AdrenoleukodystrophyPhase 2NCT00004418
Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
A Phase 2 study testing glyceryl trierucate/glyceryl trioleate in people with Adrenoleukodystrophy, run by Hugo W. Moser Research Institute at Kennedy Krieger, Inc. at 2 sites in United States.
Trial summary
OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy. II. Compare the frequency and severity of neurological disability of study patients with untreated historical controls. Read more about this study
- Who is it for?
- Ages 18 months to 8 years, men only
- Conditions the study is for
- Adrenoleukodystrophy
- What is being tested?
- glyceryl trierucate/glyceryl trioleate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 126 participants
- Where
- 2 sitesUnited States
- Study dates
- Apr 1998to Dec 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 months to 8 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Boys between the age of 18 months and 8 years of age
- Biochemically proven asymptomatic X-linked adrenoleukodystrophy determined by elevation of very long chain fatty acids or DNA analysis.
- Platelet count in normal range
Who cannot join
- Abnormal MRI consistent with childhood cerebral disease
- Boys who have undergone bone marrow transplantation
- Other medical condition which in the opinion of the investigator prevents evaluation or treatment
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Johns Hopkins Hospital
Baltimore, Maryland, 21287-6681
About this study
Official title: Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy
OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy.
II. Compare the frequency and severity of neurological disability of study patients with untreated historical controls.
Read the detailed description
PROTOCOL OUTLINE: This is an open label study. Patients must follow dietary instructions as provided by the investigator. Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. Patients complete a neuropsychological scoring scale questionnaire to measure neurological disability. Patients undergo physical examinations including magnetic resonance imaging and magnetic resonance spectroscopy of the head.
Patients are followed monthly for 6 months, then every 3 months until they reach the age of 13 years or death.
- Conditions
- Adrenoleukodystrophy
- Being tested
- glyceryl trierucate/glyceryl trioleate
- Keywords
- adrenoleukodystrophy, inborn errors of metabolism, rare disease, sphingolipidoses
- Medical subject headings
- Adrenoleukodystrophy, Metabolism, Inborn Errors, Rare Diseases, Sphingolipidoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
glyceryl trierucate/glyceryl trioleateDrug
Also called Lorenzo's oil
Administration of glyceryl trierucate/glyceryl trioleate
| Study group | Role | What they receive |
|---|---|---|
| Glyceryl trierucate/glyceryl trioleate | Experimental | Treatment of all enrolled participants. Dosage form is a liquid oil taken orally. Dose is to provide 20% of daily calories. Daily for duration of trial Receives: Drug: glyceryl trierucate/glyceryl trioleate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels
we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.
Measured: Baseline, an average of 10 years, up to age 13
Secondary outcomes (1)
Number of Participants With T2 MRI Abnormality
We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).
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 posted19 Oct 1999 (estimated)
- Study startApr 1998
- Primary completionDec 2014
- Study completionDec 2014
Why it stopped: No funding, move to expanded access
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 126 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Gerald V Raymond, M.D., Principal investigator, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004418
- Sponsor's protocol ID
- 199/13312
- Other IDs
- Nutricia-Loma Linda, KKI-FDR000685
- Status last verified
- Oct 2020
- First submitted
- 18 Oct 1999
- Last updated
- 23 Oct 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy
- ClinicalTrials.gov ID
- NCT00004418
- Acronym / sponsor ID
- 199/13312
- Conditions
- Adrenoleukodystrophy
- Keywords
- adrenoleukodystrophy, inborn errors of metabolism, rare disease, sphingolipidoses
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: No funding, move to expanded access
- Enrolment
- 126 participants (actual)
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.University, hospital or other
- Study start
- Apr 1998
- Primary completion
- Dec 2014 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 19 Oct 1999 (estimated)
- Results
- Posted 23 Oct 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy.
II. Compare the frequency and severity of neurological disability of study patients with untreated historical controls.
Detailed description
PROTOCOL OUTLINE: This is an open label study. Patients must follow dietary instructions as provided by the investigator. Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. Patients complete a neuropsychological scoring scale questionnaire to measure neurological disability. Patients undergo physical examinations including magnetic resonance imaging and magnetic resonance spectroscopy of the head.
Patients are followed monthly for 6 months, then every 3 months until they reach the age of 13 years or death.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Glyceryl trierucate/glyceryl trioleate | Experimental | Drug: glyceryl trierucate/glyceryl trioleateTreatment of all enrolled participants. Dosage form is a liquid oil taken orally. Dose is to provide 20% of daily calories. Daily for duration of trial |
Who may qualify
- Ages
- 18 months to 8 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Boys between the age of 18 months and 8 years of age
- Biochemically proven asymptomatic X-linked adrenoleukodystrophy determined by elevation of very long chain fatty acids or DNA analysis.
- Platelet count in normal range
Key exclusion criteria (3 listed)
- Abnormal MRI consistent with childhood cerebral disease
- Boys who have undergone bone marrow transplantation
- Other medical condition which in the opinion of the investigator prevents evaluation or treatment
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 |
|---|---|---|
| glyceryl trierucate/glyceryl trioleateAlso: Lorenzo's oilAdministration of glyceryl trierucate/glyceryl trioleate | Drug | Glyceryl trierucate/glyceryl trioleate |
Primary outcome
Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels
Time frame: Baseline, an average of 10 years, up to age 13
we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.
1 secondary outcome
Number of Participants With T2 MRI Abnormality
Time frame: 10 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Gerald V Raymond, M.D. principal investigator
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing glyceryl trierucate/glyceryl trioleate in people with Adrenoleukodystrophy, run by Hugo W. Moser Research Institute at Kennedy Krieger, Inc. at 2 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 3 inclusion and 3 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: stopped early, as of the sponsor's last update on 23 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Oct 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels (time frame: Baseline, an average of 10 years, up to age 13). 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 23 Oct 2020.