Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

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

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

Official Title

Exercise Study of Function and Pathology for Women With X-ALD

Brief Summary

      The purpose is to see how X-linked adrenoleukodystrophy (X-ALD) is associated with strength
      and sensation using MRI, in women with X-ALD. The investigators will also see whether
      exercise can improve these symptoms for women with X-ALD.
    

Detailed Description

      X-linked adrenoleukodystrophy (X-ALD), a [sex-linked] progressive neurodegenerative disease,
      is caused by a defect in the ABCD1 gene. The disease is expressed in multiple ways, but the
      most common adult form is adrenomyeloneuropathy (AMN), which results in slowly progressive
      changes in muscle tone and weakness, sensory loss, and dysfunction of the autonomic nervous
      system. In a previous study the investigators linked abnormalities in the [brain/spinal cord]
      to lower extremity weakness in men with AMN; however, there have been no studies evaluating
      these relationships in women carriers (i.e., women with AMN). It is unknown, in women with
      AMN, how the pattern of damage in the brain and spinal cord relates to disability and if
      these patterns predict responsiveness to treatment. The investigators hypothesize that by
      using magnetization transfer (MT) and diffusion tensor imaging (DTI), two magnetic resonance
      imaging (MRI) modalities, to track particular changes in the brain and spinal cord will
      predict disability and additionally, who is likely to respond best to a training regimen. The
      investigators expect that these more advanced imaging techniques will be more sensitive and
      accurate quantitative measures of clinical motor function and women with greater loss in the
      spinal cord compared to the brain will benefit most from training to improve disability. To
      test this hypothesis, women with AMN will receive MRI scans at baseline and complete measures
      of global walking and lower extremity impairments of vibration sensation, spasticity, and
      strength at three time-points: baseline, 12 weeks, and 18 weeks after baseline. The group
      will participate in a resistive training program for 12 weeks. MRI data will be correlated to
      changes over time in measures of impairment to determine their relationships. The linking of
      this information will not only be important for better defining disability in women with AMN
      but it will also help to guide physicians and rehabilitation therapists in predicting who is
      likely to respond to rehabilitative interventions, as well as for optimizing the effects of
      future pharmacological interventions.
    


Study Type

Interventional


Primary Outcome

Change in Maximal Voluntary Contraction of the Hip Flexors From Baseline to End of Training and to Post-training


Condition

X-linked Adrenoleukodystrophy

Intervention

exercise training

Study Arms / Comparison Groups

 exercise
Description:  Female carriers as well as healthy age and gender matched individuals will participate in an exercise paradigm.

Publications

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

Recruitment Information


Recruitment Status

Behavioral

Estimated Enrollment

31

Start Date

May 2012

Completion Date

October 2014

Primary Completion Date

October 2014

Eligibility Criteria

        Inclusion Criteria:

          -  confirmed diagnosis, X-ALD heterozygote carrier

          -  no medical contraindication to participating in a strength training program

          -  able to follow complex directions as determined by a score of ≤1 on a subset of
             questions taken from the NIH Stroke scale (Brott et al. 1989)

          -  hip flexion strength: 6.6-15.8kg

          -  hip extension strength: up to 18.3 kg

          -  normal passive range of motion at hips/knees/ankles

          -  able to walk ≥50m

        Exclusion Criteria:

          -  Evidence of other neurological deficit that could interfere, such as previous stroke
             or muscle disease

          -  congestive heart failure

          -  cancer

          -  orthopedic conditions

          -  severe pain that precludes study participation

          -  seizures

          -  pregnancy

          -  other medical condition that precludes participation in an exercise program, e.g.,
             unstable angina, uncontrolled diabetes, uncontrolled hypertension

        Healthy controls have the same age and exclusion criteria as women with AMN except that
        they will not be carriers for X-ALD. They must have normal neurological function.
      

Gender

Female

Ages

21 Years - 70 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Kathleen M Zackowski, Ph.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01594853

Organization ID

NA_00045673


Responsible Party

Principal Investigator

Study Sponsor

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Collaborators

 European Leukodystrophy Association

Study Sponsor

Kathleen M Zackowski, Ph.D., Principal Investigator, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.


Verification Date

December 2017