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

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What is this study about?

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.

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 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 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 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 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 Study?

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 Study?

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.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Aqjtnparu Ugl	Amsterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
The Netherlands	Not recruiting	01.06.2022

Trial locations

Investigated drugs:

Adeno-Associated Viral Vector Serotype 9 Containing The Human Habcd1 Gene

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.

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 ID:

2024-518451-39-00

Protocol code:

SBT101-CT101

NCT ID:

NCT05394064

Trial Phase:

Human Pharmacology (Phase I) – First administration to humans

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

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