Monday, April 20, 2009
 
 

     

Huntington Project Web Site Closing May 1, 2009

Dear Huntington Project Web Site Visitors,

The Huntington Project was created in 2003 with specific aims in mind:

  1. increase communication within the community
  2. facilitate opportunities for clinical research (COHORT)
  3. provide a forum to evaluate research initiatives (SET-HD)

Happily, the mechanisms for sustaining these processes are in place and are now operating independently. Therefore, we will close the Huntington Project web site in early May 2009.

Thank you for your support and interest.

Sincerely,
(on behalf of the Huntington Project Communications Group)

Selection Criteria
 

Operational criteria for potential systemically
or locally delivered drugs for HD
     

CRITERIA

OPERATIONAL DEFINITION
Scientific Rationale

1) Consistency of pre-clinical data in different settings and HD animal models (e,g, fragment and full length)

2) Credible mechanism/target relevant to HD although exact mechanism may be unclear
Kinetics and Blood-brain barrier (BBB) penetration

Evidence by direct experiments or inference that BBB penetration occurs and that adequate concentration of agent can be achieved with the intended route of administration. Extrapolations from animal model to human dose should be explicitly stated.
Safety and Tolerability (including drug interactions)

Phase I study: adequate pre-clinical toxicology data to merit an Investigational New Drug Application (IND).

 

Phase II/III study: Preliminary data show agent is safe and tolerable in humans in the dose and route of administration needed for the proposed effect (at least phase I data). No further safety data required before use in HD clinical trials.

Efficacy

1) demonstrated in relevant animal models of disease

 

or

 

 

2) indicated  in human clinical studies

1) Animal: Two or more independent studies demonstrate efficacy in rodent  models. Efficacy indicated by increase in survival and/or by behavioral changes and supported by changes in pathology. Evidence of dose response is supportive.

 

 

 

 

2) Human: Evidence from previous trials or observational data that is suggestive of a beneficial effect. Observational data should at least approximate criteria for causal inference.
Copyright 2003,2004 by Huntington Project