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Parkinson DiseasePhase 3LIGHTHOUSENCT05418673

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

A Phase 3 study testing BIIB122 in people with Parkinson Disease, run by Biogen at 27 sites in 6 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 30 to 80 years
Conditions the study is for
Parkinson Disease
What is being tested?
BIIB122
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
7 participants
Where
27 sitesin 6 countries
Study dates
26 Aug 2022to 27 Jul 2023
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 30 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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.

Trial locations and contacts

27 sites in 6 countries.

27 sites in 6 countries

United States12 sites
  • University of Colorado

    Aurora, Colorado, 80045

  • Parkinson's Disease and Movement Disorders Center

    Boca Raton, Florida, 33486

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Rocky Mountain Movement Disorders Center, PC

    Englewood, Colorado, 80113

  • University of Kansas Medical Center Research Institute, Inc.

    Kansas City, Kansas, 66160

  • Evergreen Hospital Medical Center

    Kirkland, Washington, 98034

  • Mount Sinai Beth Israel

    New York, New York, 10003

  • Weill Medical College of Cornell University

    New York, New York, 10021

  • Pennsylvania Hospital

    Philadelphia, Pennsylvania, 19107

  • University of California San Francisco (UCSF)

    San Francisco, California, 94143

  • Inland Northwest Research

    Spokane, Washington, 99202

  • USF Health Byrd Institute

    Tampa, Florida, 33613

Other trials in United States

Spain6 sites
  • Hospital Universitari Vall d'Hebron

    Barcelona, 8035

  • Hospital Clinic de Barcelona

    Barcelona, 8036

  • Hospital Universitario Donostia

    Donostia / San Sebastian, Guipuzcoa, 20014

  • Hospital General de Catalunya

    Sant Cugat del Vallès, Barcelona, 8190

  • Hospital Universitario Marques de Valdecilla

    Santander, Cantabria, 38003

  • Hospital Universitario Virgen del Rocio

    Seville, 41013

Other trials in Spain

France5 sites
  • Centre Hospitalier Universitaire de Lyon-Hospices Civils de Lyon-Hopital Pierre Wertheimer

    Bron, Rhone, 69500

  • Hôpital de la Timone

    Marseille, Bouches-du-Rhône, 13385

  • Groupe Hospitalier Pitie-Salpetriere

    Paris, 75013

  • CHU Rennes - Hopital Pontchaillou

    Rennes, Ille Et Vilaine, 35033

  • Hopital Purpan

    Toulouse, Haute Garonne, 31059

Other trials in France

Germany2 sites
  • Universitaetsklinikum Carl Gustav Carus TU Dresden

    Dresden, 1307

  • Universitaetsklinikum Tuebingen

    Tübingen, Baden-Wurttemberg, 72076

Other trials in Germany

Italy1 site
  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133

Other trials in Italy

United Kingdom1 site
  • Ninewells Hospital

    Dundee, Tayside Region, DD1 9SY

Other trials in United Kingdom

About this study

Official title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Efficacy and Safety of BIIB122/DNL151 in Participants With Parkinson's Disease and Pathogenic LRRK2 Variants

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.

Read more

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.
Read the detailed description

BIIB122 is an investigational central nervous system-penetrant small molecule inhibitor of LRRK2 kinase

Being tested
BIIB122, BIIB122-Matching Placebo
Medical subject headings
Parkinson Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BIIB122Drug

    Also called DNL151

    Administered as specified in the treatment arm.

  • BIIB122-Matching PlaceboDrug

    Administered as specified in the treatment arm.

Study groups in NCT05418673
Study groupRoleWhat they receive
BIIB122 225 mgExperimentalParticipants will receive 225 mg of BIIB122 tablets, orally, once daily (QD) for up to 180 weeks.
Receives: Drug: BIIB122
BIIB122-Matching PlaceboPlacebo comparatorParticipants will receive BIIB122-matching placebo tablets, orally, QD for up to 180 weeks.
Receives: Drug: BIIB122-Matching Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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).

    Measured: From Week 96 to Week 180

Secondary outcomes (5)
  1. 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.

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

  2. 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.

    Measured: From Week 96 to Week 180

  3. 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.

    Measured: Baseline, Week 96

  4. 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.

    Measured: From Week 96 to Week 180

  5. 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.

    Measured: Baseline, Week 96

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Jun 2022
  2. Study start26 Aug 2022
  3. Primary completion27 Jul 2023
  4. Study completion27 Jul 2023

Why it stopped: Sponsor Decision

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
7 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05418673
Sponsor's protocol ID
283PD302
Other IDs
2022-000747-77
Acronym
LIGHTHOUSE
Status last verified
May 2024
First submitted
10 Jun 2022
Last updated
26 Jun 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing BIIB122 in people with Parkinson Disease, run by Biogen at 27 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 26 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 27 sites in United States, Spain, France, Germany, Italy and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Jun 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Time to Confirmed Worsening in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts II and III (time frame: From Week 96 to Week 180). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2024.

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