BIIB067 (Tofersen) for Amyotrophic Lateral Sclerosis

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Research Site, Paris, France
Amyotrophic Lateral Sclerosis+2 More
BIIB067 (Tofersen) - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug can delay the onset of ALS in people with a genetic mutation that causes ALS.

See full description

Eligible Conditions

  • Amyotrophic Lateral Sclerosis
  • Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Other trials for Amyotrophic Lateral Sclerosis

Study Objectives

This trial is evaluating whether BIIB067 (Tofersen) will improve 1 primary outcome and 9 secondary outcomes in patients with Amyotrophic Lateral Sclerosis. Measurement will happen over the course of Up to 12 months.

Up to 12 months
Parts B and C: Percentage of Participants with Emergence of Clinically Manifest ALS Within 12 Months of Part B Baseline
Up to 2 years
Parts B and C: Change from Baseline in Percent Predicted Slow Vital Capacity (SVC)
Parts B and C: Change in ALS Functional Rating Scale (ALSFRS-R) Total Score
Parts B and C: Percentage of Participants with Outcome as Death or Permanent Ventilation Based on Time to Death or Permanent Ventilation Analysis
Parts B and C: Percentage of Participants with Outcome as Deaths Based on Time to Death Analysis
Parts B and C: Time to Emergence of Clinically Manifest ALS
Parts B, C and D: Change from Baseline in Plasma NfL Concentrations
Parts B, C and D: Change in Total Cerebrospinal Fluid (CSF) SOD1 Concentrations
Parts B, C and D: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) during the Treatment Period
Up to 24 months
Parts B and C: Percentage of Participants with Emergence of Clinically Manifest ALS Within 24 Months of Part B Baseline

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Amyotrophic Lateral Sclerosis

Trial Design

5 Treatment Groups

Part A: Natural History Run-in
1 of 5
Part D: Randomized, Double-Blind, Placebo-Controlled
1 of 5
Part D: Open-Label Treatment
1 of 5
Part C: Open-Label Extension
1 of 5
Part B: Randomized, Double-Blind, Placebo-Controlled
1 of 5
Active Control
Experimental Treatment

This trial requires 150 total participants across 5 different treatment groups

This trial involves 5 different treatments. BIIB067 (Tofersen) is the primary treatment being studied. Participants will be divided into 4 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Part D: Randomized, Double-Blind, Placebo-ControlledParticipants from Part A who develop clinically manifest ALS prior to randomization in Part B may be eligible to participate in Part D. During Part D, participants will be randomized to receive BIIB067 100 mg or placebo via IT injection on Days 1, 15, 29, and every 28 days thereafter for up to 2 years.
Part D: Open-Label Treatment
Drug
Participants from Part A who develop clinically manifest ALS prior to randomization in Part B may be eligible to participate in Part D. During Part D, participants will receive BIIB067 100 mg via IT injection on Days 1, 15, 29, and every 28 days thereafter for up to 2 years.
Part C: Open-Label ExtensionParticipants from Part B who develop clinically manifest ALS may be eligible to participate in Part C. During Part C, participants who received placebo in Part B will receive BIIB067 100 mg via IT injection on Days 1, 15, 29, and every 28 days thereafter for up to 2 years. Participants who received BIIB067 during Part B will receive BIIB067 100 mg on Days 1, 29, and every 28 days thereafter for up to 2 years, with a dose of placebo on Day 15 to maintain the study blind.
Part B: Randomized, Double-Blind, Placebo-ControlledParticipants from Part A who meet the protocol-defined NfL threshold and remain presymptomatic may be eligible to participate in Part B. During Part B, participants will receive BIIB067 100 milligram (mg) or placebo via intrathecal (IT) injection on Days 1, 15, 29, and every 28 days thereafter for up to 2 years.
Part A: Natural History Run-inParticipants enrolled in Part A will undergo blood draws approximately once every 28 days to assess neurofilament light chain (NfL) levels.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2810

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 2 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 2 years for reporting.

Closest Location

Washington University School of Medicine - Saint Louis, MO

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 4 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
People who are not clinically manifesting ALS are considered to be clinically presymptomatic for the disease. show original
Protocol-approved participants will have a rapidly progressive SOD1 mutation, confirmed by a central reader, or a SOD1 mutation that is approved for inclusion by an external mutation adjudication committee. show original
People with low levels of NfL in their blood. show original
Key Part A

Patient Q&A Section

What causes amyotrophic lateral sclerosis?

"L-citrulline, lysine and myelin phospholipids are all elevated in the serum of patients who have amyotrophic lateral sclerosis. The findings illustrate that the metabolism of these biomolecules is altered in the brain of patients with this disorder and may be relevant to determining the genesis of the disorder. A detailed biochemical definition of the metabolism of these biomolecules may be useful for the diagnosis and management of neurodegenerative disorders such as amyotrophic lateral sclerosis." - Anonymous Online Contributor

Unverified Answer

Can amyotrophic lateral sclerosis be cured?

"Because of its relatively recent discovery, the optimal management of ALS remains unsettled. We discuss the current management as a means of defining optimum management strategies." - Anonymous Online Contributor

Unverified Answer

What are the signs of amyotrophic lateral sclerosis?

"The symptoms of ALS can vary depending upon the severity of motor symptomatology. There are often associated disturbances of consciousness, and extrapyramidal symptoms. Patients also may develop sensory symptoms and changes in pain or temperature perception. The age of onset of ALS is typically within the fifth decade of life, and with a male to female ratio of 3:2. It affects mainly motor and lower limbs. About 10 percent of cases of ALS occur with a definite diagnosis." - Anonymous Online Contributor

Unverified Answer

What is amyotrophic lateral sclerosis?

"ALS is a very rare but progressive motor neurodegenerative condition characterized by slow, progressive weakness and wasting of muscles, leading to reduced quality of life and eventual respiratory failure. Overall, less than 1-3% of all motor neuron disorders are ALS. The epidemiological characteristics and the underlying cause of ALS are currently being explored with much need.\n" - Anonymous Online Contributor

Unverified Answer

How many people get amyotrophic lateral sclerosis a year in the United States?

"Around 100 new cases of ALS of all subtypes and the prevalence of fatal cases have been ascertained to be around 100 per 100,000 a year in the U.S. of any ethnic origins. The incidence of ALS in the U.S. is highest among non-Hispanic whites and those with presymptomatic myotonic degeneration, but the rates of incidence in other groups are similar." - Anonymous Online Contributor

Unverified Answer

What are common treatments for amyotrophic lateral sclerosis?

"The majority of treatments for amyotrophic lateral sclerosis have been tested only with animal models of the disease, and only two treatments have been tested for the treatment of amyotrophic lateral sclerosis in humans; both are supportive therapy. The treatment for amyotrophic lateral sclerosis, either by a specific or supportive approach, is highly dependent on a patient's functional level, age, and motivation to treat." - Anonymous Online Contributor

Unverified Answer

What is the latest research for amyotrophic lateral sclerosis?

"Findings from recent clinical trials have brought up a new possible treatment. A phase III trial concluded that baclofen may reduce the progression of non-focal ALS by slowing down the worsening of the disease. However, additional studies are still needed to see if this therapy will bring significant relief to patients." - Anonymous Online Contributor

Unverified Answer

Is biib067 (tofersen) typically used in combination with any other treatments?

"Only 5% of patients receiving tofersen alone or in combination with other anti-tumor agents to treat patients with stage III NSCLC used the recommended dose. It is unknown as to why patients would not be treated according to the dosing guidelines." - Anonymous Online Contributor

Unverified Answer

Does amyotrophic lateral sclerosis run in families?

"Recent findings supports a heritable component in the disease, since we found that the siblings of these patients who died without any signs of a definite neurological disease are also afflicted with ALS. The family history is the major risk factor as only 10 of the 18 patients who did not have a family history of ALS died without any neurological disease. There is no evidence of a genetic linkage between ALS and SOD1 locus." - Anonymous Online Contributor

Unverified Answer

Does biib067 (tofersen) improve quality of life for those with amyotrophic lateral sclerosis?

"The study results suggest that BIB067 does not improve QoL for people with ALS. Future clinical trials should investigate a range of quality of life measures." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating amyotrophic lateral sclerosis?

"Clinical trials of disease modifying drugs (SOD1 gene and ALZ05001, GMDC-0196, LNA-1425, LNA-1501) targeting the protein misfolding pathway have resulted in new insights in the treatment of these rare disorders. Thus, the new discoveries for these therapies are potential for future trials and studies." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in biib067 (tofersen) for therapeutic use?

"Biib067 is a novel drug candidate for treating several forms of dementia-related neurodegenerative diseases including Alzheimer's disease and vascular dementia. Recently, the U.S. FDA granted priority review to the drug application for the potential therapeutic use of Biib067. In accordance with the FDA’s guidelines, we are evaluating Biib067 as a potential treatment for patients with dementia and tofersen is under investigation in a Phase IIb, double-blind, randomized study to evaluate the cognitive symptoms of patients with Alzheimer’s disease. This is expected to complete in early 2014." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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