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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01590888
• Date of registration: 18/04/2012
• Prospective Registration: No
• Primary sponsor: Prana Biotechnology Limited
• Public title: Effect of PBT2 in Patients With Early to Mid Stage Huntington Disease Reach2HD
• 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
• Date of first enrolment: April 2012
• Target sample size: 109
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01590888
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Australia, United States

Contacts

• Name: Ray Dorsey
• Affiliation: Johns Hopkins University

Key inclusion & exclusion 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.



Age minimum: 25 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Huntington Disease

Intervention(s)

Drug: PBT2

Drug: Placebo

Primary Outcome(s)

Safety and Tolerability of PBT2 in Patients With HD [Time Frame: Baseline to 26 weeks]

Secondary Outcome(s)

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

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

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

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

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

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

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

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

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

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

Secondary ID(s)

PBT2-203

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 18/07/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01590888