Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2022-000747-77 | EudraCT Number |
Not provided
Not provided
Sponsor Decision
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Denali Therapeutics Inc. | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
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.
Researchers will also learn more about the safety of BIIB122.
A description of how the study will be done is given below.
BIIB122 is an investigational central nervous system-penetrant small molecule inhibitor of LRRK2 kinase
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BIIB122 225 mg | Experimental | Participants will receive 225 mg of BIIB122 tablets, orally, once daily (QD) for up to 180 weeks. |
|
| BIIB122-Matching Placebo | Placebo Comparator | Participants will receive BIIB122-matching placebo tablets, orally, QD for up to 180 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BIIB122 | Drug | Administered as specified in the treatment arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Time to Confirmed Worsening in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts II and III | 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). | From Week 96 to Week 180 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | 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. |
Not provided
Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California San Francisco (UCSF) | San Francisco | California | 94143 | United States | ||
| University of Colorado |
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Not provided
Not provided
Not provided
A total of 7 participants were enrolled and treated in the study, but none of the participants completed it.
Participants took part in the study at multiple investigative sites in the United States and the United Kingdom from 26 Aug 2022 to 27 Jul 2023.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants received BIIB122-matching placebo tablets, orally, once daily (QD) for up to 290 days. |
| FG001 | BIIB122 225 mg | Participants received BIIB122 225 milligrams (mg) tablets, orally, QD for up to 290 days. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Apr 9, 2023 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| BIIB122-Matching Placebo | Drug | Administered as specified in the treatment arm. |
|
| From the first dose (Day 1) of the study drug up to the end of follow-up (up to 336 days) |
| Time to Confirmed Worsening in MDS-UPDRS Part II Score | 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. | From Week 96 to Week 180 |
| Change From Baseline in MDS-UPDRS Parts II and III Combined Score at 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. | Baseline, Week 96 |
| Time to Confirmed Worsening in Modified Schwab and England Activities of Daily Living Scale (mSE-ADL) Score | 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. | From Week 96 to Week 180 |
| Change From Baseline in MDS-UPDRS Parts I, II, and III Combined Score at 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. | Baseline, Week 96 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Rocky Mountain Movement Disorders Center, PC | Englewood | Colorado | 80113 | United States |
| Parkinson's Disease and Movement Disorders Center | Boca Raton | Florida | 33486 | United States |
| USF Health Byrd Institute | Tampa | Florida | 33613 | United States |
| University of Kansas Medical Center Research Institute, Inc. | Kansas City | Kansas | 66160 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Mount Sinai Beth Israel | New York | New York | 10003 | United States |
| Weill Medical College of Cornell University | New York | New York | 10021 | United States |
| Pennsylvania Hospital | Philadelphia | Pennsylvania | 19107 | United States |
| Evergreen Hospital Medical Center | Kirkland | Washington | 98034 | United States |
| Inland Northwest Research | Spokane | Washington | 99202 | United States |
| Hôpital de la Timone | Marseille | Bouches-du-Rhône | 13385 | France |
| Hopital Purpan | Toulouse | Haute Garonne | 31059 | France |
| CHU Rennes - Hopital Pontchaillou | Rennes | Ille Et Vilaine | 35033 | France |
| Centre Hospitalier Universitaire de Lyon-Hospices Civils de Lyon-Hopital Pierre Wertheimer | Bron | Rhone | 69500 | France |
| Groupe Hospitalier Pitie-Salpetriere | Paris | 75013 | France |
| Universitaetsklinikum Tuebingen | Tübingen | Baden-Wurttemberg | 72076 | Germany |
| Universitaetsklinikum Carl Gustav Carus TU Dresden | Dresden | 1307 | Germany |
| Fondazione IRCCS Istituto Neurologico Carlo Besta | Milan | 20133 | Italy |
| Hospital General de Catalunya | Sant Cugat del Vallès | Barcelona | 8190 | Spain |
| Hospital Universitario Marques de Valdecilla | Santander | Cantabria | 38003 | Spain |
| Hospital Universitario Donostia | Donostia / San Sebastian | Guipuzcoa | 20014 | Spain |
| Hospital Universitari Vall d'Hebron | Barcelona | 8035 | Spain |
| Hospital Clinic de Barcelona | Barcelona | 8036 | Spain |
| Hospital Universitario Virgen del Rocio | Seville | 41013 | Spain |
| Ninewells Hospital | Dundee | Tayside Region | DD1 9SY | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Full analysis set (FAS) included all randomized participants who had received at least 1 dose of study treatment. Since a low number of participants were enrolled in the study and some of the participants have been rolled over to the 283PD201 (NCT05348785), the data was reported as a pooled group to avoid the identification of individual participants and to maintain the blinding.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | BIIB 122 or Matching-placebo (Pooled) | Participants either received BIIB122 225 mg or BIIB122-matching placebo tablets, orally, QD for up to 290 days. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Time to Confirmed Worsening in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts II and III | 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). | Based on the low enrolment number and to protect and maintain participant privacy/confidentiality, no data is reported, as only 1 participant completed post-baseline visit for clinical efficacy outcomes. | Posted | From Week 96 to Week 180 |
|
| ||||||||||||||||||||||
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | 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. | Safety analysis set included all randomized participants who had received at least 1 dose of study treatment. | Posted | Count of Participants | Participants | From the first dose (Day 1) of the study drug up to the end of follow-up (up to 336 days) |
| |||||||||||||||||||||
| Secondary | Time to Confirmed Worsening in MDS-UPDRS Part II Score | 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. | Based on the low enrolment number and to protect and maintain participant privacy/confidentiality, no data is reported, as only 1 participant completed post-baseline visit for clinical efficacy outcomes. | Posted | From Week 96 to Week 180 |
|
| ||||||||||||||||||||||
| Secondary | Change From Baseline in MDS-UPDRS Parts II and III Combined Score at 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. | Due to the early termination of the study (before week 48), sufficient data were not collected for the outcome measure analysis. | Posted | Baseline, Week 96 |
|
| ||||||||||||||||||||||
| Secondary | Time to Confirmed Worsening in Modified Schwab and England Activities of Daily Living Scale (mSE-ADL) Score | 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. | Based on the low enrolment number and to protect and maintain participant privacy/confidentiality, no data is reported, as only 1 participant completed post-baseline visit for clinical efficacy outcomes. | Posted | From Week 96 to Week 180 |
| |||||||||||||||||||||||
| Secondary | Change From Baseline in MDS-UPDRS Parts I, II, and III Combined Score at 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. | Due to the early termination of the study (before week 48), sufficient data were not collected for the outcome measure analysis. | Posted | Baseline, Week 96 |
|
From the first dose (Day 1) of the study drug up to the end of follow-up (up to 336 days)
Safety analysis set included all the randomized participants who had received at least 1 dose of study treatment. Since a low number of participants were enrolled in the study and some of the participants have been rolled over to the 283PD201 (NCT05348785), the data was reported as a pooled group and preferred terms are reported as system organ class to avoid the identification of individual participants and to maintain the blinding.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | BIIB 122 or Matching-placebo (Pooled) | Participants either received BIIB122 225 mg or BIIB122-matching placebo tablets, orally, QD for up to 290 days. | 0 | 7 | 0 | 7 | 5 | 7 |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ear and Labyrinth Disorders | Ear and labyrinth disorders | MedDRA 26 | Systematic Assessment |
| |
| Infections and Infestations | Infections and infestations | MedDRA 26 | Systematic Assessment |
| |
| Injury, Poisoning and Procedural Complications | Injury, poisoning and procedural complications | MedDRA 26 | Systematic Assessment |
| |
| Musculoskeletal and Connective Tissue Disorders | Musculoskeletal and connective tissue disorders | MedDRA 26 | Systematic Assessment |
| |
| Nervous System Disorders | Nervous system disorders | MedDRA 26 | Systematic Assessment |
| |
| Psychiatric Disorders | Psychiatric disorders | MedDRA 26 | Systematic Assessment |
| |
| Skin and Subcutaneous Tissue Disorders | Skin and subcutaneous tissue disorders | MedDRA 26 | Systematic Assessment |
|
Sponsor decided to stop study based on operational and strategic considerations and not for reasons related to efficacy/safety. Due to the early cessation of the study before participants reached the time window for efficacy assessments, efficacy analysis was not possible.
Our agreement is subject to confidentiality but generally the PI can publish, for non-commercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| US Biogen Clinical Trial Center | Biogen | 866-633-4636 | clinicaltrials@biogen.com |
| Feb 27, 2024 |
| Prot_SAP_000.pdf |
Not provided
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| C000730636 | DNL151 |
Not provided
Not provided
Not provided
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
| Units | Counts |
|---|---|
| Participants |
|