Ongoing, recruitingHuman Pharmacology (Phase I)- First administration to humansInterventional

A Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrathecal Administration of AAV9-ABCD1 Gene Therapy (SBT101) in Adult Patients with Adrenomyeloneuropathy

This research is looking into a new gene therapy, SBT101, for adult men living with adrenomyeloneuropathy. This is a condition that can affect movement and balance. The study's main goals are to find out if SBT101 is safe to use and to figure out the right amount (dose) to give. Researchers will also be checking if the treatment helps improve symptoms, particularly how well people can walk and their overall balance. This is a "Phase 1/2" study, which means it's one of the first times this treatment is being given to people, starting with a small number of participants to ensure safety before looking more closely at how well it works.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- First administration to humans

Sponsor

Swanbio Therapeutics Limited

Enrolment target

Start

19 Nov 2024

Adrenomyeloneuropathy

What is this study about?

This study is about a new treatment called SBT101 for a condition called adrenomyeloneuropathy (AMN). AMN is a genetic condition that mainly affects adult men, causing problems with their nerves, especially in the spinal cord. This can lead to difficulties with walking, balance, and other movements. SBT101 is a type of gene therapy, which means it aims to correct the root cause of the condition by delivering healthy genetic material to the body.

The main purpose of this study is to see if SBT101 is safe for people to use and to work out the best dose. Because this is a very early study (called Phase 1/2), participants will be carefully monitored for any side effects. Researchers will also be looking at whether the treatment helps improve symptoms like how far and well people can walk, their balance, and how easily they can move around in daily life. This helps them understand if the treatment is not only safe but also shows promise for helping patients.

Researchers will compare people who receive the treatment with others who don't (a control group) or with what's expected based on previous studies. Over a period of 12 and 24 months, they will regularly check walking ability, balance, and general overall health. The results will help decide if SBT101 can move on to larger studies and potentially become a new treatment option for AMN.

Key takeaways

This is an early-stage study for a new gene therapy (SBT101) for adrenomyeloneuropathy.
It aims to check the safety and best dose of SBT101.
Researchers will also look for improvements in walking and balance.
Only adult men aged 18 and over can take part.
Participation involves regular clinic visits and tests over two years.
You can withdraw from the study at any time.

Who may be eligible?

To join this study, you must be an adult man, aged 18 or older. This particular study is only looking for men because adrenomyeloneuropathy primarily affects men in the way it's being studied here.

More detailed health checks will be done by the study doctors to make sure the treatment is suitable for you and that you meet all the specific requirements. These checks are important to ensure your safety and that the study results are clear. They will look at your overall health and the specifics of your adrenomyeloneuropathy.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you an adult man?
Are you 18 years old or older?
Do you have adrenomyeloneuropathy?
Are you generally well enough to attend regular clinic visits and undergo assessments?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the SBT101 treatment. Researchers will then closely monitor your health for up to 24 months (two years). During this time, you will have regular visits to the clinic. These visits will involve various checks and tests, such as walking tests (like seeing how far you can walk in 6 or 2 minutes, or how quickly you can sit down and stand up five times), balance tests, and other assessments of your movement and daily activities. You'll also be asked about any side effects you might experience. The total duration of your active participation will be two years, with ongoing follow-up.

Potential risks and benefits

Like all medical studies, there are potential benefits and risks. The potential benefit is that SBT101 might help improve your adrenomyeloneuropathy symptoms, such as walking and balance, or slow down the progression of the condition. However, because this is an early-stage study, it's not guaranteed to have a benefit, and there could be side effects. The study team will carefully explain all possible risks, which might include reactions to the gene therapy. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Netherlands

Common questions

What is adrenomyeloneuropathy?

It's a genetic condition, mostly affecting men, that damages nerves, especially in the spinal cord, causing problems with walking and movement.

What is SBT101?

SBT101 is a new gene therapy being tested. It aims to deliver healthy genetic material to correct the underlying cause of adrenomyeloneuropathy.

What does 'Phase 1/2' mean?

This means it's an early study to check if the treatment is safe, to find the right dose, and to see if it shows any signs of helping patients.

How long would I be in the study?

If you join, you would be monitored for up to 24 months (two years).

Will I definitely get the active treatment?

The study design involves some participants getting the active treatment and others either a different dose or a comparison. The study team will explain this fully.