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Trial record 10 of 36 for:    Brain | "Adrenomyeloneuropathy"

A Study to Prospectively Assess Disease Progression in Male Children With X-ALD

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ClinicalTrials.gov Identifier: NCT03278899
Recruitment Status : Recruiting
First Posted : September 12, 2017
Last Update Posted : August 8, 2018
Sponsor:
Information provided by (Responsible Party):
NeuroVia, Inc.

Brief Summary:
This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice. No study drug treatment will be given and no changes to patient treatment are necessary.

Condition or disease
X-Linked Adrenoleukodystrophy

Detailed Description:

This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice. No study drug treatment will be given and no changes to the patient treatment are necessary.

Patients with X-ALD will be recruited and invited to attend a baseline visit. After eligible patients are enrolled, retrospective and baseline data will be collected. After enrollment, patients will be seen approximately every 6 months until they meet withdrawal criteria or the Sponsor terminates the study.


Study Type : Observational
Estimated Enrollment : 100 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Non-interventional Study to Prospectively Assess Baseline Status and Disease Progression in Male Children With X-Linked Adrenoleukodystrophy
Actual Study Start Date : February 16, 2018
Estimated Primary Completion Date : June 1, 2021
Estimated Study Completion Date : June 1, 2021





Primary Outcome Measures :
  1. Loes score [ Time Frame: baseline and 24 weeks ]
    Percent change from baseline in brain lesions assessed as Loes score will be calculated

  2. Plasma VLCFA levels [ Time Frame: baseline and 24 weeks ]
    Change from baseline plasma VLCFA levels

  3. Neurological symptoms [ Time Frame: baseline and 24 weeks ]
    Development of new neurological symptoms throughout the study


Biospecimen Retention:   Samples Without DNA
Biospecimen retained: Plasma


Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 13 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Males with a confirmed diagnosis of X-ALD with or without MRI evidence of cerebral involvement
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients will be selected from participating sites, referring physicians, and outreach communities
Criteria

Inclusion Criteria:

  • Signed informed consent by patient's parent(s)/legally acceptable representative(s). In addition, signed children's assent form according to local requirements.
  • Males patients 2-13 years of age with a confirmed diagnosis of X-ALD that fall into one of the following:

    • Asymptomatic patients without MRI evidence of cerebral involvement
    • Patients with MRI evidence of cerebral involvement (Loes score ≥0.5) with or without clinical symptoms
    • Patient who have HSCT within 3 months from enrollment

Exclusion Criteria:

  • Patients who are 14 years of age or older
  • Patients who are in a vegetative state
  • Patients (or their guardians) who are unwilling or unable to comply with the study procedures
  • Patients who received HSCT more than 3 months before enrollment

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03278899


Contacts
Contact: Program Director 415-870-6967 patients@neurovia-inc.com

Locations
United States, California
Stanford University Recruiting
Palo Alto, California, United States, 94304
Contact: Dalia Pena-Solorzano    650-498-9732    dpena25@stanford.edu   
Contact: Sweta Patnaik    650-721-1458    sweta@stanford.edu   
Principal Investigator: Maura Ruzhnikov, MD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Camille Corre    617-724-6374    ccorre@partners.org   
Principal Investigator: Florian S Eichler, MD         
United States, Pennsylvania
University of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Jodi Martin    412-692-6351      
Contact: Mary Branamann    412-692-6350      
Principal Investigator: Maria L Escolar, MD, MS         
Sponsors and Collaborators
NeuroVia, Inc.
Investigators
Study Director: John Henderson, MD NeuroVia, Inc.

Responsible Party: NeuroVia, Inc.
ClinicalTrials.gov Identifier: NCT03278899     History of Changes
Other Study ID Numbers: NV1205-008
First Posted: September 12, 2017    Key Record Dates
Last Update Posted: August 8, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by NeuroVia, Inc.:
Adrenoleukodystropy
X-ALD
ALD

Additional relevant MeSH terms:
Adrenoleukodystrophy
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Disease Progression
Disease Attributes
Pathologic Processes
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Leukoencephalopathies
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases