ALD (Adrenoleukodystrophy)NCT03789721
Adrenoleukodystrophy National Registry Study
An observational study of ALD (Adrenoleukodystrophy) and Adrenoleukodystrophy, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
The aim of this registry to understand the natural history and disease progression in ALD and potentially develop bio-markers using the biospecimens collected using this registry. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- ALD (Adrenoleukodystrophy), Adrenoleukodystrophy, Cerebral Adrenoleukodystrophy
- What is being tested?
- Medical Record Abstraction and Biospecimen Sample Collection
- How many people
- 1,000 participants
- Where
- 1 siteUnited States
- Study dates
- 1 May 2019to Feb 2030
- How long will I take part?
- 99 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: participants with Adrenoleukodystrophy (ALD) and known/presumed mutation for ALD.
Who may be able to join
- Age 0 - 100
- ALD patients or family member meeting any of the following criteria:
- Any patient diagnosed with ALD (confirmed by positive VLCFA testing and/or genetic mutation).
- Known or presumed mutation with ALD based on pedigree or confirmed mutation in ABCD1 gene
- Participants living in the United States and territories
Who cannot join
- Patients diagnosed with ALD who lack the capacity to consent/assent AND do not have a designated legally authorized representative or guardian.
- Patients who have undergone BMT or other cellular therapy .
- Patients not fluent in English who are unable to consent in-person at the BMT Journey Clinic.
- Patients who are illiterate
- Patient determined by the PI or designee to be unlikely to complete required study components (due to language barriers, compliance issues, etc.)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, 55455
Phil Lacher, 612-626-2977, placher@umn.edu
About this study
The aim of this registry to understand the natural history and disease progression in ALD and potentially develop bio-markers using the biospecimens collected using this registry.
Read the detailed description
This is a prospective, non-therapeutic protocol designed to create and maintain a registry of participants with Adrenoleukodystrophy (ALD) and known/presumed mutation for ALD. This study also involves maintaining a prospective biorepository to collect and store buccal swab, blood, stool and urine samples as well. In this protocol, pediatric (including infants), adolescents and adult patients with confirmed or presumed ALD (based on positive VLCFA testing and/or confirmed mutation) will be offered potential study participation. Additionally, presumed mutation for ALD (based on pedigree or confirmed mutation) will be offered potential study participation. After appropriate consent (online or in-person), subjects will be requested to provide a medical history (with authorization of release of medical records), longitudinal biospecimens, and permission to perform laboratory analyses on these samples. The overall goal is to understand the natural disease course in affected and unaffected patients (identified patients and relatives with a diagnosis of ALD), as well as women with ALD to assemble a resource of clinical, medical, and biological data from the participants. This study also aims to understand the outcomes of this disease, as well as possibly develop biomarkers to identify prognostic markers for disease progression, which may help develop effective interventions. The biospecimen bank and registry will provide access to samples and data for the ongoing studies as well as will provide an important resource for the future research.
- Being tested
- Medical Record Abstraction, Biospecimen Sample Collection
- Keywords
- Registry, VLCFA, ABCD1, X-chromosome
- Medical subject headings
- Adrenoleukodystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Medical Record AbstractionOther
Collect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually.
Biospecimen Sample CollectionOther
Collect research samples, when feasible for those diagnosed with ALD.
| Study group | Role | What they receive |
|---|---|---|
| Adrenoleukodystrophy | All patients living in the United States diagnosed with adrenoleukodystrophy, either by newborn screen, based on family history or otherwise, are eligible to participate in this study. Receives: Other: Medical Record Abstraction; Other: Biospecimen Sample Collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Collect Clinical and Epidemiological Data
Collect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually.
Measured: 10 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2018
- Study start1 May 2019
- Primary completionFeb 2030 (estimated)
- Study completionFeb 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALD (Adrenoleukodystrophy)Studies 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
- 1,000 participants (estimated)
- Target duration
- 99 Years
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03789721
- Sponsor's protocol ID
- 2019NTLS027
- Other IDs
- MT2019-01
- Status last verified
- May 2026
- First submitted
- 14 Dec 2018
- Last updated
- 12 May 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Adrenoleukodystrophy National Registry Study
- ClinicalTrials.gov ID
- NCT03789721
- Acronym / sponsor ID
- 2019NTLS027
- Keywords
- Registry, VLCFA, ABCD1, X-chromosome
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 1 May 2019 (actual)
- Primary completion
- Feb 2030 (estimated)
- Study completion
- Feb 2030 (estimated)
- First posted
- 31 Dec 2018 (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.
The aim of this registry to understand the natural history and disease progression in ALD and potentially develop bio-markers using the biospecimens collected using this registry.
Detailed description
This is a prospective, non-therapeutic protocol designed to create and maintain a registry of participants with Adrenoleukodystrophy (ALD) and known/presumed mutation for ALD. This study also involves maintaining a prospective biorepository to collect and store buccal swab, blood, stool and urine samples as well. In this protocol, pediatric (including infants), adolescents and adult patients with confirmed or presumed ALD (based on positive VLCFA testing and/or confirmed mutation) will be offered potential study participation. Additionally, presumed mutation for ALD (based on pedigree or confirmed mutation) will be offered potential study participation. After appropriate consent (online or in-person), subjects will be requested to provide a medical history (with authorization of release of medical records), longitudinal biospecimens, and permission to perform laboratory analyses on these samples. The overall goal is to understand the natural disease course in affected and unaffected patients (identified patients and relatives with a diagnosis of ALD), as well as women with ALD to assemble a resource of clinical, medical, and biological data from the participants. This study also aims to understand the outcomes of this disease, as well as possibly develop biomarkers to identify prognostic markers for disease progression, which may help develop effective interventions. The biospecimen bank and registry will provide access to samples and data for the ongoing studies as well as will provide an important resource for the future research.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 99 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adrenoleukodystrophy | – | Other: Medical Record Abstraction, Other: Biospecimen Sample CollectionAll patients living in the United States diagnosed with adrenoleukodystrophy, either by newborn screen, based on family history or otherwise, are eligible to participate in this study. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- participants with Adrenoleukodystrophy (ALD) and known/presumed mutation for ALD.
Key inclusion criteria (5 listed)
- Age 0 - 100
- ALD patients or family member meeting any of the following criteria:
- Participants living in the United States and territories
Key exclusion criteria (5 listed)
- Patients diagnosed with ALD who lack the capacity to consent/assent AND do not have a designated legally authorized representative or guardian.
- Patients who have undergone BMT or other cellular therapy .
- Patients not fluent in English who are unable to consent in-person at the BMT Journey Clinic.
- Patients who are illiterate
- Patient determined by the PI or designee to be unlikely to complete required study components (due to language barriers, compliance issues, etc.)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Medical Record AbstractionCollect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually. | Other | Adrenoleukodystrophy |
| Biospecimen Sample CollectionCollect research samples, when feasible for those diagnosed with ALD. | Other | Adrenoleukodystrophy |
Primary outcome
Collect Clinical and Epidemiological Data
Time frame: 10 Years
Collect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2026.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of ALD (Adrenoleukodystrophy) and Adrenoleukodystrophy, run by Masonic Cancer Center, University of Minnesota at 1 site 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 5 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: looking for participants now, as of the sponsor's last update on 12 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ashish Gupta, MD (612-626-2961, gupta461@umn.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Collect Clinical and Epidemiological Data (time frame: 10 Years). 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 12 May 2026.