Phase 2 Rare Disease Trial, Terminated (NCT00004418)

Phase 2 Rare Disease Trial, Terminated NCT00004418 Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc. Condition: Rare Disease

Printed from Voxsanity (voxsanity.com.au) — NCT00004418 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

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Trial results

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

According to the results reported on ClinicalTrials.gov, this trial enrolled 126 participants, all of whom completed the study with none dropping out. The trial involved a single treatment group who received a combination called GTO/GTE (types of dietary oils). The study was measuring two main things: changes in the blood levels of certain fatty acids known as very long chain fatty acids (VLCFAs — a type of fat that builds up in the body in a condition called Adrenoleukodystrophy, or ALD), and whether any participants showed brain changes on MRI scans linked to childhood ALD. The reported data shows that, on average, blood levels of a specific fatty acid (called C26:0) changed by 0.41 mcg/mL (micrograms per millilitre — a measure of how much of the substance was in the blood) from the starting point before treatment. For the secondary measure, the reported data shows that zero out of 126 participants had an MRI abnormality of the type associated with childhood ALD during the study period. It is worth noting that this trial did not include a comparison group (for example, a group receiving no treatment), so the numbers above reflect only what was observed in the one group studied. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

View full results on ClinicalTrials.gov ↗

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

Boys between 18 months and 8 years old
Boys who have been diagnosed with X-linked adrenoleukodystrophy (a genetic condition) through blood tests or DNA testing, but who do not yet have symptoms
Boys whose platelet count (a measure of certain cells in the blood that help with clotting) is within the normal range

Who may not be able to join:

Boys whose MRI brain scan shows signs of the childhood brain form of this condition
Boys who have already had a bone marrow transplant
Boys who have another medical condition that the trial doctor believes would make it unsafe or impossible for them to take part in the trial (confirm with trial site)

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.

Last synced 28 July 2026

Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.

Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

GP referral letter

Print a one-page summary to share with your doctor.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I found the following past clinical trial relevant to my condition, and wanted to raise it with you. It is no longer recruiting, but I wanted to ask whether similar active trials exist, or whether its results are relevant to my care:

Trial: NCT00004418

Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Phase: Phase 2

Status: Terminated

Condition: Rare Disease

More information: https://clinicaltrials.gov/study/NCT00004418

Eligibility summary (AI generated)

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

Boys between 18 months and 8 years old
Boys who have been diagnosed with X-linked adrenoleukodystrophy (a genetic condition) through blood tests or DNA testing, but who do not yet have symptoms
Boys whose platelet count (a measure of certain cells in the blood that help with clotting) is within the normal range

Who may not be able to join:

Boys whose MRI brain scan shows signs of the childhood brain form of this condition
Boys who have already had a bone marrow transplant
Boys who have another medical condition that the trial doctor believes would make it unsafe or impossible for them to take part in the trial (confirm with trial site)

Important: Always verify eligibility with the trial site directly before applying.

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 28 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Terminated

Phase

Phase 2

Sponsor

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Registry

ClinicalTrials.gov

Start date

1 April 1998

Est. completion

1 December 2014

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov