||||||
| Investigators ICMJE |
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| PRS Account | Biogen | ||||||||
| Verification Date | May 2021 | ||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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