Early Diagnosis Of Childhood Cerebral ALD

Related Clinical Trial
Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy and Metachromatic Leukodystrophy SMART-ALD – A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD) Quality of Life in Women With X-linked Adrenoleukodystrophy A Clinical 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) Plasma Exchange With Albumin in AMN Patients Stem Cell Transplant for Inborn Errors of Metabolism Human Placental-Derived Stem Cell Transplantation Precision Exercise in Children With Malignant Hemopathies Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy MD1003-AMN MD1003 in Adrenomyeloneuropathy A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients. Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD) A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children Early Diagnosis Of Childhood Cerebral ALD Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children HSCT for High Risk Inherited Inborn Errors Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD) UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD) A Phase III Trial of Lorenzo’s Oil in Adrenomyeloneuropathy Lentiviral Gene Therapy for X-ALD MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD) Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) Newborn Screening for Adrenoleukodystrophy A Study to Prospectively Assess Disease Progression in Male Children With X-ALD Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy Expanded Access for Lorenzo’s Oil (GTO/GTE) in Adrenoleukodystrophy Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Brief Title

Early Diagnosis Of Childhood Cerebral ALD

Official Title

Early Diagnosis Of Childhood Cerebral Adrenoleukodystrophy

Brief Summary

      The goal of this single institution study is to evaluate boys with adrenoleukodystrophy (ALD)
      diagnosed early in life, and to prospectively monitor them to determine parameters that will
      facilitate earlier detection of the childhood cerebral form of the disease. These at-risk
      subjects will be assessed yearly through travel to the University of Minnesota, where plasma
      and cerebral spinal fluid (CSF) biomarker studies, MRI based imaging and neuropsychological
      assessments will be performed at the University of Minnesota Masonic Children's Hospital and
      Clinics. The MRI and lumbar puncture to obtain CSF will be obtained under sedation. In
      addition, at intervening 6 months intervals information will be obtained remotely, including
      surveys and MRI's in their home location. Also at that time blood samples will be obtained
      locally and shipped to the University of Minnesota for study. There is no therapeutic intent
      in this study.
    



Study Type

Observational


Primary Outcome

ALD Early Evaluation

Secondary Outcome

 Emergence of cerebral disease through imaging

Condition

Adrenoleukodystrophy



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

0

Start Date

November 2018

Completion Date

July 2023

Primary Completion Date

July 2023

Eligibility Criteria

        Inclusion Criteria:

          -  Boys with confirmed adrenoleukodystrophy, as determined by very long chain fatty acid
             (VLCFA) analysis and/or genotyping. Genotyping is not necessary for diagnosis.

          -  Between 1 and 5 years of age, inclusive at the time of consent.

          -  Able to undergo a sedation

          -  English as primary language of the household, to maximize consistency of the
             neuropsychological/developmental testing.

          -  Voluntary written parental/guardian consent

        Exclusion Criteria:

          -  Evidence of cerebral disease at time of enrollment - patients over 3 years of age must
             have an MRI within 4 months of signing consent to confirm that there is no evidence of
             cerebral disease

          -  Inability or unwillingness to travel to the University of Minnesota once a year for
             the duration of the study

          -  Evidence of cerebral disease by standard T2/FLAIR MRI. If a subject develops cerebral
             ALD during the study, they will come off study, as it is anticipated that they would
             be considered for transplantation.
      

Gender

Male

Ages

1 Year - 5 Years

Accepts Healthy Volunteers

No

Contacts

Paul Orchard, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02948062

Organization ID

2016NTLS155

Secondary IDs

MT2016-29R

Responsible Party

Sponsor

Study Sponsor

Masonic Cancer Center, University of Minnesota


Study Sponsor

Paul Orchard, MD, Principal Investigator, University of Minnesota


Verification Date

July 2018