A Study to Assess if BIIB122 Tablets Are Safe and Can Slow Worsening of Early-Stage Parkinson's Disease in Participants With Specific LRRK2 Genetic Variants Between the Ages of 30 and 80 Using the Movement Disorder Society-Unified Parkinson's Disease Rating Scale
- Conditions
- Parkinson Disease
- Interventions
- Drug: BIIB122Drug: BIIB122-Matching Placebo
- Registration Number
- NCT05418673
- Lead Sponsor
- Biogen
- Brief Summary
In this study, researchers will learn more about a study drug called BIIB122 in participants with early-stage Parkinson's disease (PD). The study will focus on participants with a specific genetic variant in their LRRK2 gene.
The main question researchers are trying to answer is if taking BIIB122 slows the worsening of PD more than placebo in the early stages of PD.
To help answer this question, researchers will use a questionnaire called the Movement Disorder Society-Unified Parkinson's Disease Rating Scale, also known as the MDS-UPDRS.
* The MDS-UPDRS measures impairment and disability in people living with PD. It was created in the 1980s and is one of the most used rating scales for PD symptoms.
* The MDS-UPDRS has 4 parts, and a higher score means more severe PD symptoms.
* Part I assesses non-motor experiences of daily living, including but not limited to memory loss, problems sleeping, pain, depression, and anxiety.
* Part II measures motor experiences of daily living.
* Part III is the results of a motor symptoms exam by a medical professional.
* Part IV records PD complications caused by motor symptoms.
Researchers will also learn more about the safety of BIIB122.
A description of how the study will be done is given below.
* Participants will take BIIB122 or a placebo as tablets by mouth. A placebo looks like the study drug but contains no real medicine.
* Participants will be in the study for 103 weeks to 187 weeks. This includes the screening and follow-up periods.
* Participants will take BIIB122 or placebo 1 time a day for 96 to 180 weeks.
* Participants can continue to take certain medications for PD. Participants must be on the same dose of medication for at least 90 days before the study begins.
* Participants will visit the clinic less often as the study continues, ranging every 4 weeks to every 24 weeks.
- Detailed Description
BIIB122 is an investigational central nervous system-penetrant small molecule inhibitor of LRRK2 kinase
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 7
- Clinical diagnosis of PD meeting the Movement Disorder Society Clinical Diagnostic Criteria within 5 years of the Screening Visit, inclusive, and at least 30 years of age at the time of diagnosis
- Modified Hoehn and Yahr scale (mHY), Stages 1 to 2.5 (in OFF state), inclusive, at Screening
- MDS-UPDRS Parts II and III (in OFF state) combined score ≤40 at Screening
- Screening genetic test results verifying the presence of a pathogenic leucine-rich repeat kinase 2 (LRRK2) variant
Key
- Clinically significant neurologic disorder other than PD, including, but not limited to, stroke, dementia, or seizure within 5 years of Screening Visit, in the opinion of the Investigator
- Clinical evidence of atypical parkinsonism (e.g., multiple-system atrophy or progressive supranuclear palsy) or evidence of drug-induced parkinsonism
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description BIIB122 225 mg BIIB122 Participants will receive 225 mg of BIIB122 tablets, orally, once daily (QD) for up to 180 weeks. BIIB122-Matching Placebo BIIB122-Matching Placebo Participants will receive BIIB122-matching placebo tablets, orally, QD for up to 180 weeks.
- Primary Outcome Measures
Name Time Method Time to Confirmed Worsening in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts II and III From Week 96 to Week 180 Time to confirmed worsening is defined as a worsening event sustained over 2 consecutive assessments. MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. Part II assesses motor experiences of daily living (range 0-52). It contains 13 questions which are to be completed by the participant. Part III assesses the motor signs of PD and is administered by the rater (range 0-132). Part III contains 33 scores based on 18 items. For each question, a numeric score is assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Part II and III combined score equals the sum of Parts II and III (range 0-184). A higher score indicates more severe symptoms of Parkinson's disease (PD).
- Secondary Outcome Measures
Name Time Method Time to Confirmed Worsening in Modified Schwab and England Activities of Daily Living Scale (mSE-ADL) Score From Week 96 to Week 180 Time to confirmed worsening is defined as a worsening event sustained over 2 consecutive assessments. The mSE-ADL scale reflects the speed, ease, and independence with which an individual performs daily activities or personal chores with 100% indicating total independence, falling to 0%, which indicates a state of complete dependence. The individual is asked to rate his or her function using an 11-point scale (10% increments), from 100% (completely independent; able to do all chores without slowness, difficulty, or impairment; essentially normal; unaware of any difficulty) to 0% (vegetative functions such as swallowing, bladder and bowels are not functioning; bedridden). The lower the score, the worse the functional status.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) From the first dose (Day 1) of the study drug up to the end of follow-up (up to 336 days) An adverse event (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 AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. 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); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or is a medically important event.
Time to Confirmed Worsening in MDS-UPDRS Part II Score From Week 96 to Week 180 Time to confirmed worsening is defined as a worsening event sustained over 2 consecutive assessments. MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. Part II assesses motor experiences of daily living (range 0-52). It contains 13 questions which are to be completed by the participant. For each question, a numeric score is assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Part II score ranges from 0-52. A higher score indicates more severe symptoms of PD.
Change From Baseline in MDS-UPDRS Parts II and III Combined Score at Week 96 Baseline, Week 96 MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of 4 parts. Part II assesses motor experiences of daily living (range 0-52). It contains 13 questions which are to be completed by the participant. Part III assesses the motor signs of PD and is administered by the rater (range 0-132). Part III contains 33 scores based on 18 items. For each question, a numeric score is assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. MDS-UPDRS Part II and III combined score equals the sum of Parts II and III (range 0-184). A higher score indicates more severe symptoms of PD.
Change From Baseline in MDS-UPDRS Parts I, II, and III Combined Score at Week 96 Baseline, Week 96 The MDS-UPDRS is a multimodal scale consisting of four parts. Part I assessed non-motor experiences of daily living and has 2 components (Range 0-52). Part IA contains 6 questions and are assessed by the examiner (Range 0-24). Part IB contains 7 questions on non-motor experiences of daily living which was completed by the participant (Range 0-28). Part II assessed motor experiences of daily living (Range 0-52). It contained 13 questions completed by the participant. Part III assessed the motor signs of PD and was administered by the rater (Range 0-132). Part III contained 33 scores based on 18 items. For each question a numeric score is assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. The MDS-UPDRS Total Score equals the sum of Parts I,II, and III (Range: 0-236). A higher score indicated more severe symptoms of PD.
Trial Locations
- Locations (27)
Rocky Mountain Movement Disorders Center, PC
🇺🇸Englewood, Colorado, United States
Parkinson's Disease and Movement Disorders Center
🇺🇸Boca Raton, Florida, United States
Pennsylvania Hospital
🇺🇸Philadelphia, Pennsylvania, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Ninewells Hospital
🇬🇧Dundee, Tayside Region, United Kingdom
University of California San Francisco (UCSF)
🇺🇸San Francisco, California, United States
USF Health Byrd Institute
🇺🇸Tampa, Florida, United States
Hospital Universitario Donostia
🇪🇸San Sebastian, Guipuzcoa, Spain
Weill Medical College of Cornell University
🇺🇸New York, New York, United States
Universitaetsklinikum Tuebingen
🇩🇪Tuebingen, Baden Wuerttemberg, Germany
University of Colorado
🇺🇸Aurora, Colorado, United States
CHU Rennes - Hopital Pontchaillou
🇫🇷Rennes cedex 09, Ille Et Vilaine, France
Hôpital de la Timone
🇫🇷Marseille, Bouches-du-Rhône, France
Evergreen Hospital Medical Center
🇺🇸Kirkland, Washington, United States
Groupe Hospitalier Pitie-Salpetriere
🇫🇷Paris, France
Hospital Universitario Marques de Valdecilla
🇪🇸Santander, Cantabria, Spain
Hospital Universitari Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital Clinic de Barcelona
🇪🇸Barcelona, Spain
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Spain
Universitaetsklinikum Carl Gustav Carus TU Dresden
🇩🇪Dresden, Germany
University of Kansas Medical Center Research Institute, Inc.
🇺🇸Kansas City, Kansas, United States
Mount Sinai Beth Israel
🇺🇸New York, New York, United States
Hopital Purpan
🇫🇷Toulouse, Haute Garonne, France
Fondazione IRCCS Istituto Neurologico Carlo Besta
🇮🇹Milano, Italy
Hospital General de Catalunya
🇪🇸Sant Cugat del Valles, Barcelona, Spain
Centre Hospitalier Universitaire de Lyon-Hospices Civils de Lyon-Hopital Pierre Wertheimer
🇫🇷Bron, Rhone, France
Inland Northwest Research
🇺🇸Spokane, Washington, United States
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