Trial Outcomes & Findings for 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 (NCT NCT05418673)

Results Overview

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).

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

7 participants

Primary outcome timeframe

From Week 96 to Week 180

Results posted on

2024-06-26

Participant Flow

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.

A total of 7 participants were enrolled and treated in the study, but none of the participants completed it.

Participant milestones

Participant milestones
Measure	Placebo Participants received BIIB122-matching placebo tablets, orally, once daily (QD) for up to 290 days.	BIIB122 225 mg Participants received BIIB122 225 milligrams (mg) tablets, orally, QD for up to 290 days.
Overall Study STARTED	3	4
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	3	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo Participants received BIIB122-matching placebo tablets, orally, once daily (QD) for up to 290 days.	BIIB122 225 mg Participants received BIIB122 225 milligrams (mg) tablets, orally, QD for up to 290 days.
Overall Study Study Terminated by Sponsor	2	4
Overall Study Lost to Follow-up	1	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BIIB 122 or Matching-placebo (Pooled) n=7 Participants Participants either received BIIB122 225 mg or BIIB122-matching placebo tablets, orally, QD for up to 290 days.
Age, Continuous	66.9 years STANDARD_DEVIATION 10.7 • n=93 Participants
Sex: Female, Male Female	2 Participants n=93 Participants
Sex: Female, Male Male	5 Participants n=93 Participants
Race/Ethnicity, Customized Race · White	7 Participants n=93 Participants
Race/Ethnicity, Customized Ethnicity · Not Hispanic or Latino	7 Participants n=93 Participants

PRIMARY outcome

Timeframe: From Week 96 to Week 180

Population: 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.

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).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From the first dose (Day 1) of the study drug up to the end of follow-up (up to 336 days)

Population: Safety analysis set included all randomized participants who had received at least 1 dose of study treatment.

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.

Outcome measures

Outcome measures
Measure	Placebo n=3 Participants Participants received BIIB122-matching placebo tablets, orally, QD for up to 290 days.	BIIB122 225 mg n=4 Participants Participants received BIIB122 225 mg tablets, orally, QD for up to 290 days.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) AEs	2 Participants	3 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) SAEs	0 Participants	0 Participants

SECONDARY outcome

Timeframe: From Week 96 to Week 180

Population: 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.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 96

Population: Due to the early termination of the study (before week 48), sufficient data were not collected for the outcome measure analysis.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Week 96 to Week 180

Population: 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.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 96

Population: Due to the early termination of the study (before week 48), sufficient data were not collected for the outcome measure analysis.

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.

Outcome measures

Outcome data not reported

Adverse Events

BIIB 122 or Matching-placebo (Pooled)

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	BIIB 122 or Matching-placebo (Pooled) n=7 participants at risk Participants either received BIIB122 225 mg or BIIB122-matching placebo tablets, orally, QD for up to 290 days.
Ear and labyrinth disorders Ear and Labyrinth Disorders	14.3% 1/7 • 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.
Infections and infestations Infections and Infestations	14.3% 1/7 • 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.
Injury, poisoning and procedural complications Injury, Poisoning and Procedural Complications	28.6% 2/7 • 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.
Musculoskeletal and connective tissue disorders Musculoskeletal and Connective Tissue Disorders	14.3% 1/7 • 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.
Nervous system disorders Nervous System Disorders	14.3% 1/7 • 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.
Psychiatric disorders Psychiatric Disorders	14.3% 1/7 • 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.
Skin and subcutaneous tissue disorders Skin and Subcutaneous Tissue Disorders	14.3% 1/7 • 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.

Additional Information

US Biogen Clinical Trial Center

Biogen

Phone: 866-633-4636

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER