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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01590888




Registration number
NCT01590888
Ethics application status
Date submitted
18/04/2012
Date registered
3/05/2012
Date last updated
18/07/2016

Titles & IDs
Public title
Effect of PBT2 in Patients With Early to Mid Stage Huntington Disease
Scientific 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
Secondary ID [1] 0 0
PBT2-203
Universal Trial Number (UTN)
Trial acronym
Reach2HD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Huntington Disease 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PBT2
Treatment: Drugs - PBT2
Treatment: Drugs - Placebo

Experimental: PBT2 250mg -

Experimental: PBT2 100mg -

Placebo comparator: Sugar pill -


Treatment: Drugs: PBT2
250mg capsules administered orally once per day for 26 weeks

Treatment: Drugs: PBT2
100mg capsules administered orally once per day for 26 weeks

Treatment: Drugs: Placebo
Matching capsules administered orally once per day for 26 weeks
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and Tolerability of PBT2 in Patients With HD
Timepoint [1] 0 0
Baseline to 26 weeks
Secondary outcome [1] 0 0
Change From Baseline in Cognitive Test Battery - Composite z Scores
Timepoint [1] 0 0
Baseline to 26 weeks
Secondary outcome [2] 0 0
Change From Baseline in Motor Function
Timepoint [2] 0 0
Baseline to 26 weeks
Secondary outcome [3] 0 0
Change From Baseline in Functional Abilities
Timepoint [3] 0 0
Baseline to 26 weeks
Secondary outcome [4] 0 0
Change From Baseline in Behaviour
Timepoint [4] 0 0
Baseline to 26 weeks
Secondary outcome [5] 0 0
Change From Baseline in Investigator Global Assessments by Efficacy Index
Timepoint [5] 0 0
Baseline to 26 weeks
Secondary outcome [6] 0 0
Change From Baseline in Blood Biomarkers
Timepoint [6] 0 0
Baseline to 26 weeks
Secondary outcome [7] 0 0
Change From Baseline in Brain Function (MRI)
Timepoint [7] 0 0
Baseline to 26 weeks
Secondary outcome [8] 0 0
Change From Baseline in Blood Biomarkers
Timepoint [8] 0 0
Baseline to 26 weeks
Secondary outcome [9] 0 0
Change From Baseline in Blood Biomarkers - Selenium
Timepoint [9] 0 0
Baseline to 26 weeks
Secondary outcome [10] 0 0
Change From Baseline in Urine Biomarkers
Timepoint [10] 0 0
Baseline to 26 weeks
Secondary outcome [11] 0 0
Change From Baseline in Brain Function (MRI)
Timepoint [11] 0 0
Baseline to 26 weeks
Secondary outcome [12] 0 0
Change From Baseline in Cognitive Test Battery - TMT Part B
Timepoint [12] 0 0
Baseline to 26 weeks

Eligibility
Key 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.
Minimum age
25 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/02/2014
Sample size
Target
Accrual to date
Final
109
Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
Recruitment hospital [1] 0 0
Westmead Hospital - Sydney
Recruitment hospital [2] 0 0
Calvary Health Care Bethlehem - Clayton
Recruitment hospital [3] 0 0
University of Melbourne Normanby Unit - St Vincents/St Georges - Melbourne
Recruitment hospital [4] 0 0
Royal Melbourne Hospital - Parkville
Recruitment hospital [5] 0 0
Neurodegenerative Disorders Research - Perth
Recruitment postcode(s) [1] 0 0
2145 - Sydney
Recruitment postcode(s) [2] 0 0
3800 - Clayton
Recruitment postcode(s) [3] 0 0
3101 - Melbourne
Recruitment postcode(s) [4] 0 0
3050 - Parkville
Recruitment postcode(s) [5] 0 0
6008 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Tennessee
Country [12] 0 0
United States of America
State/province [12] 0 0
Washington

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Prana Biotechnology Limited
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ray Dorsey
Address 0 0
Johns Hopkins University
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT01590888