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Quick Info
Trial Type
Treatment Type
Drug Trials
Start Date
Contact Information
Australia, Other
Macquarie University, Sydney, 2109, Australia
Contact: Richard Gan   +61298123739
Belgium, Other
UZ Leuven, Leuven, 3000, Belgium
Contact: +3216344280
Brazil, Other
Hospital Sao Paulo, São Paulo, 04037-002, Brazil
Contact: 551155764050
Canada, Alberta
University of Calgary, Calgary, AB, T2N 4Z6, Canada
Contact: Berchman Wong   403-210-7009
Canada, Ontario
Sunnybrook Health Sciences Centre, Toronto, ON, M4N 3M5, Canada
Contact: 4164804475
Canada, Quebec
Genge Partners Inc., Montréal, QC, H4A 3T2, Canada
Contact: 5143983868
France, Other
Hôpital Pitié-Salpêtrière, Paris cedex 13, 75013, France
Contact: 33142162471
Germany, Other
Hannover Medical School, Hannover, 30625, Germany
Contact: 49511532570
Universitaetsklinikum Ulm, Ulm, 89081, Germany
Contact: 497315000
Italy, Other
Cresla "Rita Levi Montalcini" Department of Neuroscience, Torino, 10126, Italy
Contact: Christina Moglia   +390116335439
Japan, Other
University of Tokyo Hospital, Bunkyo-ku, 113-8655, Japan
Kagoshima University Hospital, Kagoshima-shi, 890-8520, Japan
Other, Other
Hanyang University Seoul Hospital, Seoul, 4763, Other
Poland, Other
Centrum Medyczne Neuro Protect, Warszawa, 01-684, Poland
Contact: +48501762789
Spain, Other
Hospital Universitari i Politecnic La Fe, Valencia, 46026, Spain
Contact: 34644490025
Sweden, Other
University Hospital of Umea, Umeå, 90185, Sweden
Contact: +46907852372
United Kingdom, Other
Sheffield, Sheffield, S10 2HQ, United Kingdom
United States, Arizona
HonorHealth Neurology, Scottsdale, AZ, 85251, United States
Contact: 6026582863
United States, California
UC San Diego, La Jolla, CA, 92037, United States
Contact: Rose Previte
California Pacific Medical Center, San Francisco, CA, 94109, United States
Contact: Ryan Razavi   415-600-0486
United States, Florida
Holy Cross Hospital Phil Smith Neuroscience Institute, Fort Lauderdale, FL, 33308, United States
Contact: Ashley Stepler   954-542-3442
University of Miami, Miami, FL, 33136, United States
Contact: Anne-Laure Grignon
United States, Georgia
Emory University, Atlanta, GA, 30322, United States
Contact: Meraida Polak   404-778-3807
United States, Illinois
Northwestern University, Chicago, IL, 60611, United States
Contact: 312-503-0671
United States, Maryland
Johns Hopkins Hospital, Baltimore, MD, 21287, United States
Contact: 4106149874
United States, Massachusetts
Massachusetts General Hospital, Boston, MA, 02114, United States
Contact: Recruitment Coordinator   617-726-5097
United States, Missouri
Washington University School of Medicine, Saint Louis, MO, 63110, United States
Contact: Dr Robert Bucelli   844-257-2273
United States, New York
Columbia University Medical Center, New York, NY, 10032, United States
United States, Texas
Austin Neuromuscular Center, Austin, TX, 78756, United States
Contact: Yessar Hussain
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
Months Since Onset
Number of months since first symptoms of ALS.
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Not Available
Open Label
Update Notes
Sites and contact info updated
Site contact update
Updates to names of drug (tofersen/Qalsody).
Site contact updates
Site contact updates
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Site contact updates
New sites added
Sites added
New site recruiting
Site updated
Site updated
New site recruiting
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No significant updates
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Site update
New sites recruiting
New sites recruiting
New sites recruiting
Site updates
Site contact update
Sites added
Protocol updates, new sites added
New site added
New site added
New site added
Site contacts updated
Study recruiting
Study added

Other Information

The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).
Key Part A Inclusion Criteria:
- Participants should have a protocol-defined rapidly progressive SOD1 mutation, confirmed by a central reader, or a SOD1 mutation that is approved for inclusion by an external mutation adjudication committee.
- Participants with plasma NfL level less than the protocol-defined threshold.
- Participants who are clinically presymptomatic for ALS (i.e., must not have clinically manifest ALS).
Key Part A Exclusion Criteria:
- History or positive test result at screening for human immunodeficiency virus (HIV).
The requirement for testing at Screening may be omitted if it is not permitted by local regulations.
- Current hepatitis C infection (defined as positive Hepatitis C Virus (HCV) antibody and detectable HCV RNA). Participants with positive HCV antibody and undetectable HCV Ribonucleic Acid (RNA) are eligible to participate in the study (United States Centers for Disease Control and Prevention).
- Current hepatitis B infection (defined as positive for hepatitis B surface antigen (HBsAg) and/or anti-Hepatitis B Core antibody (HBc)). Participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive anti-HBc, and positive anti-hepatitis B surface antibody (HBs) or vaccination (defined as negative HBsAg, negative anti-HBc, and positive anti- HBs) are eligible to participate in the study.
- History of systemic hypersensitivity reaction to tofersen, the excipients contained in the formulation, and if appropriate, any diagnostic agents to be administered during the study.
- History of confounding neuromuscular or neurological disorder that is expected to have a progressive (i.e., worsening) course during the study, and/or is expected to be associated with elevations in NF, in the opinion of the Investigator.
- Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that if not managed optimally could place a participant at an increased risk for intraoperative or postoperative bleeding.
- Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression ≤ 90 days of screening, which in the opinion of the Investigator would interfere with the study procedures.
- Treatment with riluzole, edaravone, and/or sodium phenylbutyrate/taurursodiol (also known as ursodoxicoltaurine). If the participant has been on riluzole, edaravone, and/or sodium phenylbutyrate/taurursodiol, the medication(s) must be discontinued for at least 5 half-lives prior to Screening.
- Use of off-label treatments for ALS.
- Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering RNA, stem cell therapy, or gene therapy is allowed.
- Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication (e.g., clopidogrel) that cannot be safely continued or held for an LP procedure, if necessary, according to local or institutional guidelines and/or Investigator determination.
- Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment, biological agent, device, or approved therapy for investigational use. Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator.
NOTE: Other protocol defined Inclusion/Exclusion criteria will apply.
Trial Protocol as Published on
NCT04856982 (First Published: 4/20/2021)