Adrenoleukodystrophy – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:18:36 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Adrenoleukodystrophy – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study of drug interactions between leriglitazone, gemfibrozil, itraconazole, and carbamazepine and food effects in healthy male volunteers for adrenoleukodystrophy treatment https://clinicaltrials.eu/trial/study-of-drug-interactions-between-leriglitazone-gemfibrozil-itraconazole-and-carbamazepine-and-food-effects-in-healthy-male-volunteers-for-adrenoleukodystrophy-treatment/ Wed, 29 Apr 2026 15:06:54 +0000 https://clinicaltrials.eu/trial/study-of-drug-interactions-between-leriglitazone-gemfibrozil-itraconazole-and-carbamazepine-and-food-effects-in-healthy-male-volunteers-for-adrenoleukodystrophy-treatment/ This clinical trial aims to study the effects of various medications when taken together with leriglitazone, an investigational drug, in healthy male subjects. The study will also examine how food affects the way leriglitazone works in the body. The trial involves testing interactions between leriglitazone and three different medicines: gemfibrozil (a medication used to lower blood fats), itraconazole (an antifungal medication), and carbamazepine (a medication used to treat epilepsy and nerve pain).

The study consists of four parts. In the first three parts, participants will receive leriglitazone along with one of the three mentioned medications to see how they interact. In the fourth part, researchers will study how taking leriglitazone with a high-fat meal affects the way the body processes the medication. The medication will be given as an oral suspension, which means it is a liquid form that can be swallowed.

Throughout the study, researchers will monitor participants’ health through various tests, including blood samples to measure medication levels in the body, heart recordings (electrocardiograms), vital signs, and laboratory tests. They will also look at specific genes that might affect how the body processes these medications. The study is designed to understand how these different factors might change the way leriglitazone works in the body.

]]> Study on Pramipexole for Treating Restless Legs in Women with X-linked Adrenoleukodystrophy https://clinicaltrials.eu/trial/study-on-pramipexole-for-treating-restless-legs-in-women-with-x-linked-adrenoleukodystrophy/ Wed, 29 Apr 2026 15:03:03 +0000 https://clinicaltrials.eu/trial/study-on-pramipexole-for-treating-restless-legs-in-women-with-x-linked-adrenoleukodystrophy/ This clinical trial is focused on studying the effects of a medication called Pramipexole Viatris on women with a rare genetic condition known as X-linked adrenoleukodystrophy (X-ALD). This condition can lead to various symptoms, including issues with leg movement, often referred to as restless legs syndrome (RLS). The purpose of the study is to explore how common RLS is in women with X-ALD and to see if the medication can help improve symptoms related to leg movement, sleep, and walking.

Participants in the study will take the medication in the form of a tablet, which is taken orally. The study will last for a period of up to 16 weeks. During this time, researchers will monitor changes in sleep patterns, mood, and overall quality of life. The study will also assess how the medication affects the ability to walk and perform daily activities. Some participants may receive a placebo instead of the actual medication to help compare the effects.

The trial aims to gather valuable information that could lead to better treatment options for women with X-ALD who experience restless legs. By understanding the impact of Pramipexole Viatris on these symptoms, researchers hope to improve the quality of life for those affected by this condition. The study is expected to conclude by the end of 2025.

]]> Long-term Safety and Efficacy Study of Elivaldogene Autotemcel for Patients with Cerebral Adrenoleukodystrophy https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-elivaldogene-autotemcel-for-patients-with-cerebral-adrenoleukodystrophy/ Wed, 29 Apr 2026 15:01:38 +0000 https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-elivaldogene-autotemcel-for-patients-with-cerebral-adrenoleukodystrophy/ This clinical trial focuses on a rare genetic disease called Cerebral Adrenoleukodystrophy (CALD). CALD affects the brain and can lead to severe neurological problems. The study is examining a treatment known as elivaldogene autotemcel, also referred to as Lenti-D. This treatment involves using a patient’s own blood stem cells, which are modified to include a healthy version of a gene that is faulty in people with CALD. The modified cells are then infused back into the patient through a process called intravenous infusion.

The purpose of this study is to monitor the long-term safety and effectiveness of the Lenti-D treatment in individuals who have already received it in previous studies. Participants will be followed for up to 15 years after their initial treatment. During this time, researchers will observe how well the treatment works in preventing major disabilities and whether any side effects or new health issues arise. This includes checking for conditions like graft versus host disease (GVHD), which can occur when the infused cells attack the patient’s body, and other immune-related or neurological disorders.

Throughout the study, participants will undergo regular health assessments, including MRI scans to monitor changes in the brain. The study aims to provide valuable information on the long-term outcomes of using Lenti-D for treating CALD, helping to understand its potential benefits and risks over an extended period. This information will be crucial for improving treatment strategies for individuals affected by this challenging condition.

]]> Study on the Safety and Effectiveness of SBT101 Gene Therapy for Adults with Adrenomyeloneuropathy https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-sbt101-gene-therapy-for-adults-with-adrenomyeloneuropathy/ Wed, 29 Apr 2026 14:57:36 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-sbt101-gene-therapy-for-adults-with-adrenomyeloneuropathy/ 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.

]]> Study on the Effects of Dimethyl Fumarate for Patients with Adrenomyeloneuropathy https://clinicaltrials.eu/trial/study-on-the-effects-of-dimethyl-fumarate-for-patients-with-adrenomyeloneuropathy/ Wed, 29 Apr 2026 14:57:22 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-dimethyl-fumarate-for-patients-with-adrenomyeloneuropathy/ This clinical trial is focused on studying the effects of a medication called dimethyl fumarate on patients with a condition known as adrenomyeloneuropathy (AMN). AMN is a rare genetic disorder that affects the nervous system and adrenal glands, leading to symptoms such as difficulty walking and muscle weakness. The trial aims to determine if dimethyl fumarate can improve the clinical condition of patients with AMN compared to a placebo. Dimethyl fumarate is administered in the form of gastro-resistant tablets, which are designed to withstand stomach acid and dissolve in the intestines.

Participants in the study will receive either dimethyl fumarate or a placebo over a period of 36 months. The study will monitor changes in the participants’ ability to walk, using tests like the 2 Minute Walk Test (2MWT) and the 6 Minute Walk Test (6MWT). These tests help assess improvements in walking ability and overall mobility. Other assessments will include tests for balance, strength, and the ability to climb stairs, as well as questionnaires about urinary and bowel function. The study will also involve neuroimaging techniques, such as MRI, to observe changes in the brain, and blood tests to measure markers of nerve damage and inflammation.

In addition to dimethyl fumarate, the study will also involve the use of magnesium stearate and microcrystalline cellulose, which are common substances used in tablet formulations. These substances are included in the study to ensure the tablets are properly formed and effective. The trial is designed to provide valuable information on the potential benefits of dimethyl fumarate for individuals with AMN, with the hope of improving their quality of life and managing the symptoms of this challenging condition.

]]> Study on the Effects of Leriglitazone in Male Children with Cerebral X-Linked Adrenoleukodystrophy Before Stem Cell Transplant https://clinicaltrials.eu/trial/study-on-the-effects-of-leriglitazone-in-male-children-with-cerebral-x-linked-adrenoleukodystrophy-before-stem-cell-transplant/ Wed, 29 Apr 2026 14:28:08 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-leriglitazone-in-male-children-with-cerebral-x-linked-adrenoleukodystrophy-before-stem-cell-transplant/ This clinical trial is focused on studying a rare genetic disease called cerebral X-linked adrenoleukodystrophy (CALD), which affects the brain and is primarily found in young boys. The study is investigating the effects of a treatment using a medication known as Min-102, which contains the active substance leriglitazone. The purpose of the study is to see if Min-102 can slow down the progression of CALD before patients undergo a procedure called a human stem cell transplant.

Participants in the study will receive Min-102 as an oral suspension, which means it is taken by mouth in liquid form. The study will take place over a period of time, with regular check-ups and assessments to monitor the effects of the treatment. These assessments will include clinical evaluations and imaging tests like magnetic resonance imaging (MRI) to observe changes in the brain. The study aims to determine if the treatment can help stabilize the disease and improve the quality of life for those affected by CALD.

Throughout the study, researchers will collect data to evaluate the effectiveness of Min-102 in halting disease progression. This will involve tracking changes in specific symptoms and overall health. The study will also compare the outcomes of those receiving Min-102 with those who may receive a placebo, to better understand the treatment’s impact. The ultimate goal is to provide valuable insights into managing CALD and potentially offer a new treatment option for patients with this challenging condition.

]]> Study on the Effects of Leriglitazone for Adult Men with Cerebral Adrenoleukodystrophy https://clinicaltrials.eu/trial/study-on-the-effects-of-leriglitazone-for-adult-men-with-cerebral-adrenoleukodystrophy/ Wed, 29 Apr 2026 14:22:43 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-leriglitazone-for-adult-men-with-cerebral-adrenoleukodystrophy/ This clinical trial is focused on studying a rare genetic disease called cerebral adrenoleukodystrophy (cALD), which affects the brain and is primarily found in adult males. The study will evaluate the effectiveness and safety of a medication called leriglitazone, which is taken as an oral suspension. Participants in the study will either receive leriglitazone or a placebo, which looks like the medication but does not contain the active ingredient.

The main goal of the study is to see if leriglitazone can help increase survival in patients with cALD. Participants will be monitored over a period of time to assess their health and any changes in their condition. The study will track important health outcomes, such as the time it takes for a participant to become bedridden or require permanent ventilatory support. Additionally, changes in brain function and quality of life will be evaluated.

Throughout the study, participants will undergo various assessments to measure the effects of leriglitazone. These assessments will include tests for cognitive function, physical abilities, and overall health status. The study aims to provide valuable information on whether leriglitazone can be a beneficial treatment option for individuals with cerebral adrenoleukodystrophy.

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