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Antisense Oligonucleotide

Tofersen for ALS

Phase 3
Waitlist Available
Research Sponsored by Biogen
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
If taking edaravone, participant must have initiated edaravone ≥60 days (2 treatment cycles) prior to Day 1. Edaravone may not be administered on dosing days during this study
Must have diagnosis of superoxide dismutase 1-amyotrophic lateral sclerosis (SOD1-ALS) and must have completed the End of Study Visit for either Parts A, B, or C of Study 233AS101 (NCT02623699) (i.e., were not withdrawn)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to week 364
Awards & highlights

Study Summary

This trial is looking at the safety and effectiveness of a drug for people with ALS and a specific gene mutation.

Who is the study for?
This trial is for individuals with ALS who have a specific SOD1 mutation. They must have been part of an earlier study (233AS101), be on stable doses of riluzole or edaravone, and able to follow the study schedule. Women who can bear children must use effective contraception during the study and for 5 months after.Check my eligibility
What is being tested?
The trial is testing the long-term safety and effects of Tofersen in people with ALS due to SOD1 mutations. It will look at how the body processes Tofersen, its impact on disease markers, and any potential benefits in managing ALS symptoms.See study design
What are the potential side effects?
While not explicitly listed here, common side effects may include reactions at injection sites, potential liver issues indicated by blood tests, fatigue, headache, respiratory infections like colds or sinusitis.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I started taking edaravone more than 60 days ago and won't take it on study dosing days.
Select...
I have SOD1-ALS and finished a part of Study 233AS101 without withdrawing.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to week 364
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to week 364 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Secondary outcome measures
Change from Baseline in Handheld Dynamometry (HHD) Megascore and Individual Muscle Strength
Change from Baseline in Neurofilament Light Chain (NfL) Concentration in Plasma
Change from Baseline in Slow Vital Capacity (SVC)
+6 more

Side effects data

From 2021 Phase 3 trial • 176 Patients • NCT02623699
33%
Ear infection
33%
Infusion site swelling
33%
Lower respiratory tract infection
33%
Sinusitis
33%
Rash
33%
Sinus headache
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part A-SAD: Combined Placebo
Part A-SAD: Cohort 1: BIIB067 10 mg
Part A-SAD: Cohort 2: BIIB067 20 mg
Part A-SAD: Cohort 3: BIIB067 40 mg
Part A-SAD: Cohort 4: BIIB067 60 mg
Part B-MAD: Combined Placebo
Part B-MAD: Cohort 5: BIIB067 20 mg
Part B-MAD: Cohort 6: BIIB067 40 mg
Part B-MAD: Cohort 7: BIIB067 60 mg
Part B-MAD: Cohort 8: BIIB067 100 mg
Part C-Pivotal: Placebo
Part C-Pivotal: BIIB067 100 mg

Trial Design

1Treatment groups
Experimental Treatment
Group I: BIIB067Experimental Treatment1 Intervention
Participants who have completed Parts A, B, or C of study 233AS101 will be placed in this arm.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tofersen
2018
Completed Phase 3
~190

Find a Location

Who is running the clinical trial?

BiogenLead Sponsor
638 Previous Clinical Trials
467,288 Total Patients Enrolled
12 Trials studying Amyotrophic Lateral Sclerosis
2,848 Patients Enrolled for Amyotrophic Lateral Sclerosis
Ionis Pharmaceuticals, Inc.Industry Sponsor
145 Previous Clinical Trials
15,127 Total Patients Enrolled
3 Trials studying Amyotrophic Lateral Sclerosis
286 Patients Enrolled for Amyotrophic Lateral Sclerosis
Medical DirectorStudy DirectorBiogen
2,766 Previous Clinical Trials
8,060,655 Total Patients Enrolled
8 Trials studying Amyotrophic Lateral Sclerosis
948 Patients Enrolled for Amyotrophic Lateral Sclerosis

Media Library

BIIB067 (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT03070119 — Phase 3
Amyotrophic Lateral Sclerosis Research Study Groups: BIIB067
Amyotrophic Lateral Sclerosis Clinical Trial 2023: BIIB067 Highlights & Side Effects. Trial Name: NCT03070119 — Phase 3
BIIB067 (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03070119 — Phase 3
Amyotrophic Lateral Sclerosis Patient Testimony for trial: Trial Name: NCT03070119 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any patients who have not yet been enrolled in this research?

"Unfortunately, this specific clinical trial is no longer looking for patients. However, there are 3 other trials with similar treatments that are still recruiting candidates."

Answered by AI

Have these treatments been tested before in a clinical setting?

"BIIB067 has been trialed in 32 cities and 16 countries. The first clinical trial was completed in 2017 by 138 participants. These trials are sponsored by Biogen and, as of now, 2 studies are still active."

Answered by AI

Are there any know risks associated with taking BIIB067?

"There is both efficacy and safety data available for BIIB067 because it has progressed to Phase 3 trials. As such, our team has given it a score of 3."

Answered by AI

In how many different medical institutions is this research being conducted currently?

"Right now, this trial is being conducted at 20 different sites. While the majority of these locations are in close proximity to major cities like Orlando, Jacksonville and Boston, it is still important that patients select the location closest to them to minimize travel time and costs."

Answered by AI

How many individuals are currently taking part in this clinical research?

"Unfortunately, this study is no longer recruiting patients. The trial was first posted on March 8th, 2017 and ended recruitment on September 2nd, 2021. However, there may be other studies you are eligible for; as of right now, there is one active clinical trial for patients with ALS caused by a superoxide dismutase 1 (SOD1) mutation and two trials for BIIB067 that are still enrolling participants."

Answered by AI

Are there any other instances in which BIIB067 has been studied?

"There are 80 clinical trial sites for BIIB067 across the globe, with 2 of them currently in Phase 3."

Answered by AI

Who else is applying?

What state do they live in?
California
What site did they apply to?
Research Site
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
1

Why did patients apply to this trial?

I have been diagnosed with bulbar palsy and the C9orf72 gene, I am seeking genetic trial or trial with no placebo period.
PatientReceived 2+ prior treatments
~3 spots leftby Jun 2024