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Study on the Safety and Effectiveness of SBT101 Gene Therapy for Adults with Adrenomyeloneuropathy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare genetic condition called Adrenomyeloneuropathy (AMN). AMN is a disorder that affects the spinal cord and can lead to problems with movement and coordination. The study is testing a new treatment called SBT101, which is a type of gene therapy. Gene therapy involves using a specially designed virus to deliver a healthy copy of a gene into the body. In this case, the treatment uses an adeno-associated viral vector serotype 9 to carry the human HABCD1 gene. This treatment is given through a procedure called intrathecal administration, which means it is delivered directly into the space around the spinal cord.

The purpose of the study is to evaluate the safety and effectiveness of this one-time treatment in adults with AMN. Participants in the study will receive either the SBT101 treatment or a placebo. The study will follow participants over a period of time to monitor their health and any changes in their condition. This includes regular check-ups and assessments to see how well they can walk and move, as well as any changes in their symptoms. The study aims to gather information on how the treatment affects the disease and whether it can help improve the quality of life for those affected by AMN.

Throughout the study, participants will be closely monitored for any side effects or adverse reactions to the treatment. The study will also compare the results of those who receive the treatment with those who do not, to better understand its impact. This research is important for developing new therapies that could potentially help people with AMN and improve their daily lives.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, diagnosis of adrenomyeloneuropathy, and genetic testing results.

    The assessment also checks for clinical evidence of spinal cord involvement and evaluates the Expanded Disability Status Scale (EDSS) score.

  2. Step 2

    Consent and preparation

    Written informed consent is required before any study procedures begin.

    Participants must agree to use double-barrier contraception methods and refrain from donating sperm for at least 6 months after the procedure.

  3. Step 3

    Treatment administration

    The treatment involves a one-time administration of SBT101, a gene therapy delivered via intrathecal injection. This means the medication is injected into the space around the spinal cord.

    The active substance is an adeno-associated viral vector containing the human ABCD1 gene.

  4. Step 4

    Monitoring and follow-up

    Participants are monitored for safety and any adverse effects. The primary focus is on identifying any Grade III or Grade IV adverse events related to SBT101.

    Follow-up assessments include various physical tests such as the 6-minute Walk Test, 2-minute Walk Test, and others at 12 and 24 months.

  5. Step 5

    Long-term evaluation

    The study continues to monitor participants up to 60 months to evaluate long-term safety and efficacy.

    Changes in physical abilities, balance, and overall health impressions are assessed and compared to control groups.

Who can join the trial?

6 criteria

  • Must be a male adult aged between 18 and 65 years.
  • Must have a diagnosis of **X-linked adrenoleukodystrophy (ALD)**, confirmed by a genetic test showing a mutation in the **ABCD1 gene** and supported by past tests showing high levels of **very long-chain fatty acids (VLCFA)**.
  • Must show signs of spinal cord involvement with an **EDSS score** (a scale used to measure disability in people with multiple sclerosis) between 1 and 4.5, and a **pyramidal function score** of at least 1 in the Functional System Score of the EDSS. Signs of **pyramidal tract dysfunction** do not include increased reflexes (**hyperreflexia**).
  • Must agree to use two forms of birth control (such as a condom and another method like a diaphragm, intrauterine device, spermicidal jelly, or hormonal contraceptive) and agree not to donate sperm for at least 6 months after the study procedure.
  • If receiving any other treatment for ALD, including medications or supplements like antioxidants, Lorenzo's oil, or statins, or physical rehabilitation, the treatment must have been stable for at least 4 weeks before the study starts, and the patient must agree to continue at the same dose and frequency during the first part of the study.
  • Must have provided written informed consent before any study procedures are performed.

Who cannot join the trial?

4 criteria

  • Only males can participate in the study. Females are not eligible.
  • Participants must be adults. Children and teenagers cannot take part.
  • Individuals with conditions other than Adrenomyeloneuropathy are not eligible. Adrenomyeloneuropathy is a specific medical condition being studied.
  • Participants must not belong to any vulnerable populations. This means groups that might need special protection or care.
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Verified sites

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  • Verified

    Amsterdam UMC

    The NetherlandsAmsterdam, The Netherlands

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Investigated drugs

SBT101 is a gene therapy being studied for its potential to treat adrenomyeloneuropathy (AMN). It is administered through an injection into the spinal canal, a method known as intrathecal administration. The therapy uses a virus called AAV9 to deliver a healthy copy of the ABCD1 gene to the patient's cells. The goal is to help the body produce the protein it needs to function properly, potentially improving symptoms or slowing the progression of the disease.

What is already known about the treatment

SBT101 – SBT101 is an investigational gene therapy administered through an injection into the spinal canal, known as intrathecal administration. It is currently being studied in clinical trials to assess its safety and effectiveness for treating adrenomyeloneuropathy, a rare genetic disorder. The therapy works by delivering a functional copy of the ABCD1 gene using a viral vector, aiming to correct the underlying genetic defect at the molecular level. SBT101 is classified as a gene therapy, a type of treatment that involves modifying or manipulating genes to treat or prevent disease.

Investigated diseases

Adrenomyeloneuropathy – Adrenomyeloneuropathy is a genetic disorder that affects the nervous system and adrenal glands. It is a form of X-linked adrenoleukodystrophy, primarily affecting males. The disease typically begins in adulthood and is characterized by progressive stiffness and weakness in the legs, leading to difficulty walking. Over time, individuals may experience problems with balance and coordination. The condition can also cause adrenal insufficiency, leading to fatigue, weight loss, and skin changes. Symptoms and progression can vary widely among individuals.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-518451-39-00Protocol codeSBT101-CT101Estimated enrolment16 patientsSponsorSwanbio Therapeutics Limited

sourced from the EU Clinical Trials Register and site verification

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).