Pediatric SDLT CNS DisordersPhase 1/2EOM-MP1NCT07410143
Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions
A Phase 1/2 study testing Individualized ASO in people with Pediatric SDLT CNS Disorders, run by EveryONE Medicines Inc. at 1 site in United Kingdom.
Trial summary
This study is being conducted to evaluate individualised antisense oligonucleotides (ASOs) in participants with severely debilitating, life threatening (SDLT) central nervous system (CNS) conditions caused by unique genetic variants amenable to correction by an ASO. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Pediatric SDLT CNS Disorders
- What is being tested?
- Individualized ASO
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 13 Jan 2026to 31 Oct 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- The first participant receiving the individualised ASO, must be between 1 and 17 years of age (inclusive) at the time they receive their first ASO dose.
- The CNS condition is severely debilitating and/or life threatening.
- The identified genetic variant is unique.
- The identified genetic variant is considered the underlying cause of disease.
- The identified genetic variant is amenable to correction by an ASO.
- In the opinion of the investigator, the disease is at a stage that, if halted or slowed by treatment with the individualised ASO, has a reasonable chance to improve the participant's overall disease burden/impact on quality of life.
- In the opinion of the investigator, participant, and/or the participant's legally authorised representative, existing therapies have not resulted in meaningful benefit.
Who cannot join
- Known history or presence of any clinically significant hepatic, renal/genitourinary, gastrointestinal, cardiovascular, cerebrovascular, pulmonary, endocrine, immunological, musculoskeletal, neurological, psychiatric, dermatological, or haematological disease or condition other than the primary disease for which the individualised ASO is being developed that in the opinion of the Investigator could affect patient safety or interfere with study outcomes.
- Any contraindication to brain MRI scans.
- Any contraindication to sedation or anaesthesia.
- Any contraindication to lumbar punctures or IT infusions.
- Treatment with another ASO within 24 weeks of Screening.
- Treatment with any gene replacement therapy at any time.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions.
This study is being conducted to evaluate individualised antisense oligonucleotides (ASOs) in participants with severely debilitating, life threatening (SDLT) central nervous system (CNS) conditions caused by unique genetic variants amenable to correction by an ASO.
Read the detailed description
This phase 1/2 multicentre, open-label, within-participant dose escalation clinical trial is designed to evaluate individualised antisense oligonucleotides (ASOs) in participants aged 1 year or older with severely debilitating, life threatening (SDLT) central nervous system (CNS) conditions caused by unique genetic variants amenable to correction by an ASO. The trial consists of two parts: Part A: a 30-day Screening period and a minimum 4-week Run-in period followed by Part B: a 48-week Treatment period and Safety Follow-up. For each part, the investigator will determine if the participant is appropriate for participation based on disease stage, rate of disease progression, and likelihood of benefit from ASO treatment at the time that the ASO is available.
- Conditions
- Pediatric SDLT CNS Disorders
- Being tested
- Individualized ASO
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Individualized ASODrug
Individualized ASO
| Study group | Role | What they receive |
|---|---|---|
| Individualized ASO | Experimental | Not described Receives: Drug: Individualized ASO |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Treatment-related incidence and severity of adverse events (AEs), including any unfavorable and unintended signs such as abnormal laboratory or test findings
Measured: 48 Weeks
Secondary outcomes (3)
Peak Plasma Concentration (Cmax)
Estimates of ASO maximum plasma concentration (Cmax)
Measured: Plasma collected pre-dose and at .5, 1, 2, 6, 24, and 48 hour post-infusion
Area Under the Plasma Concentration-time Curve (AUC)
Area under the plasma concentration-time curve (AUC) from time zero to infinity following a dosing of study drug. It is an integrated measure of study drug plasma exposure.
Measured: Plasma collected Pre-dose and .5, 1, 2, 6, 24, and 48 hours post infusion
Plasma Half-life (T1/2)
Apparent terminal plasma half-life (T1/2) is the amount of time for plasma concentrations to decline by 50%.
Measured: Plasma collected pre-dose and .5, 1, 2, 6, 24, and 48 hours post infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2026
- Study start13 Jan 2026
- Primary completion30 Sep 2026 (estimated)
- Study completion31 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric SDLT CNS DisordersStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (estimated)
Sponsor and investigators
- Lead sponsor
- EveryONE Medicines Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07410143
- Sponsor's protocol ID
- EOM-MP1
- Other IDs
- 1012261
- Acronym
- EOM-MP1
- Status last verified
- Mar 2026
- First submitted
- 12 Jan 2026
- Last updated
- 17 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions.
- ClinicalTrials.gov ID
- NCT07410143
- Acronym / sponsor ID
- EOM-MP1 / EOM-MP1
- Conditions
- Pediatric SDLT CNS Disorders
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 1 participants (estimated)
- Lead sponsor
- EveryONE Medicines Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Jan 2026 (actual)
- Primary completion
- 30 Sep 2026 (estimated)
- Study completion
- 31 Oct 2026 (estimated)
- First posted
- 13 Feb 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 study is being conducted to evaluate individualised antisense oligonucleotides (ASOs) in participants with severely debilitating, life threatening (SDLT) central nervous system (CNS) conditions caused by unique genetic variants amenable to correction by an ASO.
Detailed description
This phase 1/2 multicentre, open-label, within-participant dose escalation clinical trial is designed to evaluate individualised antisense oligonucleotides (ASOs) in participants aged 1 year or older with severely debilitating, life threatening (SDLT) central nervous system (CNS) conditions caused by unique genetic variants amenable to correction by an ASO. The trial consists of two parts: Part A: a 30-day Screening period and a minimum 4-week Run-in period followed by Part B: a 48-week Treatment period and Safety Follow-up. For each part, the investigator will determine if the participant is appropriate for participation based on disease stage, rate of disease progression, and likelihood of benefit from ASO treatment at the time that the ASO is available.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Individualized ASO | Experimental | Drug: Individualized ASO |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- The first participant receiving the individualised ASO, must be between 1 and 17 years of age (inclusive) at the time they receive their first ASO dose.
- The CNS condition is severely debilitating and/or life threatening.
- The identified genetic variant is unique.
- The identified genetic variant is considered the underlying cause of disease.
- The identified genetic variant is amenable to correction by an ASO.
Key exclusion criteria (6 listed)
- Known history or presence of any clinically significant hepatic, renal/genitourinary, gastrointestinal, cardiovascular, cerebrovascular, pulmonary, endocrine, immunological, musculoskeletal, neurological, psychiatric, dermatological, or haematological disease or condition other than the primary disease for which the individualised ASO is being developed that in the opinion of the Investigator could affect patient safety or interfere with study outcomes.
- Any contraindication to brain MRI scans.
- Any contraindication to sedation or anaesthesia.
- Any contraindication to lumbar punctures or IT infusions.
- Treatment with another ASO within 24 weeks of Screening.
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 |
|---|---|---|
| Individualized ASOIndividualized ASO | Drug | Individualized ASO |
Primary outcome
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Time frame: 48 Weeks
Treatment-related incidence and severity of adverse events (AEs), including any unfavorable and unintended signs such as abnormal laboratory or test findings
3 secondary outcomes
Peak Plasma Concentration (Cmax)
Time frame: Plasma collected pre-dose and at .5, 1, 2, 6, 24, and 48 hour post-infusion
Area Under the Plasma Concentration-time Curve (AUC)
Time frame: Plasma collected Pre-dose and .5, 1, 2, 6, 24, and 48 hours post infusion
Plasma Half-life (T1/2)
Time frame: Plasma collected pre-dose and .5, 1, 2, 6, 24, and 48 hours post infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2026.
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Individualized ASO in people with Pediatric SDLT CNS Disorders, run by EveryONE Medicines Inc. at 1 site in United Kingdom.
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 7 inclusion and 6 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: under way, not looking for participants, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) (time frame: 48 Weeks). It is what the study was designed to measure, not a finding.
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 17 Mar 2026.