ALS study: Tofersen
Official title Tofersen in Non-SOD1 ALS
ClinicalTrials.gov ID: NCT07294144
What this study is testing
What is Tofersen?
Tofersen is an investigational medicine being studied to see if it helps people with a specific type of ALS. It is designed to lower the amount of a specific protein in the body that is linked to nerve damage.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study checks if tofersen lowers certain protein levels in the blood and spinal fluid. It also looks at safety, side effects, and how it affects daily life and strength.
- Who it's for
- Adults who have ALS that is not caused by a SOD1 gene mutation.
- What's involved
- Participants get a 100mg injection of tofersen into the spinal fluid through a lumbar puncture. They also complete questionnaires and tests for strength and breathing.
- Time commitment
- The study lasts 32 weeks, with injections every 2 to 4 weeks for the first 24 weeks and two follow-up visits at the end.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use PHI in accordance...
- Aged 18 years or older at the time of informed consent.
- Confirmed diagnosis of ALS.
- Time since onset of weakness due to ALS ≤ 24 months at the time of the screening visit.
- Prior confirmed genetic testing negative for SOD1 and FUS mutations.
You likely can't join if
- Treatment with another investigational drug (including investigational drugs for ALS through compassionate use or expanded access programs)...
- Current enrollment in any other treatment study.
- History of drug abuse or alcoholism within ≤ 6 months of study enrollment that would limit participation in the study, as determined by the...
- Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the...
- Ongoing medical condition (e.g., wasting or cachexia, severe anemia) that according to the Investigator would interfere with the conduct or...
- History of confounding neuromuscular or neurological disorder that is expected to have a progressive (i.e., worsening) course during the study...
See the full eligibility criteria
- Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use PHI in accordance with national and local participant privacy regulations.
- Aged 18 years or older at the time of informed consent.
- Confirmed diagnosis of ALS.
- Time since onset of weakness due to ALS ≤ 24 months at the time of the screening visit.
- Prior confirmed genetic testing negative for SOD1 and FUS mutations. Participants with mutations in genes other than SOD1 and FUS may be enrolled at the discretion of the Site Investigator.
- SVC ≥ 50% of predicted value as adjusted for sex, age, and height (from the sitting position).
- Medically able to undergo the study procedures and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.
- All participants must agree to practice effective contraception during the study and be willing and able to continue contraception for 5 months after their last dose of study treatment.
- If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit.
- If taking edaravone, participant must have initiated edaravone ≥ 60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that...
- Treatment with another investigational drug (including investigational drugs for ALS through compassionate use or expanded access programs), biological agent, or device within 1 month or 5 half-lives of study agent...
- Current enrollment in any other treatment study.
- History of drug abuse or alcoholism within ≤ 6 months of study enrollment that would limit participation in the study, as determined by the Investigator.
- Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the screening period.
- Ongoing medical condition (e.g., wasting or cachexia, severe anemia) that according to the Investigator would interfere with the conduct or assessments of 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 neurofilament, in...
- Female participants who are pregnant or currently breastfeeding.
- Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression ≤ 90 days, as determined by the Investigator.
- History of allergies to a broad range of anesthetics.
- Tracheostomy.
- Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally could place a participant at an increased risk for intraoperative or postoperative bleeding.
- Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication that cannot be safely held before and/or after an LP procedure according to local or institutional...
- Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter.
- Clinically significant abnormalities in hematology or clinical chemistry parameters, as determined by the Investigator, which would render the participant unsuitable for enrollment.
- Inability to comply with study requirements.
- Other unspecified reasons that, in the opinion of the Investigator, make the participant unsuitable for enrollment.
The study team makes the final eligibility decision.
Where it is enrolling
- Atlanta, Georgia, United States
- Boston, Massachusetts, United States
- St Louis, Missouri, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Atlanta, Georgia, United States; Boston, Massachusetts, United States; St Louis, Missouri, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.