MedTrace Logo

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

NCT01590888 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 109

Last updated 2016-07-18

Study results available
· View outcomes & findings →

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 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.

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 a six month 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.

Conditions

  • Huntington Disease

Interventions

DRUG

PBT2

250mg capsules administered orally once per day for 26 weeks

DRUG

PBT2

100mg capsules administered orally once per day for 26 weeks

DRUG

Placebo

Matching capsules administered orally once per day for 26 weeks

Sponsors & Collaborators

  • Prana Biotechnology Limited

    lead INDUSTRY

Principal Investigators

  • Ray Dorsey · Johns Hopkins University

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-04-30
Primary Completion
2013-07-31
Completion
2014-02-28

Countries

  • United States
  • Australia

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01590888 on ClinicalTrials.gov