X-Linked AdrenoleukodystrophyNCT03278899
A Study to Prospectively Assess Disease Progression in Male Children With X-ALD
An observational study of X-Linked Adrenoleukodystrophy, run by NeuroVia, Inc. at 3 sites in United States.
Trial 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 more about this study
- Who is it for?
- Ages 2 to 13 years, men only
- Conditions the study is for
- X-Linked Adrenoleukodystrophy
- How many people
- 100 participants
- Where
- 3 sitesUnited States
- Study dates
- 16 Feb 2018to 1 Jun 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 13 years
- Men only
- No healthy volunteers
- Child
Study population: Patients will be selected from participating sites, referring physicians, and outreach communities
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites, 3 recruiting
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
Camille Corre, 617-724-6374, ccorre@partners.org
Florian S Eichler, MD
- Recruiting
Stanford University
Palo Alto, California, 94304
Dalia Pena-Solorzano, 650-498-9732, dpena25@stanford.edu
Sweta Patnaik, 650-721-1458, sweta@stanford.edu
- Recruiting
University of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Jodi Martin, 412-692-6351
Mary Branamann, 412-692-6350
About this study
Official title: 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.
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.
- Conditions
- X-Linked Adrenoleukodystrophy
- Keywords
- Adrenoleukodystropy, X-ALD, ALD
- Medical subject headings
- Adrenoleukodystrophy
What is being measured
The primary outcome is the main question the study is designed to answer.
Loes score
Percent change from baseline in brain lesions assessed as Loes score will be calculated
Measured: baseline and 24 weeks
Plasma VLCFA levels
Change from baseline plasma VLCFA levels
Measured: baseline and 24 weeks
Neurological symptoms
Development of new neurological symptoms throughout the study
Measured: baseline and 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2017
- Study start16 Feb 2018
- Primary completion1 Jun 2021 (estimated)
- Study completion1 Jun 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-Linked AdrenoleukodystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- NeuroVia, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- John Henderson, MD, Study director, NeuroVia, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03278899
- Sponsor's protocol ID
- NV1205-008
- Status last verified
- May 2018
- First submitted
- 30 Aug 2017
- Last updated
- 8 Aug 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Non-interventional Study to Prospectively Assess Baseline Status and Disease Progression in Male Children With X-Linked Adrenoleukodystrophy
- ClinicalTrials.gov ID
- NCT03278899
- Acronym / sponsor ID
- NV1205-008
- Conditions
- X-Linked Adrenoleukodystrophy
- Keywords
- Adrenoleukodystropy, X-ALD, ALD
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- NeuroVia, Inc.Industry (pharma, biotech, devices)
- Study start
- 16 Feb 2018 (actual)
- Primary completion
- 1 Jun 2021 (estimated)
- Study completion
- 1 Jun 2021 (estimated)
- First posted
- 12 Sep 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 to 13 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Patients will be selected from participating sites, referring physicians, and outreach communities
Key inclusion criteria (5 listed)
- 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:
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Loes score
Time frame: baseline and 24 weeks
Percent change from baseline in brain lesions assessed as Loes score will be calculated
Plasma VLCFA levels
Time frame: baseline and 24 weeks
Change from baseline plasma VLCFA levels
Neurological symptoms
Time frame: baseline and 24 weeks
Development of new neurological symptoms throughout the study
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Aug 2018.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Henderson, MD study director
NeuroVia, Inc.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of X-Linked Adrenoleukodystrophy, run by NeuroVia, Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 Aug 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Program Director (415-870-6967, patients@neurovia-inc.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Loes score (time frame: baseline and 24 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Aug 2018.