A Phase III Trial of Lorenzo’s Oil in Adrenomyeloneuropathy

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Brief Title

A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

Official Title

A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

Brief Summary

      Study of the use of Lorenzo's oil in adults with adrenomyeloneuropathy, the adult form of
      Lorenzo's oil.
    

Detailed Description

      This is a double-masked placebo controlled study of glyceryl trioleate-glyceryl trierucate
      (Lorenzo's Oil (LO)) therapy in adrenomyeloneuropathy (AMN), the adult form of X-linked
      adrenoleukodystrophy (X-ALD). AMN is a slowly progressive distal axonopathy that involves the
      long tracts of the spinal cord and differs from the rapidly progressive inflammatory cerebral
      forms that most commonly affect boys and adolescents. All forms of X-ALD are associated with
      the abnormal accumulation of very long chain fatty acids (VLCFA) in plasma and tissues. The
      oral administration of LO normalizes plasma VLCFA levels within 4 weeks. While previous
      therapeutic trials of LO therapy in patients with the cerebral forms of X-ALD have been
      disappointing, recent studies suggest that it is beneficial in two types of X-ALD: 1) as a
      preventive of neurological involvement in asymptomatic boys; and 2) in AMN, where it appears
      to slow the rate of progression. None of the previous studies have been controlled, and we
      are now conducting the first placebo-controlled trial.

      The 4-year study will include 120 men with AMN who do not have evidence of cerebral
      involvement, and 120 women who are heterozygous for X-ALD and have an AMN-like syndrome. The
      rate of progression will be compared in the LO and placebo groups using the Kurtzke EDSS
      score as the primary outcome and a variety of secondary outcomes.

      Adrenomyeloneuropathy affects about 1 in 40,000 men and 1 in 30,000 women. It is a
      progressive disorder that leads to inability to walk and other severe deficits. This study
      will provide definitive information whether Lorenzo's Oil therapy can slow progression.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Clinical Progression of the disorder

Secondary Outcome

 Determine the degree to which newly developed methods to assess spinal cord function and structure in adrenomyeloneuropathy, namely quantitative sensorimotor tests and spinal cord imaging, can act as early surrogate markers of disease progression.

Condition

Adrenomyeloneuropathy

Intervention

Lorenzo's oil


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

240

Start Date

March 2005

Completion Date

October 2007

Primary Completion Date

October 2007

Eligibility Criteria

        Inclusion Criteria:

          -  Males or females age 18 years or older in whom the diagnosis of X-ALD has been
             confirmed by VLCFA assay and/or mutation analysis.

          -  Clinical evidence of spinal cord involvement with EDSS score between 1 and 6.5.
             Patients with an EDSS score of 6.5 are severely affected but have retained the
             capacity to walk 20 meters with the aid of a walker, crutch, or two canes.

          -  Either a normal brain MRI, or a type 3 pattern of MRI abnormality in which the
             abnormality is considered to represent the centripetal extension of the distal
             axonopathy.

          -  Adrenal function assessed by measurement of plasma ACTH and appropriate steroid
             replacement if adrenal insufficiency is present.

        Exclusion Criteria:

          -  Kurtzke EDSS score of >6.5.

          -  Cognitive or behavioral abnormalities that impair capacity to give informed consent or
             carry out procedures that form part of the protocol.

          -  Current use, or use within 3 months, of Lorenzo's oil or other therapies that may
             alter the course of X-ALD. Bone marrow transplant will be a permanent exclusion
             criterion.

          -  Contraindications for MRI procedure such as subjects with paramagnetic materials in
             the body, such as aneurysm clips, pacemakers, intraocular metal or cochlear implants.

          -  Subjects who are pregnant.

          -  Allergies to peppermint

          -  Presence of non-specific conditions that may interfere with clinical assessment or
             participation in the protocol.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Gerald V Raymond, M.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00545597

Organization ID

R01HD039276

Secondary IDs

R01FD003030

Responsible Party

Sponsor

Study Sponsor

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborators

 Food and Drug Administration (FDA)

Study Sponsor

Gerald V Raymond, M.D., Principal Investigator, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.


Verification Date

October 2007