Adrenoleukodystrophy (ALD)

The University of Minnesota is the world’s most experienced center in the treatment of patients with cerebral ALD.

We have several clinical trials for ALD patients, and we transplant more cerebral ALD patients than any other center in the United States. We have published extensively on the clinical aspects of ALD, including biomarkers, oxidative stress, neuropsychology, and imaging.

We understand the difficult choices that parents and guardians have to make when considering a blood and marrow transplant. Our specialists have had the opportunity to care for and follow many patients with ALD, and they are prepared to address your child’s complex medical needs. This chart shows how our experience compares to other transplant centers.

Adrenoleukodystrophy Registry

One of the keys to understanding adrenoleukodystrophy is the ability to include large numbers of patients and their families from around the world. 

The Leukodystrophy Center at the University of Minnesota is uniquely positioned to answer important questions about ALD related to biomarkers, imaging, the initiation of disease, neuropsychology, oxidative stress, and possible genotype-phenotype correlations.

Our goal is to assemble an even larger resource of clinical and biological data from families affected by ALD to help us understand the natural history and outcomes of disease.   Learn more about the ALD National Registry Study

Adrenoleukodystrophy Comprehensive Clinic

Paul and TroyALD Clinic Expands to Help More ALD Patients and Families

Our Adrenoleukodystrophy Comprehensive Clinic is a multidisciplinary team approach that ensures the care and monitoring we provide best meets the unique needs of each patient and family. 

The University of Minnesota is the largest center in the world in the assessment and treatment of boys with cerebral adrenoleukodystrophy. We have a commitment to ALD patients and their families to achieve the best outcomes, and to do so as safely as possible. The availability of newborn screening will prove extremely important in the identification of patients at risk, and in minimizing the long-term effects of their disease. This can best be achieved through the partnering of the relevant subspecialists caring for those with ALD in a comprehensive clinic structure, which we believe is the first of its kind.

Our ALD Comprehensive Clinic is located in the Journey Clinic. Please contact the Leukodystrophy Center (leukodystrophy@lists.umn.edu).

Research

A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

This trial will assess the efficacy and safety of autologous CD34+ hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of cerebral adrenoleukodystrophy (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.

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Adrenoleukodystrophy National Registry Study

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.

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Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Study ALD-104 is an international, non-randomized, open-label, multi-site study in male subjects (≤17 years of age at enrollment) with cerebral adrenoleukodystrophy (CALD). Approximately 35 subjects will be infused with Lenti-D Drug Product after myeloablative conditioning with busulfan and fludarabine.

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Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor.

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Imd-002: A Follow-up Study to Evaluate the Safety and Clinical Outcomes of Patients with Non-malignant Diseases Who Have Undergone Hematopoietic Stem Cell Transplantation with Mgta-456

This is a follow-up study to evaluate the long-term safety and efficacy outcomes of patients with inherited metabolic disorders (IMDs) who received MGTA-456 for HSCT in the core study. MGTA-456 is an expanded CD34+ cell therapy product candidate given after myeloablative conditioning to induce rapid and sustained hematopoietic engraftment. In patients with selected IMDs, transplant is expected to replace defective or missing protein, and preserve neurodevelopment. Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) enrolled in the core study will be eligible to participate in this follow-up evaluation.

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Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

This is a multi-center, long-term safety and efficacy follow-up study for subjects with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study.

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MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).

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