Effect of PBT2 in Patients With Early to Mid Stage Huntington Disease

Completed · Phase 2 · Has a placebo group

Conditions studied: Huntington Disease

In brief

Huntington disease (HD) is an inherited neurodegenerative disease which affects over 30,000 people in both the United States and Australia. HD is characterized by brain cell death that usually begins between the ages of 30 to 50, and results in motor, cognitive and behavioral signs and symptoms. While there are medications to help relieve some of the disease symptoms, there is no known treatment to address the cognitive impairment associated with HD. Normally occurring metals in the brain play a significant role in diseases such as Alzheimer disease and HD. PBT2 is a drug designed to interrupt interactions between these biological metals and target proteins in the brain, to prevent deterioration of brain cells. PBT2, has shown in animal models, and as well as in a small group of patients with Alzheimer's disease, it may improve cognition. There is some indication in animal models of HD, that the drug may improve motor function and control and reduce the amount of brain cell degeneration. PBT2-203 will evaluate how safe and well tolerated PBT2 is at a dose of 100 mg or 250 mg a day administered as oral daily capsules compared to a placebo over a six month treatment period. The trial will also measure whether there is an effect on cognitive abilities as well as other HD symptoms including motor and overall functioning of individuals with HD.

Key facts

Study ID

NCT01590888

Run by

Prana Biotechnology Limited

People needed

109

Starts

2012-04-01

Expected to finish

2014-02-01

Last updated by the study team

2016-07-18

Who can join

Age: 25 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients who:
• Provide signed informed consent in accordance with local regulations.
• Have Huntington disease including clinical features of HD and a CAG repeat number ≥ 36.
• Have a Total Functional Capacity between 6 and 13, inclusive.
• Have cognitive impairment as demonstrated by a MoCA score of ≥ 12.
• Are ≥ 25 years of age.
• If taking tetrabenazine, have been on a stable dose for at least 3 months.
• If female, are either a) of childbearing potential and compliant in using adequate birth control or b) not of childbearing potential.
• If male, is either a) of reproductive potential and compliant in using adequate birth control or b) not of reproductive potential.
• Have a study partner who is willing to provide consent and spends on average at least two hours a day for at least four days a week with the patient, is fluent in the English language, and who agrees to attend certain study visits and provide accurate information about the patient.
• Are able to swallow oral capsules.
• Are fluent in the English language for the administration of rating scales and have sufficient visual, hearing and motor skills to complete procedures.

You may not qualify if…

• Patients who:
• Have an allergy to PBT2 or its excipients.
• Have other known primary neurodegenerative disorders associated with dementia.
• Have known dementia syndromes due to non-primary CNS disease.
• Have another condition that in the investigator's judgment is resulting in clinically significant cognitive impairment.
• In the opinion of the investigator, have any clinically significant uncontrolled medical or psychiatric illness, including history of seizures.
• Have clinically significant cardiovascular, hepatic, renal, pulmonary, metabolic or endocrine disease that, in the opinion of the investigator, would interfere with an individual's participation in the study.
• Have a calculated creatinine clearance at Screening of <50mL/min.
• Have a history of malignancy diagnosed within 2 years of Screening.
• Are pregnant or lactating females.

Where it is running

• University of California San Diego — San Diego, California, United States
• Colorado Neurological Institute — Englewood, Colorado, United States
• University of Connecticut Health Center — Farmington, Connecticut, United States
• University of Miami — Miami, Florida, United States
• University of Maryland School of Medicine — Baltimore, Maryland, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• Massachusetts General Hospital East — Charlestown, Massachusetts, United States
• Struthers Parkinson's Center — Golden Valley, Minnesota, United States
• Washington University — St Louis, Missouri, United States
• Albany Medical College — Albany, New York, United States
• Columbia University Medical Center — New York, New York, United States
• Ohio State University Medical Center — Columbus, Ohio, United States
• University of Tennessee Health Science Center — Memphis, Tennessee, United States
• Booth Gardner Parkinson's Care Center — Kirkland, Washington, United States
• Westmead Hospital — Sydney, New South Wales, Australia
• Calvary Health Care Bethlehem — Clayton, Victoria, Australia
• University of Melbourne Normanby Unit - St Vincents/St Georges — Melbourne, Victoria, Australia
• Royal Melbourne Hospital — Parkville, Victoria, Australia
• Neurodegenerative Disorders Research — Perth, Western Australia, Australia

Full record on ClinicalTrials.gov

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