Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes

NCT00887562 | Completed Phase 2 Results

• 2 other identifiers: AAAC9240SNT-II-007
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to compare the efficacy of two (2) different doses of idebenone with that of a placebo over a one month period on cerebral lactate concentration as measured by magnetic resonance spectroscopy.

Conditions

MELAS Syndrome

Outcome Measures

Primary Outcomes (1)

• Mean Change in Cerebral Lactate Concentration (as Measured by Magnetic Resonance Spectroscopy)

  To compare the efficacy of 1 month treatment with 2 different doses of idebenone with that of placebo on cerebral lactate concentration as measured by magnetic resonance spectroscopy (MRS)

  Up to 4 weeks from baseline

Secondary Outcomes (2)

• Mean Change in Venous Lactate Concentration

  Up to 4 weeks from baseline

• Mean Change in Score on the Fatigue Severity Scale (FSS)

  Baseline and Week 4

Study Arms (3)

Idebenone 900 mg/day

EXPERIMENTAL

Idebenone 900 mg/day

Drug: Idebenone

Idebenone 2250 mg/day

EXPERIMENTAL

Idebenone 2250 mg/day

Drug: Idebenone

placebo

PLACEBO COMPARATOR

Placebo

Other: Placebo

Interventions

Idebenone DRUG

900 mg/day for 1 month

Also known as: active drug

Idebenone 900 mg/day

Placebo OTHER

Placebo - No idebenone

Also known as: No active drug

placebo

Eligibility Criteria

• Age: 8 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of MELAS with confirmed A3243G mtDNA mutation, or evidence of central nervous system involvement (cognitive problems, migraines, memory loss)
• Cerebral lactate level equal to or greater than 5.0 i.u. at baseline
• Patients at least 8 and \< 65 years of age at baseline
• Patients with a body weight \> 37 kg/82 lbs at baseline
• Stable co-medication/vitamins/supplements within 1 month prior to baseline
• Patients who in the opinion of the investigator are able to comply with the requirements of the study, including swallowing the study medication
• Negative urine pregnancy test at screening and baseline (female patients of childbearing potential)

You may not qualify if:

• Contraindication to MRS (e.g. metal implant, claustrophobia)
• Stroke like event within 2 months prior to baseline
• Treatment with idebenone at any dose, or coenzyme Q10 at doses above 100mg/d within 1 month prior to baseline
• Inadequate contraception use
• Pregnancy and/or breast-feeding
• Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of aspartate aminotransferase (AST), alanine aminotransferase (ALT) or creatinine
• Current abuse of drugs or alcohol
• Participation in a trial of another investigational drug within the last month
• Other factor that, in the investigator's opinion, excludes the patient from entering the study

Sponsors & Collaborators

• Michio Hirano: lead
• Santhera Pharmaceuticals: collaborator

Study Sites (1)

Columbia University Medical Center

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

MELAS Syndrome

Interventions

idebenone; Bulk Drugs

Condition Hierarchy (Ancestors)

Mitochondrial Encephalomyopathies; Mitochondrial Myopathies; Muscular Diseases; Musculoskeletal Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Neuromuscular Diseases; Vascular Diseases; Cardiovascular Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Mitochondrial Diseases

Intervention Hierarchy (Ancestors)

Pharmaceutical Preparations

Limitations and Caveats

Clinical variability in severity of disease among participants

Results Point of Contact

• Title: Michio Hirano, MD
• Organization: Columbia University

mh29@cumc.columbia.edu

12123051048

Study Officials

• Michio Hirano, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Neurology

Study Record Dates

• First Submitted: April 23, 2009
• First Posted: April 24, 2009
• Study Start: May 1, 2009
• Primary Completion: July 1, 2012
• Study Completion: July 1, 2012
• Last Updated: October 26, 2016
• Results First Posted: August 10, 2016

Record last verified: 2016-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)