A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

• Status: Terminated
• Trial end date: 2007-10-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
• Collaborator: Food and Drug Administration (FDA)

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.