Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions (EOM-MP1)

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Pediatric SDLT CNS Disorders
• Intervention / Treatment: Drug: Individualized 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 Type: Interventional
• Enrollment (Estimated): 1
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United Kingdom
  • London, United Kingdom
    • Great Ormond Street Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. 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.
2. The CNS condition is severely debilitating and/or life threatening.
3. The identified genetic variant is unique.
4. The identified genetic variant is considered the underlying cause of disease.
5. The identified genetic variant is amenable to correction by an ASO.
6. 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.
7. In the opinion of the investigator, participant, and/or the participant's legally authorised representative, existing therapies have not resulted in meaningful benefit.

Exclusion Criteria:

1. 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.
2. Any contraindication to brain MRI scans.
3. Any contraindication to sedation or anaesthesia.
4. Any contraindication to lumbar punctures or IT infusions.
5. Treatment with another ASO within 24 weeks of Screening.
6. Treatment with any gene replacement therapy at any time.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Individualized ASO	Drug: Individualized ASO; Individualized ASO

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	48 Weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Peak Plasma Concentration (Cmax)	Estimates of ASO maximum plasma concentration (Cmax)	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.	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%.	Plasma collected pre-dose and .5, 1, 2, 6, 24, and 48 hours post infusion

Collaborators and Investigators

• Sponsor: EveryONE Medicines Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 13, 2026
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): October 31, 2026

Study Registration Dates

• First Submitted: January 12, 2026
• First Submitted That Met QC Criteria: February 7, 2026
• First Posted (Actual): February 13, 2026

Study Record Updates

• Last Update Posted (Actual): March 17, 2026
• Last Update Submitted That Met QC Criteria: March 16, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Other Study ID Numbers: EOM-MP1; 1012261 (Other Identifier: IRAS ID)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No