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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Prana Biotechnology Limited
ClinicalTrials.gov Identifier:
NCT01590888
First received: April 18, 2012
Last updated: June 7, 2016
Last verified: June 2016
Results First Received: November 17, 2015  
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
Condition: Huntington Disease
Interventions: Drug: PBT2
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Twenty sites were initiated for the study, with participants enrolled at 14 sites in the US and 5 sites in Australia.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
PBT2 250mg PBT2: 250mg capsules administered orally once per day for 26 weeks
PBT2 100mg PBT2: 100mg capsules administered orally once per day for 26 weeks
Sugar Pill Placebo: Matching capsules administered orally once per day for 26 weeks

Participant Flow:   Overall Study
    PBT2 250mg     PBT2 100mg     Sugar Pill  
STARTED     36     38     35  
COMPLETED     32     38     34  
NOT COMPLETED     4     0     1  
Adverse Event                 3                 0                 1  
Lost to Follow-up                 1                 0                 0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
PBT2 250mg PBT2: 250mg capsules administered orally once per day for 26 weeks
PBT2 100mg PBT2: 100mg capsules administered orally once per day for 26 weeks
Sugar Pill Placebo: Matching capsules administered orally once per day for 26 weeks
Total Total of all reporting groups

Baseline Measures
    PBT2 250mg     PBT2 100mg     Sugar Pill     Total  
Number of Participants  
[units: participants]
  36     38     35     109  
Age  
[units: years]
Mean (Standard Deviation)
  50.3  (10.4)     54.1  (12.1)     51.2  (10.4)     51.9  (11.0)  
Gender  
[units: participants]
       
Female     17     19     19     55  
Male     19     19     16     54  
Ethnicity (NIH/OMB)  
[units: participants]
       
Hispanic or Latino     2     0     0     2  
Not Hispanic or Latino     34     38     35     107  
Unknown or Not Reported     0     0     0     0  
Race (NIH/OMB)  
[units: participants]
       
American Indian or Alaska Native     0     0     0     0  
Asian     0     0     0     0  
Native Hawaiian or Other Pacific Islander     0     0     0     0  
Black or African American     0     0     0     0  
White     35     38     35     108  
More than one race     0     0     0     0  
Unknown or Not Reported     1     0     0     1  
Region of Enrollment  
[units: participants]
       
United States     26     28     25     79  
Australia     10     10     10     30  
Body Mass Index  
[units: kg/m^2]
Mean (Standard Deviation)
  25.51  (4.89)     25.40  (4.53)     27.09  (6.03)     25.98  (5.18)  
Total Function Capacity [1]
[units: units on a scale]
Mean (Standard Deviation)
  9.1  (2.2)     9.3  (2.3)     9.3  (1.6)     9.2  (2.0)  
CAG Larger Allele Repeat Length [2]
[units: CAG repeats]
Mean (Standard Deviation)
  44.4  (4.6)     43.2  (2.8)     44.1  (3.9)     43.9  (3.8)  
CAG Smaller Allele Repeat Length [3]
[units: CAG repeats]
Mean (Standard Deviation)
  18.8  (4.4)     18.5  (3.0)     20.0  (4.8)     19.1  (4.1)  
Disease Burden [4]
[units: CAG repeats*years]
Mean (Standard Deviation)
  411.68  (104.96)     392.54  (96.10)     410.07  (110.02)     404.49  (103.06)  
[1] Total function capacity (TFC) is a scale measuring the participants capacity to perform 5 areas - their capacity to work, capacity to handle financial affairs, capacity to manage domestic responsibilities, capacity to perform activities of daily living and level of care required (home or care facility). The scale used is a TOTAL of each of the 5 areas, which range from 0 (unable to perform or require full care) to either 2 or 3 (normal, no assistance required). The TFC can scored from 0 to 13. The lower the score, the more severe the disease state of Huntington disease is.
[2] Huntington disease (HD) is a dominant genetic disorder. Mutations in the HTT gene cause HD. The HTT mutation involves a DNA segment known as a Cytosine, Adenine and Guanine (CAG) trinucleotide repeat, a series of three DNA building blocks. Normally, the CAG segment is repeated 10 to 35 times within the gene. In people with HD, the CAG segment is repeated more than 36 times with higher repeat numbers normally indicating earlier onset of the disease and increased severity of symptoms.
[3]

Huntington disease (HD) is a dominant genetic disorder. Mutations in the HTT gene cause HD. The HTT mutation involves a DNA segment known as a Cytosine, Adenine and Guanine (CAG) trinucleotide repeat, a series of three DNA building blocks. Normally, the CAG segment is repeated 10 to 35 times within the gene. In people with HD, the CAG segment is repeated more than 36 times with higher repeat numbers normally indicating earlier onset of the disease and increased severity of symptoms.

HD is a dominant genetic disorder.

[4] Disease burden in patients with Huntington disease is calculated by (CAG repeat length - 35) x Age at Baseline, with the higher score indicating a greater Disease Burden for a patient.



  Outcome Measures
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1.  Primary:   Safety and Tolerability of PBT2 in Patients With HD   [ Time Frame: Baseline to 26 weeks ]

2.  Secondary:   Change From Baseline in Cognitive Test Battery - Composite z Scores   [ Time Frame: Baseline to 26 weeks ]

3.  Secondary:   Change From Baseline in Motor Function   [ Time Frame: Baseline to 26 weeks ]

4.  Secondary:   Change From Baseline in Functional Abilities   [ Time Frame: Baseline to 26 weeks ]

5.  Secondary:   Change From Baseline in Behaviour   [ Time Frame: Baseline to 26 weeks ]

6.  Secondary:   Change From Baseline in Investigator Global Assessments by Efficacy Index   [ Time Frame: Baseline to 26 weeks ]

7.  Secondary:   Change From Baseline in Blood Biomarkers   [ Time Frame: Baseline to 26 weeks ]

8.  Secondary:   Change From Baseline in Brain Function (MRI)   [ Time Frame: Baseline to 26 weeks ]

9.  Secondary:   Change From Baseline in Blood Biomarkers   [ Time Frame: Baseline to 26 weeks ]

10.  Secondary:   Change From Baseline in Blood Biomarkers - Selenium   [ Time Frame: Baseline to 26 weeks ]

11.  Secondary:   Change From Baseline in Urine Biomarkers   [ Time Frame: Baseline to 26 weeks ]

12.  Secondary:   Change From Baseline in Brain Function (MRI)   [ Time Frame: Baseline to 26 weeks ]

13.  Secondary:   Change From Baseline in Cognitive Test Battery - TMT Part B   [ Time Frame: Baseline to 26 weeks ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr Caroline Herd
Organization: Prana Biotechnology
phone: +61393494906
e-mail: info@pranabio.com


Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Prana Biotechnology Limited
ClinicalTrials.gov Identifier: NCT01590888     History of Changes
Other Study ID Numbers: PBT2-203
Study First Received: April 18, 2012
Results First Received: November 17, 2015
Last Updated: June 7, 2016
Health Authority: United States: Food and Drug Administration
Australia: Department of Health and Ageing Therapeutic Goods Administration