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

A Non-interventional Study to Prospectively Assess Baseline Status and Disease Progression in Male Children With X-Linked Adrenoleukodystrophy

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.

Study Overview

• Status: Unknown
• Conditions: 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
• Enrollment (Anticipated): 100

Contacts and Locations

Study Locations

• United States
  • California
    • Palo Alto, California, United States, 94304
      • Recruiting
      • Stanford University
      • Contact: Dalia Pena-Solorzano; Phone Number: 650-498-9732; Email: dpena25@stanford.edu
      • Contact: Sweta Patnaik; Phone Number: 650-721-1458; Email: sweta@stanford.edu
      • Principal Investigator: Maura Ruzhnikov, MD
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Recruiting
      • Massachusetts General Hospital
      • Contact: Camille Corre; Phone Number: 617-724-6374; Email: ccorre@partners.org
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • Recruiting
      • University of Pittsburgh
      • Contact: Jodi Martin; Phone Number: 412-692-6351
      • Contact: Mary Branamann; Phone Number: 412-692-6350
      • Principal Investigator: Maria L Escolar, MD, MS

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 9 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Probability Sample

Study Population

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

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: NeuroVia, Inc.
• Investigators: Study Director: John Henderson, MD, NeuroVia, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 16, 2018
• Primary Completion (Anticipated): June 1, 2021
• Study Completion (Anticipated): June 1, 2021

Study Registration Dates

• First Submitted: August 30, 2017
• First Submitted That Met QC Criteria: September 7, 2017
• First Posted (Actual): September 12, 2017

Study Record Updates

• Last Update Posted (Actual): August 8, 2018
• Last Update Submitted That Met QC Criteria: August 7, 2018
• Last Verified: May 1, 2018

More Information

Terms related to this study

• Keywords: ALD; X-ALD; Adrenoleukodystropy
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Demyelinating Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Endocrine System Diseases; Disease Attributes; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Leukoencephalopathies; Adrenal Gland Diseases; Hereditary Central Nervous System Demyelinating Diseases; Peroxisomal Disorders; Adrenal Insufficiency; Disease Progression; Adrenoleukodystrophy
• Other Study ID Numbers: NV1205-008

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No