This study has been transitioned to CTIS with ID 2023-505645-12-00 check the CTIS register for the current data. Main objective: To evaluate the efficacy of BIIB122 225 mg compared with placebo.Secondary objective: To evaluate the efficacy, safety…
ID
Source
Brief title
Condition
- Movement disorders (incl parkinsonism)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Time to confirmed worsening in Movement Disorder Society Unified Parkinson*s
Disease Rating Scale (MDS-UPDRS) Parts II and III combined score over the
treatment period.
Secondary outcome
• Incidence of AEs and SAEs during the treatment period
• Time to confirmed worsening in MDS-UPDRS Part II score over the treatment
period
• Change in MDS-UPDRS Parts II and III combined score
• Time to confirmed worsening in Schwab and England Activities of Daily Living
Scale (SEADL) over the treatment period
• Change in MDS-UPDRS Parts I, II, and III combined score
Background summary
This Phase 2b, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study
is designed to determine the efficacy and safety of BIIB122 in Participants
with Parkinson*s Disease
Study objective
This study has been transitioned to CTIS with ID 2023-505645-12-00 check the CTIS register for the current data.
Main objective:
To evaluate the efficacy of BIIB122 225 mg compared with placebo.
Secondary objective:
To evaluate the efficacy, safety and tolerability of BIIB122 225 mg compared
with placebo in participants with early-stage PD
Objective optional (non-mandatory) biofluid sub-study
The objective of the biofluid substudy is to evaluate the change in central and
peripheral biomarkers over time. Collection will include repeat LPs for CSF
only or CSF and blood sampling for PBMC.
Study design
This Phase 2b, multicenter, randomized, double-blind, placebo-controlled,
parallel-group study is designed to evaluate the efficacy and safety of BIIB122
in participants with early-stage PD. The study population is defined as
participants with a clinical diagnosis of PD within the past 2 years who do not
carry a pathogenic variant in the LRRK2 gene.
Optional (non-mandatory) biofluid sub-study
A biofluid substudy may be conducted to evaluate exploratory biofluid
biomarkers in a subset of participants.All biofluid substudy participants are
planned to participate in the CSF portion of the substudy. A subset of those
participants that particpate in the CSF portion of the substudy will also
participate in the PBMC portion of the substudy. Samples will be evaluated for
measures of LRRK2 pathway, lysosomal function, and exploratory measures
relevant to PD.
Optional (non-mandatory) genetic research
Participants will be offered the option for residual DNA samples to be retained
for future exploratory genetic research that may be used to understand the
biology of diseases and traits of interest to the Sponsor and/or to develop
diagnostic and analytical tests.Participation in genetic research does not
involve additional visits for the participant.
Intervention
Eligible participants will be randomly assigned in a 1:1 ratio to receive
BIIB122 225 mg or matching placebo tablets orally QD during the double-blind,
placebo-controlled treatment period.
Study burden and risks
Participants will participate in the study for the duration up to 152 weeks.
Subjects will need to come to the hospital more often than they normally would
and they undergo additional tests. These include physical and neurological
examination, Dat/SPECT scan, respiratory assessments, ECG*s, pregnancy tests,
urine/blood tests and questionnaires. Subjects will receive medication orally
and subjects cannot be pregnant at the start or during the study. Aside from
these interventions, participation in this study involves blood draws
(venapunction) and in the course of the study 390 ml up to 535 ml blood will be
taken.
Risks associated with the study drugs include allergy-related reactions. Common
side effects of BIIB122 include headache, fatigue (feeling tired), nausea
(feeling of having to vomit), vomiting, muscle pain, dizziness, back pain,
diarrhea, nasopharyngitis (common cold), flu-like symptoms and difficulty in
sleeping. Subject may also feel discomfort during some of the tests.
Furthermore the subjects more receptive to risks such as;
• Blood collection: Pain and/or bruising at the needle site of puncture.
Although very rare, localized clot formation and infections may occur.
• ECG: skin irritation is rare but could occur from the electrodes or gel that
is used.
• Mental health assessments: As some of the questionnaires/scales used in this
study will ask questions about subjects feelings, mental health and well-being,
subject experience these as distressing.
In conclusion, the risks identified from nonclinical and clinical safety
studies are manageable. Given the unmet need for a therapeutic treatment that
may slow the progression of PD, BIIB122 has an overall favorable benefit-risk
profile supporting further continued development of BIIB122 in PD patients.
Binney Street 225
Cambridge MA 02142
US
Binney Street 225
Cambridge MA 02142
US
Listed location countries
Age
Inclusion criteria
Key Inclusion Criteria:
• Clinical diagnosis of PD meeting the Movement Disorder Society Clinical
Diagnostic Criteria within 2 years of the screening visit, inclusive, and at
least 30 years of age at the time of diagnosis
• Modified Hoehn and Yahr scale, stages 1 to 2 (in OFF state), inclusive
• MDS-UPDRS Parts II and III (in OFF state) combined score less than or equal
to (<=)40 at screening
• Screening genetic test results verifying the absence of a pathogenic
leucine-rich repeat kinase 2 (LRRK2) variant (i.e., G2019S, N1437H, R1441G,
R1441C, R1441H, Y1699C, or I2020T). Participants with additional LRRK2 variants
may be excluded if data emerge to convincingly support an association of the
variants with LRRK2-PD pathogenicity.
NOTE: Other protocol defined Inclusion criteria may apply. See Protocol section
6.1.
Exclusion criteria
Key Exclusion Criteria:
• Clinically significant neurological 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.
• Montreal Cognitive Assessment (MoCA) score <24 at the screening visit
NOTE: Other protocol defined Exclusion criteria may apply. See Protocol section
6.2
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EU-CTR | CTIS2023-505645-12-00 |
| EudraCT | EUCTR2021-004849-20-NL |
| ClinicalTrials.gov | NCT05348785 |
| CCMO | NL80677.056.22 |