Custom Gene-Targeting drug tested in single child with rare brain disease
NCT ID NCT07410143
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one child with a rare, severe genetic brain disorder. The drug aims to correct the specific genetic mistake causing the disease. The trial focuses on safety and how the drug moves through the body, with only one participant enrolled.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- individualized antisense oligonucleotide (ASO)
- What this could lead to
- If successful, this approach could point toward a way to treat rare genetic brain diseases by correcting the specific genetic error in each patient.
- What could go wrong
- This is a very early trial with only 1 participant, so results may not apply to others. The treatment is experimental and may cause side effects or not work at all.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 1 person
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2026
- Expected to finish
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Oct 2026
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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1 year and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Great Ormond Street Hospital
London, United Kingdom