Recruiting

Observational Study

Sponsor:

NeuroVia, Inc.

Code:

NCT03278899

Conditions

X-Linked Adrenoleukodystrophy

Eligibility Criteria

Sex: Male

Age: 2 - 13

Healthy Volunteers: Not accepted

Study Details

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

Study ID

NCT03278899

Start date

Feb 16, 2018

Status verified date

May, 2018

Completion date

Jun 1, 2021

Anticipated

Primary completion date

Jun 1, 2021

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 2 - 13

Healthy Volunteers: Not accepted

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 Design

Enrollment

100 participants

Anticipated

Interventions and Outcome Measures

Primary outcome measure

  • Loes score [ Time Frame: baseline and 24 weeks ]
  • Plasma VLCFA levels [ Time Frame: baseline and 24 weeks ]
  • Neurological symptoms [ Time Frame: baseline and 24 weeks ]

Central Contacts and Locations

Central contacts

Program Director

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

Locations

Stanford University

Recruiting

Palo Alto, California, United States, 94304

Contacts

Dalia Pena-Solorzano

650-498-9732dpena25@stanford.edu

Sweta Patnaik

650-721-1458sweta@stanford.edu

Principal Investigator:

Maura Ruzhnikov, MD

Massachusetts General Hospital

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Camille Corre

617-724-6374ccorre@partners.org

Principal Investigator:

Florian S Eichler, MD

University of Pittsburgh

Recruiting

Pittsburgh, Pennsylvania, United States, 15224

Contacts

Jodi Martin

412-692-6351

Mary Branamann

412-692-6350

Principal Investigator:

Maria L Escolar, MD, MS

More Information

Sponsor

NeuroVia, Inc.

Last update posted

Aug 8, 2018

Last verified

May, 2018

Keywords

  • Adrenoleukodystropy
  • X-ALD
  • ALD

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by NeuroVia, Inc. on 2018-08-08.