Amyotrophic Lateral SclerosisNCT07862959
Early Access Program for Ulefnersen in Children, Adolescents and Adults With FUS-associated Amyotrophic Lateral Sclerosis (FUS-ALS)
A study testing Ulefnersen in people with Amyotrophic Lateral Sclerosis, run by Otsuka Pharmaceutical Development & Commercialization, Inc..
Trial summary
This expanded access program provides access to ulefnersen for eligible patients with FUS-associated amyotrophic lateral sclerosis (FUS-ALS) who meet protocol-defined eligibility criteria. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Ulefnersen
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Ability to provide written informed consent (and assent, when required based on age and local requirements) before participation.
- Confirmed disease-causing (pathogenic) variant in the FUS gene, verified by an approved genetic testing laboratory and reviewed by the program geneticist.
- Age 10 years or older, with test results, signs, or symptoms showing ongoing loss of motor nerve cells consistent with amyotrophic lateral sclerosis (ALS).
- Medical ability to undergo repeated injections into the fluid surrounding the spinal cord and other program-related procedures.
- Lung function (slow vital capacity [SVC]) of 30% or greater.
- Stable medications and nutritional support for at least 1 month before entering the program.
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- Willingness to follow the program's pregnancy prevention requirements:
- Females must not be pregnant or breastfeeding and must either be unable to become pregnant, practice true abstinence, or use a highly effective method of birth control during the program and for at least 40 weeks after the last dose.
- Males must practice true abstinence, be surgically sterile, or use a highly effective method of birth control during the program and for at least 40 weeks after the last dose if sexually active with a partner who could become pregnant.
- Patients who previously participated in the FUSION study and completed Part 3 can enroll.
Who cannot join
- Any active or ongoing medical condition that, in the opinion of the treating physician, may increase the risk of treatment with ulefnersen.
- Any medical condition or circumstance that makes repeated spinal taps (lumbar punctures) unsafe or unsuitable.
- Expected need for treatment with blood-thinning medicines (anticoagulants) or medicines that reduce blood clotting (antiplatelet medications) during the program, as determined by the treating physician.
Trial locations and contacts
No sites are listed yet.
About this study
This expanded access program provides access to ulefnersen for eligible patients with FUS-associated amyotrophic lateral sclerosis (FUS-ALS) who meet protocol-defined eligibility criteria. Access is intended from the availability of FUSION clinical trial results until commercial availability of ulefnersen in participating countries. Ulefnersen is provided free of charge.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Ulefnersen
- Keywords
- FUS-associated amyotrophic lateral sclerosis (FUS-ALS)
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UlefnersenDrug
Antisense oligonucleotide solution for intrathecal injection.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Otsuka Pharmaceutical Development & Commercialization, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07862959
- Sponsor's protocol ID
- 362-201-00013
- Status last verified
- Oct 2026
- First submitted
- 2 Oct 2026
- Last updated
- 7 Oct 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Access Program for Ulefnersen in Children, Adolescents and Adults With FUS-associated Amyotrophic Lateral Sclerosis (FUS-ALS)
- ClinicalTrials.gov ID
- NCT07862959
- Acronym / sponsor ID
- 362-201-00013
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- FUS-associated amyotrophic lateral sclerosis (FUS-ALS)
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.Industry (pharma, biotech, devices)
- First posted
- 7 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This expanded access program provides access to ulefnersen for eligible patients with FUS-associated amyotrophic lateral sclerosis (FUS-ALS) who meet protocol-defined eligibility criteria. Access is intended from the availability of FUSION clinical trial results until commercial availability of ulefnersen in participating countries. Ulefnersen is provided free of charge.
Study design
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Ability to provide written informed consent (and assent, when required based on age and local requirements) before participation.
- Confirmed disease-causing (pathogenic) variant in the FUS gene, verified by an approved genetic testing laboratory and reviewed by the program geneticist.
- Age 10 years or older, with test results, signs, or symptoms showing ongoing loss of motor nerve cells consistent with amyotrophic lateral sclerosis (ALS).
- Medical ability to undergo repeated injections into the fluid surrounding the spinal cord and other program-related procedures.
- Lung function (slow vital capacity [SVC]) of 30% or greater.
Key exclusion criteria (3 listed)
- Any active or ongoing medical condition that, in the opinion of the treating physician, may increase the risk of treatment with ulefnersen.
- Any medical condition or circumstance that makes repeated spinal taps (lumbar punctures) unsafe or unsuitable.
- Expected need for treatment with blood-thinning medicines (anticoagulants) or medicines that reduce blood clotting (antiplatelet medications) during the program, as determined by the treating physician.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UlefnersenAntisense oligonucleotide solution for intrathecal injection. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2026.
- Otsuka Early Access Programs:Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Questions about this trial
What is this clinical trial studying?
A study testing Ulefnersen in people with Amyotrophic Lateral Sclerosis, run by Otsuka Pharmaceutical Development & Commercialization, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 7 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Otsuka Call Center (844-687-8522, EarlyAccessProgram@otsuka-us.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Oct 2026; AzTrials last refreshed its copy on 6 Nov 2026.