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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01590888
Other study ID # PBT2-203
Secondary ID
Status Completed
Phase Phase 2
First received April 18, 2012
Last updated February 9, 2014
Start date April 2012
Est. completion date February 2014

Study information

Verified date February 2014
Source Prana Biotechnology Limited
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug AdministrationAustralia: Department of Health and Ageing Therapeutic Goods Administration
Study type Interventional

Clinical Trial Summary

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 more recently, 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. Based on these results, this clinical trial is investigating whether the drug will have similar effects with HD patients.

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 six months 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.


Recruitment information / eligibility

Status Completed
Enrollment 109
Est. completion date February 2014
Est. primary completion date July 2013
Accepts healthy volunteers No
Gender Both
Age group 25 Years and older
Eligibility Inclusion Criteria:

- Patients who:

1. Provide signed informed consent in accordance with local regulations.

2. Have Huntington disease including clinical features of HD and a CAG repeat number = 36.

3. Have a Total Functional Capacity between 6 and 13, inclusive.

4. Have cognitive impairment as demonstrated by a MoCA score of = 12.

5. Are = 25 years of age.

6. If taking tetrabenazine, have been on a stable dose for at least 3 months.

7. If female, are either a) of childbearing potential and compliant in using adequate birth control or b) not of childbearing potential.

8. If male, is either a) of reproductive potential and compliant in using adequate birth control or b) not of reproductive potential.

9. 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.

10. Are able to swallow oral capsules.

11. Are fluent in the English language for the administration of rating scales and have sufficient visual, hearing and motor skills to complete procedures.

Exclusion Criteria:

- Patients who:

1. Have an allergy to PBT2 or its excipients.

2. Have other known primary neurodegenerative disorders associated with dementia.

3. Have known dementia syndromes due to non-primary CNS disease.

4. Have another condition that in the investigator's judgment is resulting in clinically significant cognitive impairment.

5. In the opinion of the investigator, have any clinically significant uncontrolled medical or psychiatric illness, including history of seizures.

6. 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.

7. Have a calculated creatinine clearance at Screening of <50mL/min.

8. Have a history of malignancy diagnosed within 2 years of Screening.

9. Are pregnant or lactating females.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
PBT2
250mg capsules administered orally once per day for 26 weeks
PBT2
100mg capsules administered orally once per day for 26 weeks
Placebo
Matching capsules administered orally once per day for 26 weeks

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Prana Biotechnology Limited

Countries where clinical trial is conducted

United States,  Australia, 

References & Publications (2)

Faux NG, Ritchie CW, Gunn A, Rembach A, Tsatsanis A, Bedo J, Harrison J, Lannfelt L, Blennow K, Zetterberg H, Ingelsson M, Masters CL, Tanzi RE, Cummings JL, Herd CM, Bush AI. PBT2 rapidly improves cognition in Alzheimer's Disease: additional phase II analyses. J Alzheimers Dis. 2010;20(2):509-16. doi: 10.3233/JAD-2010-1390. — View Citation

Lannfelt L, Blennow K, Zetterberg H, Batsman S, Ames D, Harrison J, Masters CL, Targum S, Bush AI, Murdoch R, Wilson J, Ritchie CW; PBT2-201-EURO study group. Safety, efficacy, and biomarker findings of PBT2 in targeting Abeta as a modifying therapy for Alzheimer's disease: a phase IIa, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2008 Sep;7(9):779-86. doi: 10.1016/S1474-4422(08)70167-4. Epub 2008 Jul 30. Erratum in: Lancet Neurol. 2009 Nov;8(11):981. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary Safety and Tolerability of PBT2 in patients with HD by measuring Frequency of Adverse Events. 26 weeks Yes
Secondary Change from Baseline in Cognitive Test Battery 12 and 26 weeks No
Secondary Change from Baseline in Motor Function 12 and 26 weeks No
Secondary Change from Baseline in Functional Abilities 12 and 26 weeks No
Secondary Change from Baseline in Behaviour 12 and 26 weeks No
Secondary Change from Baseline in Subject and Investigator Global Assessments 12 and 26 weeks No
Secondary Change from Baseline in plasma and urine Biomarkers 12 and 26 weeks No
Secondary Change from Baseline in Brain Volumes and Function (MRI) 12 and 26 weeks No
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