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

NCT03278899 | Unknown

• 1 other identifier: NV1205-008
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 3

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.

Conditions

X-Linked Adrenoleukodystrophy

Keywords

Adrenoleukodystropy; X-ALD; ALD

Outcome Measures

Primary Outcomes (3)

• Loes score

  Percent change from baseline in brain lesions assessed as Loes score will be calculated

  baseline and 24 weeks

• Plasma VLCFA levels

  Change from baseline plasma VLCFA levels

  baseline and 24 weeks

• Neurological symptoms

  Development of new neurological symptoms throughout the study

  baseline and 24 weeks

Eligibility Criteria

• Age: 2 Years - 13 Years
• Gender Eligibility Details: Males with a confirmed diagnosis of X-ALD with or without MRI evidence of cerebral involvement
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

Patients will be selected from participating sites, referring physicians, and outreach communities

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• NeuroVia, Inc.: lead

Study Sites (3)

Stanford University

Palo Alto, California, 94304, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

University of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

Biospecimen retained: Plasma

MeSH Terms

Conditions

Adrenoleukodystrophy

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases

Study Officials

• John Henderson, MD

  NeuroVia, Inc.

  STUDY DIRECTOR

Central Study Contacts

Program Director

CONTACT

415-870-6967; patients@neurovia-inc.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 30, 2017
• First Posted: September 12, 2017
• Study Start: February 16, 2018
• Primary Completion: June 1, 2021
• Study Completion: June 1, 2021
• Last Updated: August 8, 2018

Record last verified: 2018-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)