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ULEFNERSEN

Clinical trials investigating ULEFNERSEN are studying its use in people with amyotrophic lateral sclerosis linked to FUS mutations. The trials are designed to assess efficacy, safety, pharmacokinetics, pharmacodynamics, and survival-related outcomes in this target group. They focus on how participants function over time and when rescue treatment may be needed.

Table of contents

Trial overview

The available trial for ULEFNERSEN is titled A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS).[1] It is an interventional study, which means researchers are giving a study treatment and comparing outcomes over time.[1] The trial is authorised and plans to enroll 95 participants.[1]

Who is being studied

This trial focuses on people with amyotrophic lateral sclerosis, also called ALS, who have FUS mutations.[1] The condition is described in the trial as amyotrophic lateral sclerosis with fused in sarcoma mutations, or FUS-ALS.[1] This means the study is not for all ALS patients, but for a specific genetic group.[1]

The trial data do not list detailed inclusion or exclusion rules beyond the FUS-ALS population.[1] Because of that, the exact participation rules are not fully shown in the source provided.[1]

What researchers measure

The main goal is to evaluate the effects of ULEFNERSEN versus placebo on functional impairment, which means how much the disease affects daily ability.[1] Researchers use the ALSFRS-R, a scale with 12 questions and a total score from 0 to 48, where higher scores mean better function.[1] The scale looks at bulbar function, gross motor skills, fine motor skills, and respiratory function.[1]

The trial also measures time of rescue.[1] In this study, rescue can happen if a participant’s ALSFRS-R total score falls below 15 and drops by at least 10 points from baseline at Study Day 253 or later, and the change is confirmed after at least 4 weeks.[1] If rescue happens, the participant may stop Part 1 and enter Part 2 of the study.[1]

Another key outcome is ventilation assistance-free survival, shortened to VAFS.[1] This means the time until either death or permanent ventilation, which is defined in the trial as more than 22 hours of mechanical ventilation per day for more than 21 consecutive days without an acute reversible event.[1]

The study title also states that researchers are evaluating safety, pharmacokinetics, and pharmacodynamics.[1] In simple terms, pharmacokinetics means how the body handles the treatment, and pharmacodynamics means what the treatment does in the body.[1]

Study design and treatment groups

The trial compares ULEFNERSEN with placebo, which is a look-alike treatment without the active study drug.[1] The intervention list shows intrathecal use, which means the study treatment is given into the fluid around the spinal cord.[1]

The source also notes two parts of the study, with Part 1 using the joint rank analysis of ALSFRS-R outcomes and rescue-related measures.[1] The trial data do not provide a full breakdown of all study procedures, but they do show that the design includes follow-up over time and comparison against placebo.[1]

Trial phase and status

The study is listed as Phase 4.[1] Phase 4 trials are later-stage studies that look at how a treatment performs in a clinical setting after earlier development work.[1] The current status is Authorised.[1]

The enrollment target is 95 participants.[1] This size suggests a focused study in a specific patient group rather than a very large population.[1]

What the trial results mean for patients

This trial is trying to learn whether ULEFNERSEN can help people with FUS-ALS keep better function for longer and delay major worsening.[1] The main patient-centered outcomes are everyday function, the need for rescue treatment, and survival without permanent breathing support.[1]

Because the trial is focused on a specific genetic form of ALS, its results may be most relevant to people with FUS mutations rather than all people with ALS.[1] The available source gives a clear picture of the study’s goals, but not all detailed eligibility rules or all visit procedures.[1]

Trial IDPhaseCondition studiedStatusEnrollment
NCT04768972Phase 4Amyotrophic Lateral Sclerosis with Fused in Sarcoma mutationsAuthorised95

FAQ

  • What condition is being studied in ULEFNERSEN trials? The trial is studying amyotrophic lateral sclerosis, also called ALS, in people with fused in sarcoma mutations, often written as FUS-ALS. ALS is a serious disease that affects nerve cells and can reduce movement and breathing ability.
  • Who can take part in these trials? The study is for participants with ALS and FUS mutations. The trial data do not give more detailed entry rules, so participation depends on the study team’s screening process.
  • What is the main goal of the trial? The main goal is to see whether ULEFNERSEN helps with clinical function and survival in FUS-ALS. The trial also looks at safety and how the body handles the study treatment.
  • What phase is the ULEFNERSEN trial? The trial is listed as Phase 4. This means it is being studied in a later-stage setting, after earlier development work has already taken place.
  • What outcomes are the researchers measuring? Researchers are measuring change in ALS function using the ALSFRS-R score, time to rescue treatment, and ventilation assistance-free survival. These outcomes help show how the disease changes over time and whether the study treatment may delay worsening.

Ongoing Clinical Trials on ULEFNERSEN

  • Study on the Effects of ION363 for Patients with Amyotrophic Lateral Sclerosis with Fused in Sarcoma Mutations

    Not recruiting

    4 1
    Investigated drugs:
    Belgium Germany Ireland Italy The Netherlands Poland +2

Glossary

  • Amyotrophic lateral sclerosis (ALS): A serious disease that affects nerve cells controlling muscles. It can lead to weakness, trouble moving, and breathing problems.
  • FUS mutation: A change in the FUS gene. In this trial, the mutation is part of the condition being studied.
  • FUS-ALS: ALS that is linked to mutations in the FUS gene.
  • Interventional study: A clinical trial where participants receive a study treatment or placebo so researchers can compare results.
  • Phase 4: A later stage of clinical research. It usually studies a treatment in a more real-world setting after earlier trials.
  • Placebo: A look-alike treatment with no active study drug. It helps researchers compare whether the study treatment works better than no active treatment.
  • ALSFRS-R: A score used to measure how well a person with ALS can do daily activities. It checks speech, movement, hand function, and breathing.
  • Functional impairment: Loss of ability to do normal daily tasks because of disease.
  • Rescue treatment: A study option used if a participant gets much worse. In this trial, it may mean leaving Part 1 and entering Part 2.
  • Ventilation assistance-free survival (VAFS): The length of time before death or the need for permanent breathing support. It is used to measure survival without long-term ventilation.
  • Pharmacokinetics: How the body absorbs, moves, and removes a treatment.
  • Pharmacodynamics: How a treatment affects the body.

References