New pill aims to slow Parkinson's in patients with genetic mutation

NCT ID NCT05819359

Disease control Sponsor: Bial R&D Investments, S.A. Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests an experimental drug called BIA 28-6156 in people with Parkinson's disease who have a specific change in the GBA1 gene. The goal is to see if the drug can delay the worsening of movement-related daily living skills over 78 weeks. About 237 participants will receive either the drug or a placebo, and researchers will track their symptoms using standard rating scales.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: BIA 28-6156 (an oral drug taken once daily)
What this could lead to: If successful, this could lead to a treatment that slows the worsening of motor symptoms in people with Parkinson's who have a specific genetic variant (GBA1).
What could go wrong: This is a Phase 2 trial, so it is still early. The drug may not prove effective or could have side effects. Results may not apply to all Parkinson's patients, only those with the GBA1 gene variant.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 237 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2023

Expected to finish

Jul 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

35 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: Subjects who satisfy all of the following criteria will be eligible for Part A (Genetic Screening) of the study: * The subject is ≥35 and ≤80 years of age at the time of informed consent. * The subject has a clinical diagnosis of PD for at least 1 year and for no longer than 7 years before initiation of screening (for Part A), as confirmed by a neurologist using the MDS Criteria for Parkinson's Disease. * The subject has a modified Hoehn and Yahr score ≤2.5. * The subject is receiving symptomatic treatment for PD. * The subject is capable of giving signed informed consent. Subjects who satisfy all the following criteria will be eligible for Part B (Double-Blind Treatment) of the study: * Informed Consent - The subject is capable of giving signed informed consent. * The subject has a known GBA-PD risk-associated variant (as determined in Part A \[Genetic Screening\] of this study). * The subject has a score ≥22 on the Montreal Cognitive Assessment (MoCA) scale. * The subject does not have severe motor fluctuations or disabling dyskinesias in the clinical judgment of the investigator. * The subject has been on stable doses of PD medications for at least 30 days (at least 60 days for rasagiline) before initiation of screening in Part B (Double-Blind Treatment). * The subject is able to comply with the study restrictions. * The subject has a body mass index (BMI) of 18 to 40 kg/m2. * If a sexually active man or a women of childbearing potential, the subject agrees to use highly effective birth control or to remain abstinent during the trial and for 30 days after the last dose of IMP. Complete abstinence from sexual intercourse if this is the subject's usual and preferred lifestyle; or sexual partner with surgical sterilization (e.g., tubal ligation, hysterectomy and/or bilateral oophorectomy, vasectomy). Exclusion Criteria: • Individuals who do not satisfy the inclusion criteria for Part A (Genetic Screening) will be excluded. Subjects who meet any of the following criteria for Part B (Double-Blind Treatment) are not eligible for the study. * The subject has Gaucher's disease (GD), as defined by clinical signs and symptoms (i.e., hepatosplenomegaly, cytopenia, skeletal disease), and/or a medical history of marked deficiency of GCase activity compatible with GD. * The subject is homozygous for a GBA1 pathogenic variant that is known to be associated with GD or compound heterozygous for 2 alleles that are known to be associated with GD. * The subject carries a known PD-associated LRRK2 pathogenic variant. * The subject has atypical or secondary parkinsonism by medical history or in the opinion of the investigator. Atypical parkinsonism includes, but is not limited to, diagnoses of progressive supranuclear palsy, cortico-basal syndrome, and multiple system atrophy. Secondary parkinsonism includes drug-induced, toxin-induced, postinfectious, posttraumatic, or vascular parkinsonism. * The subject has a history of (within 60 days before initiation of screening) or has planned upcoming major surgery that could interfere with, or for which the treatment might interfere with, the conduct of the study or that would pose an unacceptable risk to the subject in the opinion of the investigator. * The subject has any active or chronic disease or condition other than PD that could interfere with, or for which the treatment might interfere with, the conduct of the study or pose an unacceptable risk to the subject in the opinion of the investigator based on medical history, physical examination, vital signs, 12-lead ECG, or clinical laboratory tests. Minor deviations of laboratory values from the normal range may be acceptable if judged by the investigator to have no/minor clinical relevance. * The subject has a recent history (last 6 months) of abuse of addictive substances (alcohol, illegal substances), currently uses \>21 units of alcohol per week, or is a regular recreational user of sedatives, hypnotics, tranquillizers, or any other addictive agent in the opinion of the investigator. * The subject has a positive test for drugs of abuse at screening or before administration of the first dose of investigational medicinal product (IMP) that the investigator judges as clinically relevant. A positive test for tetrahydrocannabinol (THC) is exclusionary. A positive test for cannabinoids (not containing THC) is not exclusionary if the subject is a recreational user (not an abuser) of cannabinoids, in the opinion of the investigator, and agrees to abstain from using cannabinoids within 12 hours before study visits. A positive drug screen that is attributed to an allowed prescription drug is not exclusionary but should be agreed with the medical monitor. * The subject is currently pregnant, is planning pregnancy within the timeframe of the study, or is breastfeeding. * The subject is using a strong inhibitors and inducers CYP3A4 at the time of screening for Part B (Double-Blind Treatment). * The subject is using a breast cancer resistance protein (BCRP) substrate (e.g., pravastatin, rosuvastatin, glyburide) at the time of screening for Part B (Double-Blind Treatment). * The subject has used any of the following medications within 60 days before Baseline: typical or atypical antipsychotics (including, but not limited to, clozapine, pimavanserin, olanzapine, risperidone, and aripiprazole), metoclopramide, prochlorperazine, methyldopa, tetrabenazine, deutetrabenazine, valbenazine, or reserpine. * The subject has received a vaccination within 14 days before administration of the first dose of IMP. * The subject has a prior history of or there is a plan to conduct deep brain stimulation (DBS), lesional procedures, (i.e., thalamotomy), or focused ultrasound; to initiate gene therapy treatment for PD; or to initiate use of any formulation of intestinal infusion or continuous subcutaneous infusion of PD medications. * The subject is currently participating in or has participated in an investigational drug study within 3 months or 5 half-lives, whichever is longer; in a therapeutic device study within 3 months before the first dose of IMP; or has previously participated in a gene therapy trial. Concurrent participation in an observational study is acceptable. * The subject has a positive test result for hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (anti-HCV), or human immunodeficiency virus 1 (HIV-1) or 2 (HIV-2) at screening. If reflex testing for hepatitis B or HCV DNA is negative, the subject may be eligible for the study. * The subject has renal insufficiency as defined by an estimated glomerular filtration rate (eGFR) of \<60 mL/min at screening. * The subject has cirrhosis (Child-Pugh A, B, or C) or any of the following laboratory values at screening: serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2 times the upper limit of normal (ULN) or bilirubin \>2 × ULN except if the subject has known or suspected Gilbert's disease. * The subject has a QT interval corrected for heart rate by Fridericia's method (QTcF) value \>450 msec if male or \>470 msec if female at screening. * The subject provides a positive response on Question 4 or 5 of the Columbia-Suicide Severity Rating Scale (C-SSRS) based on the last 6 months or, in the opinion of the investigator, presents a serious risk of suicide at screening. * The subject had a positive severe acute respiratory syndrome-related coronavirus-2 (SARS-CoV-2) test (any type) result within the 30 days before signing informed consent for Part B (Double-Blind Treatment) or has 2 or more current symptoms (e.g., sore throat, cough, fever) at the same time that are consistent with the Coronavirus disease 2019 (COVID-19) infection (not tested) in the opinion of the investigator. * The subject has a clinical history that is consistent with a previous COVID-19 infection and has not recovered fully, maintaining nonspecific symptoms like, for example, fatigue, shortness of breath, difficulty concentrating, sleep disorders, fever, anxiety, and depression. * The subject has previously received BIA 28-6156 or has a known allergy or hypersensitivity to BIA 28-6156 or any components of the formulation. * The subject is an unsuitable candidate to receive BIA 28-6156 or is unable or unlikely to comply with the dosing schedule or study evaluations in the judgment of the investigator.

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Conditions

The condition(s) this trial relates to.

basal ganglia disorder central nervous system disorder GBA1-related Parkinson disease, susceptibility Motor Activity movement disorder neurodegenerative disease Parkinson disease parkinsonian disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Parkinson's disease

Contacts and locations

Locations

A.O.U. San Giovanni di Dio Ruggi d'Aragona Centro Parkinson- Piano Rialzato Corpo QT

Salerno, 84125, Italy
Amsterdam Medical Center UMC

Amsterdam, Netherlands
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Pitie-Salpetriere - Centres d'Investigation Clinique (CIC) Paris-Est

Paris, France
Barrow Neurological Institute

Phoenix, Arizona, 85013, United States
Baylor College of Medicine

Houston, Texas, 77030, United States
Baylor University Medical Center

Baltimore, Maryland, 21287, United States
Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02115, United States
CHU de Nantes - Hopital Nord Laennec

Nantes, France
CHU de Nice Hopital Pasteur

Nice, 6002, France
CHU de Nimes

Nîmes, France
CHU de Rennes Hopital Pontchaillou

Rennes, France
CIC Toulouse

Toulouse, France
CNS - Campus Neurologico

Torres Vedras, Portugal
Cedars-Sinai

Los Angeles, California, 90048, United States
Centro Hospitalar Universitario de Coimbra

Coimbra, Portugal
Centro Hospitalar Universitario de Santo Antonio

Porto, 4099-001, Portugal
Centrum Medyczne NEUROMED Sp. z o.o. ul.

Bydgoszcz, 85-163, Poland
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
Clinique Neuro-Outaouais (Neuro-Outaouais Clinic)

Gatineau, Quebec, Canada
Columbia University Medical Center

New York, New York, 10032, United States
Evergreen Neuroscience Institute

Kirkland, Washington, 98034, United States
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy
Gertrudis Clinic Biskirchen, Parkinson-Center

Biskirchen, 35638, Germany
Glasgow Memory Clinic

Glasgow, United Kingdom
Hopital Paule de Viguier

Toulouse, France
Hospital Ruber Internacional

Madrid, 28034, Spain
Hospital S.JOÃO

Porto, 4200-319, Portugal
Hospital Senhora da Oliveira de Guimaraes

Guimarães, 4835-044, Portugal
Hospital Universitaio de La Princesa

Madrid, 28006, Spain
Hospital Universitari Germans Trias i Pujol

Badalona, 08916, Spain
Hospital Universitario Cruces

Barakaldo, 48903, Spain
Hospital Universitario Virgen del Rocio

Seville, 41013, Spain
Hospital Vall D´Hebron

Barcelona, 08035, Spain
Hospital de la Santa Creu I Sant Pau

Barcelona, 08025, Spain
IRCCS Carlo Besta Neurological Institute

Milan, 20133, Italy
IRCCS Istituto Delle Scienze Neurologiche DI

Bologna, Italy
IRCSS San Raffaele Pisana

Roma, 163, Italy
Icahn School of Medicine at Mount Sinai Beth Israel

New York, New York, 10003, United States
Inland Northwest Research

Spokane, Washington, 99202, United States
Intermountain Healthcare

Salt Lake City, Utah, 84107, United States
Istituto Clinico Humanitas

Rozzano, 20086, Italy
King's College London - David Goldberg Centre

London, United Kingdom
Klinikum der Universität München, Campus Grosshadern, Neurologische Klinik und Poliklinik

Munich, 81377, Germany
Krakowkska Akademia Neurologii Sp. z o.o

Krakow, 31-505, Poland
Ludwig-Maximilians University Munich

Munich, Germany
MUSC

Charleston, South Carolina, 29425, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Mayo Clinic

Rochester, Minnesota, 55905, United States
Montreal Neurological Institute & Hospital

Montreal, Quebec, 3801, Canada
Morehouse School of Medicine

Atlanta, Georgia, 30310, United States
NHS Tayside-Ninewells Hospital and Medical School

Dundee, United Kingdom
NeuroKlinika Gabinet Lekarski

Lodz, 90-640, Poland
Neurologisches Fachkrankenhaus für, Bewegungsstörungen und Parkinson

Beelitz-Heilstätten, 14547, Germany
Neurologmottagningen, QD 62

Uppsala, Sweden
Northwell Health

New York, New York, 10075, United States
Northwell Health Physician Partners

New York, New York, 10075-1851, United States
Northwestern University

Chicago, Illinois, 60611, United States
Oregon Health and Science University

Portland, Oregon, 97239, United States
Ospedale Antonio Perrino

Brindisi, 72100, Italy
Ottawa Hospital Research Institute

Ottawa, Canada
Paracelsus-Elena-Klinik

Kassel, 34128, Germany
Park Nicollet Struther's Parkinson's Center (Struthers Parkinsons Center at HealthPartners)

Saint Paul, Minnesota, 55427, United States
Parkinson's Center and Movement Disorders of Boca Raton

Boca Raton, Florida, 33486, United States
Parkinson's Disease and Movement Disorders Cente at University of Pennyslvania

Philadelphia, Pennsylvania, 19107, United States
Parkinson-Klinik Ortenau GmbH&Co KG

Wolfach, 77709, Germany
Quest Research Institute, LLC

Farmington Hills, Michigan, 48334, United States
Renstar Medical Research

Ocala, Florida, 34470, United States
Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States
Rush University Medical Center

Chicago, Illinois, 60612, United States
Skane University Hospital, Lund University

Lund, 221 85, Sweden
Spedali Civilia di Brescia

Brescia, 25123, Italy
St. Antonius Ziekenhuis (St. Antonius Hospital) - Utrecht

Utrecht, Netherlands
Struthers Parkinson's Center- East

Saint Paul, Minnesota, 55130, United States
The Johns Hopkins University School of Medicine

Baltimore, Maryland, 21287, United States
The Newcastle upon Tyne Hospitals NHS Foundation Trust, Freeman Hospital

Newcastle upon Tyne, United Kingdom
Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States
Univerity of Toledo

Toledo, Ohio, 43614, United States
Universita degli Studi della Campania Luigi Vanvitelli - Clinica Neurologia I

Naples, 80138, Italy
Universita degli Studi di Padova - Azienda Ospedaliera di Padova - Clinica Neurologica

Padova, 35128, Italy
Universitats klinikum Marburg

Marburg, 35039, Germany
University Hospitals Cleveland Medical Center

South Euclid, Ohio, 44121, United States
University Hospitals Plymouth NHS Trust

Plymouth, United Kingdom
University Medical Center Groningen

Groningen, Netherlands
University of California San Diego

La Jolla, California, 92037, United States
University of Colorado

Aurora, Colorado, 80045, United States
University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States
University of Kansas Medical Center

Kansas City, Kansas, 66103, United States
University of Kentucky

Lexington, Kentucky, 40536, United States
University of Maryland Medical Center

Baltimore, Maryland, 21201, United States
University of Miami, Dept. of Neurology

Miami, Florida, 33136, United States
University of Rochester Neurology

Rochester, New York, 14642, United States
University of Texas Health Science Center - San Antonio

San Antonio, Texas, 78229, United States
University of Washington

Seattle, Washington, 98195, United States
Vanderbilt Medical Center

Nashville, Tennessee, 37232, United States
Weil Cornell Medical Center

New York, New York, 10021, United States

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