AdrenoleukodystrophyNCT00004450
Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
A study testing glyceryl trierucate, glyceryl trioleate and interferon beta in people with Adrenoleukodystrophy, run by FDA Office of Orphan Products Development.
Trial summary
OBJECTIVES: I. Evaluate the efficacy of interferon beta and thalidomide in male patients with adrenoleukodystrophy who show evidence of brain inflammatory response and are receiving concurrent glyceryl trierucate and glyceryl trioleate (Lorenzo's oil). II. Read more about this study
- Who is it for?
- Ages 4 years and older, men only
- Conditions the study is for
- Adrenoleukodystrophy
- What is being tested?
- glyceryl trierucate, glyceryl trioleate and interferon beta
- How many people
- 60 participants
- Where
- No sites listed yet
- Study dates
- Aug 1998to Nov 2000
- Phase
- Interventional
- 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 4 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics-- Adrenoleukodystrophy (ALD) diagnosis based on history and examination, MRI, and biochemical assay
Clinical evidence of rapidly progressive phase of cerebral ALD must include 2 or more of the following during the preceding year:
Significant and progressive impairment of school performance Significant loss of cognitive function leading to an IQ of 75 or less Progressive impairment of the ability to understand spoken words Progressive impairment of vision Progressive deterioration of handwriting Progressive difficulty in walking Progressive impairment in speech articulation, and vocabulary Progressive weakness of one or more limbs
Must have MRI abnormalities characteristic of cerebral ALD, especially evidence of the breakdown of the blood-brain barrier using gadolinium contrast medium and magnetization transfer technique Evidence of brain white matter inflammatory response Must not meet criteria for bone marrow transplantation Not in an apparent vegetative state --Prior/Concurrent Therapy-- Concurrent glyceryl trierucate and glyceryl trioleate (Lorenzo's oil) therapy required --Patient Characteristics-- Effective contraception required of all patients
About this study
OBJECTIVES: I. Evaluate the efficacy of interferon beta and thalidomide in male patients with adrenoleukodystrophy who show evidence of brain inflammatory response and are receiving concurrent glyceryl trierucate and glyceryl trioleate (Lorenzo's oil).
II. Evaluate the progress of the disease and possible side effects of the medication in these patients.
Read the detailed description
PROTOCOL OUTLINE: This is randomized, double blind, placebo controlled study. Patients are randomized to receive beta interferon and thalidomide placebo (arm I), thalidomide and beta interferon placebo (arm II), or placebo for both beta interferon and thalidomide (arm III). Patients receive interferon beta by subcutaneous injection and thalidomide orally. All patients are maintained on glyceryl trierucate and glyceryl trioleate (Lorenzo's oil) therapy.
Patients are followed at 3, 6, and 12 months and then may be followed every 6 months thereafter.
- Conditions
- Adrenoleukodystrophy
- Being tested
- glyceryl trierucate, glyceryl trioleate, interferon beta, thalidomide
- 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 trierucateDrug
glyceryl trioleateDrug
interferon betaDrug
thalidomideDrug
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 startAug 1998
- Study completionNov 2000
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Masking
- Double blind
- Enrolment
- 60 participants
Sponsor and investigators
- Lead sponsor
- FDA Office of Orphan Products Development
- Sponsor type
- US federal agency
- Collaborators
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Investigators
- Hugo Wolfgang Moser, Study chair, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004450
- Sponsor's protocol ID
- 199/13532
- Other IDs
- KKI-94-06-16-01, KKI-FDR001052
- Status last verified
- Jan 2001
- First submitted
- 18 Oct 1999
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
- ClinicalTrials.gov ID
- NCT00004450
- Acronym / sponsor ID
- 199/13532
- Conditions
- Adrenoleukodystrophy
- Keywords
- adrenoleukodystrophy, inborn errors of metabolism, rare disease, sphingolipidoses
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants
- Lead sponsor
- FDA Office of Orphan Products DevelopmentUS federal agency
- Collaborators
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Study start
- Aug 1998
- Study completion
- Nov 2000
- First posted
- 19 Oct 1999 (estimated)
- 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.
OBJECTIVES: I. Evaluate the efficacy of interferon beta and thalidomide in male patients with adrenoleukodystrophy who show evidence of brain inflammatory response and are receiving concurrent glyceryl trierucate and glyceryl trioleate (Lorenzo's oil).
II. Evaluate the progress of the disease and possible side effects of the medication in these patients.
Detailed description
PROTOCOL OUTLINE: This is randomized, double blind, placebo controlled study. Patients are randomized to receive beta interferon and thalidomide placebo (arm I), thalidomide and beta interferon placebo (arm II), or placebo for both beta interferon and thalidomide (arm III). Patients receive interferon beta by subcutaneous injection and thalidomide orally. All patients are maintained on glyceryl trierucate and glyceryl trioleate (Lorenzo's oil) therapy.
Patients are followed at 3, 6, and 12 months and then may be followed every 6 months thereafter.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Masking
- Double blind
Who may qualify
- Ages
- 4 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 | Drug | – |
| glyceryl trioleate | Drug | – |
| interferon beta | Drug | – |
| thalidomide | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
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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.
Hugo Wolfgang Moser study chair
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Questions about this trial
What is this clinical trial studying?
A study testing glyceryl trierucate, glyceryl trioleate and interferon beta in people with Adrenoleukodystrophy, run by FDA Office of Orphan Products Development.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: finished, as of the sponsor's last update on 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
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 25 Mar 2015.