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Effect of PBT2 in Patients With Early to Mid Stage Huntington Disease (Reach2HD)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Prana Biotechnology Limited
ClinicalTrials.gov Identifier:
NCT01590888
First received: April 18, 2012
Last updated: January 22, 2013
Last verified: January 2013
History of Changes
  Purpose

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.


Condition Intervention Phase
Huntington Disease
 Drug: PBT2
Drug: Placebo
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Placebo-controlled Study to Assess the Safety and Tolerability, and Efficacy of PBT2 in Patients With Early to Mid-stage Huntington Disease

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Prana Biotechnology Limited:

Primary Outcome Measures:
  • Safety and Tolerability of PBT2 in patients with HD by measuring Frequency of Adverse Events. [ Time Frame: 26 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Change from Baseline in Cognitive Test Battery [ Time Frame: 12 and 26 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline in Motor Function [ Time Frame: 12 and 26 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline in Functional Abilities [ Time Frame: 12 and 26 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline in Behaviour [ Time Frame: 12 and 26 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline in Subject and Investigator Global Assessments [ Time Frame: 12 and 26 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline in plasma and urine Biomarkers [ Time Frame: 12 and 26 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline in Brain Volumes and Function (MRI) [ Time Frame: 12 and 26 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 100
Study Start Date: April 2012
Estimated Study Completion Date: October 2013
Estimated Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: PBT2 250mg Drug: PBT2
250mg capsules administered orally once per day for 26 weeks
Experimental: PBT2 100mg Drug: PBT2
100mg capsules administered orally once per day for 26 weeks
Placebo Comparator: Sugar pill Drug: Placebo
Matching capsules administered orally once per day for 26 weeks

  Eligibility

Ages Eligible for Study:   25 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01590888

Locations
United States, California
University of California San Diego
San Diego, California, United States, 92161
United States, Colorado
Colorado Neurological Institute
Englewood, Colorado, United States, 80113
United States, Connecticut
University of Connecticut Health Center
Farmington, Connecticut, United States, 06030
United States, Florida
University of Miami
Miami, Florida, United States, 33136
United States, Maryland
University of Maryland School of Medicine
Baltimore, Maryland, United States, 21201
Johns Hopkins University
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Massachusetts General Hospital East
Charlestown, Massachusetts, United States, 02129
United States, Minnesota
Struthers Parkinson's Center
Golden Valley, Minnesota, United States, 55427
United States, Missouri
Washington University
St. Louis, Missouri, United States, 63110
United States, New York
Albany Medical College
Albany, New York, United States, 12208
Columbia University Medical Center
New York City, New York, United States, 10032
United States, Ohio
Ohio State University Medical Center
Columbus, Ohio, United States, 43210
United States, Tennessee
University of Tennessee Health Science Center
Memphis, Tennessee, United States, 38163
United States, Washington
Booth Gardner Parkinson's Care Center
Kirkland, Washington, United States, 98034
Australia, New South Wales
Westmead Hospital
Sydney, New South Wales, Australia, 2145
Australia, Victoria
Monash University
Clayton, Victoria, Australia, 3800
University of Melbourne Normanby Unit
Melbourne, Victoria, Australia, 3101
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Australia, Western Australia
Neurodegenerative Disorders Research
Perth, Western Australia, Australia, 6008
Sponsors and Collaborators
Prana Biotechnology Limited
Investigators
Principal Investigator: Ray Dorsey Johns Hopkins University
  More Information

Publications:

Responsible Party: Prana Biotechnology Limited
ClinicalTrials.gov Identifier: NCT01590888     History of Changes
Other Study ID Numbers: PBT2-203
Study First Received: April 18, 2012
Last Updated: January 22, 2013
Health Authority: United States: Food and Drug Administration
Australia: Department of Health and Ageing Therapeutic Goods Administration

Additional relevant MeSH terms:
Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
 Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Delirium, Dementia, Amnestic, Cognitive Disorders
Mental Disorders

ClinicalTrials.gov processed this record on May 23, 2013