Clinical trials are research studies designed to evaluate the safety, efficacy, and potential benefits of new treatments, drugs, or therapies in human participants. For adrenoleukodystrophy (ALD), clinical trials are crucial in advancing our understanding of the disease and developing effective treatments. These trials may involve gene therapy, medications, or other interventions aimed at slowing disease progression, improving symptoms, or correcting the underlying genetic mutations. Participants in ALD clinical trials contribute to scientific research that could lead to new therapies, offering hope for better management of the disease and improving the quality of life for those affected.
Participation in clinical trials for ALD is not only a step toward potentially accessing new treatments but also a vital contribution to the broader ALD community. When individuals with ALD participate in trials, they provide researchers with critical data that helps to deepen the understanding of the disease, its progression, and how it responds to different interventions. This collective knowledge accelerates the development of new therapies, which can benefit everyone in the ALD community.
Additionally, clinical trial participation helps to identify which treatments are most effective and safe, paving the way for approval by regulatory bodies and wider availability. For families affected by ALD, participating in clinical trials is a way to be actively involved in the fight against the disease, offering hope not just for their own loved ones, but for all those who may be impacted by ALD in the future.
ClinicalTrials.gov provides patients, their family members, health care professionals, researchers, and the public easy access to information on publicly and privately supported clinical trials on a wide range of diseases and conditions. The website is maintained by the U.S. National Library of Medicine (NLM) at the National Institutes of Health (NIH). We have also included information below.
This information is accurate as of June 19, 2023. Please reach out to ALD Connect with any questions.
These clinical trials are active, and are currently recruiting.
This is a prospective, non-therapeutic protocol designed to create and maintain a registry of participants with adrenoleukodystrophy (ALD) and known/presumed carriers of ALD. This study also involves maintaining a prospective biorepository to collect and store buccal swab, blood, stool and urine samples as well. In this protocol, pediatric (including infants), adolescents and adult patients with confirmed or presumed ALD (based on positive VLCFA testing and/or confirmed mutation) will be offered potential study participation. After appropriate consent (online or in-person), subjects will be requested to provide a medical history (with authorization of release of medical records), longitudinal biospecimens, and permission to perform laboratory analyses on these samples. The overall goal is to understand the natural disease course in affected and unaffected patients (identified patients and relatives with a diagnosis of ALD) to assemble a resource of clinical, medical, and biological data from the participants. This study also aims to understand the outcomes of this disease, as well as possibly develop biomarkers to identify prognostic markers for disease progression, which may help develop effective interventions. The biospecimen bank and registry will provide access to samples and data for the ongoing studies as well as will provide an important resource for the future research.
Click here to view the clinical trial on clinicaltrials.gov.
The primary objectives of this study are 1) to assess the prevalence of symptomatic courses in females with X-ALD and 2) to determine the impact of AMN symptoms on the quality of life of affected women in various areas (including everyday life, work, social network, sleep quality, sexuality, mood).
Click here, or watch our webinar for more information.
Click here to view the clinical trial on clinicaltrials.gov.
A randomized controlled first-in-human Phase 1/2 study to assess the safety and explore the efficacy of SBT101, an investigational gene therapy, over the course of 2 years in men with AMN. Participants will be asked to continue follow up for a total of 5 years after SBT101.
Click here to view the IRB-approved flyer for this study.
Click here to view the clinical trial on clinicaltrials.gov.
Subjects will be administered multiple doses of VK0214 in a cohort dose escalation process. A total of up to 36 subjects will be enrolled in the study, into one of three dose cohorts. For each cohort, up to 12 subjects will be randomized to receive VK0214 or placebo in a 3:1 ratio so that there will be a total of up to 9 subjects for each of the active doses and up to 3 subjects dosed with placebo in each cohort.
Click here to view the clinical trial on clinicaltrials.gov.
The investigators recently observed that up to 25% of women with X-linked adrenoleukodystrophy (ALD) have moderate to severe Restless Leg Syndrome (RLS). In this study, the investigators aim to estimate the prevalence of RLS among women with ALD and to assess whether pramipexole improves RLS symptoms as well as sleep and gait measures in women with ALD.
Click here to view the clinical trial on clinicaltrials.gov.
These clinical trials are not yet recruiting.
A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).
Click here to view the clinical trial on clinicaltrials.gov.
A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL770 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).
Click here to view the clinical trial on clinicaltrials.gov.
These clinical trials are active, but not currently recruiting.
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study.
After completing a parent clinical study (approximately 2 years), eligible participants will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.
Click here to view the clinical trial on clinicaltrials.gov.
An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (CALD) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT).
Click here to view the clinical trial on clinicaltrials.gov.
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments. This study will help obtain a better understanding of the progression of disease with AMN and facilitate efficient clinical development of future interventional medications.
Click here to view the IRB-approved flyer for this study.
Click here to view the clinical trial on clinicaltrials.gov.
This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the AMN phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).
Minoryx completed a phase 2/3 study on MIN-102 in AMN in Europe and the United States, and an open-label extension phase is still ongoing.
Click here to view the clinical trial on clinicaltrials.gov.
These clinical trials are complete.
Study ALD-104 is an international, non-randomized, open-label, multi-site study in male participants (<or=17 years of age at enrollment) with cerebral adrenoleukodystrophy (CALD). Approximately 35 participants will be infused with Lenti-D Drug Product after myeloablative conditioning with busulfan and fludarabine.
This trial will evaluate the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of CALD. A subject’s blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the subject following myeloablative conditioning.
Click here to view the clinical trial on clinicaltrials.gov.
The aim of this study is to evaluate the effectiveness of a multi-approach intervention (“SMART-ALD”) on physical and mental well-being and quality of life in n=30 X-ALD symptomatic heterozygous females compared to a waiting list control group (n=30) using electronic health (ehealth) technology.
Click here or watch our webinar for more information.
Click here to view the clinical trial on clinicaltrials.gov.
Prior research suggests that higher vitamin D levels in the blood are associated with reduced brain inflammation among individuals with multiple sclerosis, a disease that is similar to the cerebral demyelinating form of ALD. However, serious side effects (e.g. hypercalcemia, kidney stones) can occur if vitamin D levels get too high.
The current study is designed to establish a safe dose of vitamin D for boys with ALD. Although the doses chosen for this study are expected to be safe, the investigators will monitor participants for early signs of vitamin D-related toxicity. The investigators will also examine whether or not vitamin D supplementation affects markers of oxidative stress and inflammation in the blood and brains of ALD boys.
Click here to view the clinical trial on clinicaltrials.gov.
This trial will assess the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of cerebral adrenoleukodystrophy (CALD). A participant’s blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the participant following myeloablative conditioning.
Click here to view the clinical trial on clinicaltrials.gov.
Considering participation in a clinical trial forALD or AMN is a significant decision that involves weighing potential benefits and risks. Clinical trials offer access to cutting-edge therapies and medical care that might not be available outside the research setting, providing hope for slowing disease progression or managing symptoms. Participation also plays a crucial role in advancing scientific knowledge, contributing to the development of treatments that could benefit the entire ALD community. However, it’s important to thoroughly understand the trial’s purpose, procedures, potential side effects, and the commitment required. Discussing the decision with healthcare providers, family members, and other individuals who have participated in clinical trials can provide valuable insights. Ultimately, participating in a clinical trial is a personal choice, but for those affected by ALD, it’s an opportunity to be part of a larger effort to combat this challenging disease and improve outcomes for all who are impacted by it.
When considering participation in a clinical trial for adrenoleukodystrophy (ALD), patients and caregivers can ask their doctor the following comprehensive set of questions to make an informed decision:
Understand the goals of the trial and how it aims to address ALD.
Learn about the participant demographics, including age, gender, and disease stage, to see how closely they match your situation.
Inquire about the scientific basis for the treatment and any previous research or trials that support its use.
Clarify the procedures, treatments, and interventions that will be part of the trial.
Compare the trial treatment with the existing standard of care to understand the potential trade-offs.
Ask about the time commitment, travel requirements, and any changes to your routine that the trial might necessitate.
Learn about the duration of the trial, including all phases and follow-up periods.
Find out if the trial involves any hospital stays and, if so, for how long.
Clarify whether the treatment costs are covered by the trial sponsors, your insurance, or out-of-pocket.
Ask about compensation for travel, lodging, and other expenses incurred due to trial participation.
Understand the post-trial care plan and how your health will be monitored after the study ends.
Inquire about how progress will be measured and when and how you will receive trial results.
Clarify who will oversee your care during the trial and how your regular doctor will be involved.
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