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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-002995-42 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A (SAD): BIIB094 Dose 1 | Experimental | Participants will receive a single IT injection of BIIB094 during Part A [Single Ascending Dose (SAD)]. |
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| Part A (SAD): BIIB094 Dose 2 | Experimental | Participants will receive a single IT injection of BIIB094 during Part A (SAD). |
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| Part A (SAD): BIIB094 Dose 3 | Experimental | Participants will receive a single IT injection of BIIB094 during Part A (SAD). |
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| Part A (SAD): BIIB094 Dose 4 | Experimental | Participants will receive a single IT injection of BIIB094 during Part A (SAD). |
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| Part A (SAD): BIIB094 Dose 5 | Experimental | Participants will receive a single IT injection of BIIB094 during Part A (SAD). |
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| Part A (SAD): BIIB094 Dose 6 | Experimental |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BIIB094 | Drug | Administered as specified in the treatment arm. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | Part A: Screening (Day -42) up to Day 85, Part B: Screening (Day -77) up to Day 253 |
| Measure | Description | Time Frame |
|---|---|---|
| Serum Concentrations of BIIB094 | 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 | Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Northwestern University PD and Movement Disorders Center | Chicago | Illinois | 60611 | United States | ||
| Quest Research Institute |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41876647 | Derived | Mabrouk OS, Tichler B, Arnold HM, Thoma EC, Alexanian SM, Chen J, Liu TY, Hirschhorn B, Llorens Arenas R, Annand JW, Kapadnis U, Shomo AA, Glajch KE, Ferber K, Moroishi Y, Czerkowicz J, Inra J, Postuma RB, Gurevich T, Mir P, Morris HR, Aldred J, Brodsky MA, Ellenbogen A, Larson D, Tolleson CM, Siderowf A, Tzoulis C, Balaguer E, Marti MJ, Zhao HT, Kordasiewicz HB, Lane R, Hirst WD, Fradette S, Graham DL. LRRK2-targeting antisense oligonucleotide in Parkinson's disease: a phase 1 randomized controlled trial. Nat Med. 2026 Apr;32(4):1421-1431. doi: 10.1038/s41591-026-04262-4. Epub 2026 Mar 24. |
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Participants will receive a single IT injection of BIIB094 during Part A (SAD). |
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| Part B (MAD): BIIB094 Dose 1 | Experimental | Participants will receive a single IT injection of BIIB094 on multiple days during Part B [Multiple Ascending Dose (MAD)]. |
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| Part B (MAD): BIIB094 (Non LRRK2) Dose 2 | Experimental | Participants [Non leucine-rich repeat kinase 2 (Non LRRK2)] will receive a single IT injection of BIIB094 on multiple days during Part B (MAD). |
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| Part B (MAD): BIIB094 (LRRK2) Dose 2 | Experimental | Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD). |
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| Part B (MAD): BIIB094 (Non LRRK2) Dose 3 | Experimental | Participants (Non LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD). |
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| Part B (MAD): BIIB094 (LRRK2) Dose 3 | Experimental | Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD). |
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| Part A (SAD): Matching Placebo | Placebo Comparator | Participants will receive matching placebo during Part A [Single Ascending Dose (SAD)]. |
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| Part B (MAD): Matching Placebo | Placebo Comparator | Participants will receive matching placebo on multiple days during Part B (MAD). |
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| Placebo | Drug | Administered as specified in the treatment arm. |
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| Area Under the Concentration-Time Curve From Time Zero to Last Quantifiable Concentration (AUClast) of BIIB094 | Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 |
| Maximum Concentration (Cmax) of BIIB094 | Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 |
| Time to Reach Maximum Concentration (Tmax) of BIIB094 | Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 |
| Terminal Elimination Half-Life (t1/2) of BIIB094 | Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 |
| Farmington Hills |
| Michigan |
| 48334 |
| United States |
| The Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| Oregon Health and Science University | Portland | Oregon | 97239 | United States |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19107 | United States |
| Alliance for Multispecialty Research | Knoxville | Tennessee | 37920 | United States |
| Inland Northwest Research | Spokane | Washington | 99202 | United States |
| Research Site | Toronto | Ontario | Canada |
| Montreal Neurological Institute and Hospital | Montreal | Quebec | QC H3A 2B4 | Canada |
| Sourasky Medical Center | Tel Aviv | 64239 | Israel |
| Neuro-SysMed Center | Bergen | 5053 | Norway |
| St. Olav University Hospital | Trondheim | 7030 | Norway |
| Laboratorios de Investigación Biocruces 3., Hospital de Cruces | Barakaldo | Bizkaia | 48903 | Spain |
| Hospital General de Catalunya | Barcelona | Vizcaya | 08195 | Spain |
| Hospital Clinic de Barcelona | Barcelona | 08036 | Spain |
| Hospital General Universitario Gregorio Marañón | Madrid | 28007 | Spain |
| Research Site | Seville | Spain |
| Queen Square (Neurology) CRF Site Institute of Neurology & the National Hospital for Neurology and Neurosurgery UCLH | London | WC1N 3BG | United Kingdom |
| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |
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| ID | Term |
|---|---|
| C000730637 | BIIB094 |
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