A Randomized, Double-blind, Placebo-controlled Trial of Curcumin in Leber's Hereditary Optic Neuropathy (LHON)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00528151
Recruitment Status : Completed
First Posted : September 12, 2007
Last Update Posted : December 24, 2012
Information provided by:
Mahidol University

Brief Summary:

Background Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited ocular disorder associated with a mutation in mtDNA . The common manifestation is visual loss which caused by the respiratory chain enzymes complex dysfunction resulting in increased oxidative stress enzymes production.

Purpose To determine whether curcumin which is an antioxidant agent is beneficial to the patients with 11778 LHON mutation.

Material and Method Seventy patients with 11778 LHON mutation were randomly treated with oral curcumin (500 mg/day) and placebo for 1 year. The visual acuity, computerized visual field, electrophysiologic parameters and oxidative stress enzymes in plasma were compared before and after treatment at 3, 6, and 12 months interval.

Condition or disease Intervention/treatment Phase
Optic Atrophy, Hereditary, Leber Drug: curcumin Phase 3

Detailed Description:

Leber's hereditary optic neuropathy (LHON) is a maternally inherited disease that is characterized by a simultaneous or more common sequential bilateral loss of central vision, which typically occurs during the teenager years or early adulthood. The disease may be progressive and the patients eventually become blind. In contrast to blindness in patients caused by congenital diseases, the patients who develop blindness caused by LHON will have more trouble in their life because they previously had better vision. Although LHON is associated with mitochondrial DNA mutation at a variable nucleotide position but no certain mechanism of optic nerve injury has been found. It has been postulated that a defect in a complex of respiratory chain enzyme which is caused by mitochondrial DNA dysfunction, results in an increase of free radical substances that interfere with optic nerve function in LHON. Some antioxidant substances have been used to decrease the progression of visual impairment. However, there have been few therapeutic trials for LHON.

Curcumin, a component of tumeric, which comes from the root Curcumin longa has antioxidant, anti-inflammatory and anti-carcinogenic activities. There is some evidence which suggests that curcumin contributes to the in vitro removal of free radical gradients in thalassemic serum and to a clinical improvement of thalassemic patients. We propose a randomized controlled trial study of curcumin, which is an antioxidant substance, for LHON. This study will provide not only an insight into the therapeutic efficacy of curcumin, a kind of Thai herb, for LHON but also the development of future therapeutic strategies to prevent blindness.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Placebo-controlled Trial of Curcumin in Leber's Hereditary Optic Neuropathy (LHON)
Study Start Date : May 2005
Study Completion Date : December 2007

Arm Intervention/treatment
Placebo Comparator: 2
  1. curcumin
  2. placebo
Drug: curcumin
curcumin 250 mg twice a day in the first group. placebo 1 capsule twice a day in the second group.

Primary Outcome Measures :
  1. visual outcome [ Time Frame: 1 year ]

Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

Inclusion Criteria:

  • LHON patient with 11778 point mutation

Exclusion Criteria:

  • LHON patient with other point mutation

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00528151

Professor Wanicha Chuenkongkaew
Bangkok, Thailand, 10700
Sponsors and Collaborators
Mahidol University
Principal Investigator: Professor Wanicha L Chuenkongkaew, MD Mahidol University Identifier: NCT00528151     History of Changes
Other Study ID Numbers: 123/2547
First Posted: September 12, 2007    Key Record Dates
Last Update Posted: December 24, 2012
Last Verified: August 2004

Keywords provided by Mahidol University:
Curcumin, LHON, antioxidant, visual outcome

Additional relevant MeSH terms:
Optic Atrophy, Hereditary, Leber
Optic Atrophies, Hereditary
Eye Diseases, Hereditary
Optic Nerve Diseases
Optic Atrophy
Pathological Conditions, Anatomical
Cranial Nerve Diseases
Nervous System Diseases
Eye Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents
Antirheumatic Agents
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action