The PREVAiLS Study is sponsored by Prilenia and Ferrer, who are developing pridopidine, an investigational medication, called the “study drug”, for people living with amyotrophic lateral sclerosis (ALS).
PREVAiLS
Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS
Learn More about the PREVAiLS StudyAbout the study
Study overview
Evaluating pridopidine
The PREVAiLS (Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS) Study is a clinical research study that is being done to evaluate whether the study drug, pridopidine, can help people living with ALS. The study will look at the study drug to see if it may slow disease progression and improve speech, breathing, and survival, and to see how safe it is.
Investigational medication
The study drug is “investigational”, which means that it has not been approved for use by health authorities, and its effects are still being studied.
Study medication
In this study, participants will receive either the study drug or a placebo (together called the “study medication”). A placebo looks the same as the study drug but does not contain active medication. The study medication comes in capsules that are taken by mouth.
What to expect if you participate in the PREVAiLS Study
Participation in the study will last for about 2 years.
During the study, participants will have 11 visits to the study center, and 9 phone calls with the study team.
Participants will continue taking certain ALS medications, as long as the dose is unchanged for at least 4 weeks before starting the study and the dose is planned to remain the same throughout the study.
There will be no cost for any study-related visits, calls, medications, tests or assessments. Further, participants will be reimbursed for study-related costs, such as travel, parking, and food during study center visits (restrictions may apply).
The study will be conducted in accordance with international ethical guidelines, Good Clinical Practice (GCP), and local regulatory requirements.
An Institutional Review Board (IRB)/Ethics Committee (EC) has reviewed and approved this study. An IRB/EC protects the rights, safety, and well-being of participants.
Timeline of the Study
Screening
Up to 4 weeks
During the screening period, you will visit the study center and the study doctor will check if you are able to take part in the study.
See if you can take partStudy treatment period
Double-blind period
48 weeks
During the first 48 weeks, participants will be assigned by chance to receive either the study drug or placebo. This part of the study is “double-blind”, which means you, the study doctor, and the study team will not know which one you are taking. For every 3 people who receive the study drug, 2 people will receive the placebo.
The study medication is taken once a day for the first 2 weeks, then twice a day for the remainder of this period. You will have 6 visits to the study center for tests and assessments, and 5 phone calls with the study team.
Open-label extension (OLE) period
48 weeks
Participants who complete the study treatment period will continue to a 48-week OLE period. During this period, all participants will receive the study drug.
During this 48-week period you will visit the study center 4 times for tests and assessments, and have 4 phone calls with the study team.
What the study will measure
The study will compare the study drug to placebo across several measures of safety and effectiveness in ALS, including:
Disease progression
Survival
Speech
Respiratory function
Bulbar function – swallowing and salivation
Quality of life
About ALS & the study drug
ALS is a fatal, progressive disease that affects the nerve cells that control muscle movement. This causes muscle weakness and, over time, makes it difficult or impossible for people to move their muscles and speak clearly, eventually making it hard for them to breathe 1,2.
Pridopidine (the study drug) is an investigational medication being studied in ALS and other neurodegenerative diseases. It works by activating a specific protein called the sigma-1 receptor (S1R). S1R helps to protect nerve cells and support their function. It is hoped that by activating S1R the study drug may help protect nerve cells from damage and slow the progression of ALS 3,4.
The study drug comes in capsules and is taken by mouth.
Eligibility
You, or someone you care for, may be eligible to take part in the PREVAiLS Study if you/they:
are 18–80 years of age
have ALS and are within 18 months of first symptoms appearing
are able to swallow a capsule.
A description of PREVAiLS, including full eligibility criteria, is available on ClinicalTrials.gov (opens in new tab) and EU Clinical trials (opens in new tab), as required by U.S. Law.
Locations
Where will the PREVAiLS Study take place?
The PREVAiLS Study will take place at multiple centers in the United States, Canada, Europe, and Israel.
Click on the map below to see study site locations. If you are interested in participating in the PREVAiLS Study or other clinical studies for ALS, or wish to learn more about possible study participation, speak with your doctor.
Prilenia and Ferrer are unable to provide medical advice or additional study details beyond what is included on this website.
Trial sites: ALS Clinic Bonn University in Bonn, Germany; Azienda Ospedaliero Universitaria Di Modena in Italy; Barrow Neurological Institute in Phoenix, Arizona; Baylor College of Medicine in Houston, Texas; California Pacific Medical Center in San Francisco, California; Centro Clinico NEMO Fondazione Serena Onlus – ASST Niguarda Hospital in Milan, Italy; Centrum Medyczne Linden in Krakow, Poland; Centrum Medyczne Neuromed, Bydgoszcz in Bydgoszcz, Poland; Charité Universitätsmedizin Berlin- ALS outpatient clinic in Berlin, Germany; CHU Bretonneau in France; CHU Lille in France; CHU Nice in France; Clinical Hospital Santiago de Compostela in Santiago de Compostela, A Coruna; Columbia University in New York, United States; Emory University in Atlanta, Georgia; Genge Partners in Montreal, Quebec; Hadassah Medical Center in Jerusalem, Israel; Healey & AMG Center for ALS Research at Massachusetts General Hospital in Boston, Massachusetts; Hôpital de la Salpêtrière in France; Hôpitaux Universitaires de Marseille Timone in France; Hospital del Mar in Barcelona, Spain; Hospital Universitari i Politècnic La Fe in Valencia, Spain; Hospital Universitario La Paz – Carlos III in Madrid, Spain; Johns Hopkins University School of Medicine Outpatient Center in Baltimore, Maryland; Karolinska Institutet in Solna, Sweden; King’s College Hospital in Denmark Hill, London; Lewis Katz School of Medicine at Temple University in Philadelphia, Pennsylvannia; Mayo Clinic Jacksonville in United States; McMaster University Medical Centre in Hamilton, Ontario; MDN Centre Queen Square Institute of Neurology in United Kingdom; Medical School Hannover, ALS Centre in Hannover, Germany; Medical University of Warsaw in Warsaw, Poland; Neuromuscular Unit, University Hospital Jena in Jena, Thuringia; Northwestern University in Chicago, Illinois; Royal Stoke University Hospital in Stoke on Trent, United Kingdom; Somnos Clinical Research in Lincoln, Nebraska; Swedish Neuroscience Institute in 500 17th Ave, Seattle; Tel Aviv Sourasky Medical Center in Tel Aviv, Israel; Texas Neurology in Dallas, Texas; Trinity College Dublin in Dublin, Ireland; UMC Utrecht in Utrecht, Netherlands; Umeå University Hospital in Daniel Naezéns väg, 907 37 Umeå; Universitätsklinikum Schleswig-Holstein in Germany; University Hospital of Bellvitge in LHospitalet del Llobregat, Barcelona; University of Alberta in Canada; University of Campania “Luigi Vanvitelli” in Italy; University of Colorado in United States; University of Kansas in Fairway, Kansas; University of Milan Medical School in Milan, Italy; University of Padova in Italy; University of South Florida in Tampa, Florida; University of Torino in Italy; University of Ulm in Ulm, Germany; University of Washington in Seattle, Washington; UZ Leuven in Leuven, Belgium; Washington University School of Medicine in St. Louis, Missouri.
Frequently Asked Questions (FAQs)
ALS or amyotrophic lateral sclerosis is a progressive disease of the nervous system that affects the motor neurons, which are responsible for controlling muscle movement, causing muscle weakness and paralysis. As the disease progresses, people living with ALS may experience significant physical limitations, including loss of mobility and breathing and speaking difficulties 1,2.
The study will evaluate the effectiveness and safety of the study drug. The study will look at whether the study drug can help slow disease progression and improve speech, breathing, and survival in ALS.
The study involves two 48-week periods. In the first, double-blind period participants will be assigned by chance to receive either the study drug (pridopidine) or placebo. In the first 48-week period, participants will be required to visit the study center 7 times (including first screening visit), and to have 5 phone calls.
In the second 48-week period, the open-label extension, all participants will receive the study drug. During this period, participants will be required to visit the study center 4 times, and have 4 phone calls.
The study drug (pridopidine) works by activating a specific protein called the sigma-1 receptor (S1R) that helps to protect nerve cells and support their function 3,4. It is hoped that, by activating this protein, the study drug may help protect nerve cells from damage and slow the worsening of ALS.
Participation in any clinical study is completely voluntary. Your decision to participate – or not participate – in this clinical study will have no effect on the medical care that you receive now or in the future. If you are eligible and choose to participate in the study, you may leave the clinical study at any time, and for any reason.
Yes, all participants will be reimbursed for study-related costs (travel, parking and food during study center visits). For more information, please speak with a member of the study team (restrictions may apply).
To learn more about this study, please visit ClinicalTrials.gov (opens in new tab) and EU Clinical trials (opens in new tab).
A description of this study is available on ClinicalTrials.gov (opens in new tab) and EU Clinical trials (opens in new tab), as required by U.S. Law