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Status unknownNCT03278899Updated Aug 8, 2018

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

An observational study in X-Linked Adrenoleukodystrophy, sponsored by NeuroVia, Inc.. Status unknown at 3 sites in United States. Open to male participants aged 2 Years to 13 Years. Per ClinicalTrials.gov, last updated 2018-08-08.

Sponsored by NeuroVia, Inc. · Observational

The sponsor has not verified this record recently (last verified May 2018), so the status shown — last known as Recruiting — may be out of date.
Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
100
Ages
2 Years to 13 Years
Sex
Male
01

Study 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.

Read the 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.

02

Conditions studied

  • X-Linked Adrenoleukodystrophy

Keywords

  • Adrenoleukodystropy
  • X-ALD
  • ALD
03

Who can participate

Ages eligible
2 Years to 13 Years
Sexes eligible
Male
Accepts healthy volunteers
No
Sampling method
Probability sample

Study population

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

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

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
04

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
100 participants (estimated)
Patient registry
No
Biospecimen retention
Samples without dna
05

What researchers measure

Primary outcomes

  1. Loes score

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

    Time frame: baseline and 24 weeks

  2. Plasma VLCFA levels

    Change from baseline plasma VLCFA levels

    Time frame: baseline and 24 weeks

  3. Neurological symptoms

    Development of new neurological symptoms throughout the study

    Time frame: baseline and 24 weeks

06

Study locations

3 of 3 sites recruiting
  • Stanford University
    Palo Alto, California 94304, United States
    Recruiting
  • Massachusetts General Hospital
    Boston, Massachusetts 02114, United States
    • Camille Corre · Contact · ccorre@partners.org · 617-724-6374
    • Florian S Eichler, MD · Principal investigator
    Recruiting
  • University of Pittsburgh
    Pittsburgh, Pennsylvania 15224, United States
    • Jodi Martin · Contact · 412-692-6351
    • Mary Branamann · Contact · 412-692-6350
    • Maria L Escolar, MD, MS · Principal investigator
    Recruiting
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT03278899
Lead sponsor
NeuroVia, Inc.
Responsible party
Sponsor
First posted
Sep 12, 2017
Start date
Feb 16, 2018
Primary completion
Jun 1, 2021 (estimated)
Completion
Jun 1, 2021 (estimated)
Last update
Aug 8, 2018

Study contacts

Program Director
Contact
patients@neurovia-inc.com
415-870-6967
John Henderson, MD
study director · NeuroVia, Inc.

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in May 2018. You cannot join it, but the record below documents what was studied.

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