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Symptomatic Relief

Huntington Society of Canada and Huntington Study Group
Request for Applications

Required letter of intent due:  February 1, 2010
Deadline for full proposals:  March 23, 2010

1. Goal

In keeping with the principles of evidence based medicine and the many unmet clinical needs of patients living with HD, the Huntington Society of Canada and Huntington Study Group are interested in developing therapies in the near term that offer relief to individuals living with Huntington disease today – while essential basic science and translational research proceed to develop therapies that will favorably modify the underlying disease.

The goal of this project is to test experimental interventions that show promise of providing demonstrable symptomatic benefit for the cognitive (intellectual), motor, behavioral or weight maintenance problems facing patients with manifest HD. As a byproduct of these experiments, it is understood that investigators may help to refine scales or tools to measure symptoms of HD. It is anticipated that such research might provoke interest among commercial entities to further develop the treatment for HD

2. Award and Proposal

The Huntington Society of Canada is making available up to $150,000 Canadian each for meritorious research projects to be funded by June 30 2010 and completed by October 2011 (i.e. 16 months). The award is intended to support projects involving a controlled clinical study of a promising experimental intervention that meets the above-mentioned goals.

Proposals that address the following symptoms will be given highest priority (listed in rough rank order):

1. Irritability and Aggression
2. Depression
3. Obsessive Compulsive Symptoms/Perseveration
4. Apathy
5. Sleep Disorder
6. Bradykinesia/Rigidity   
7. Dysphagia

The proposed controlled study should test an experimental treatment (pharmaco-therapeutic, cognitive or behavioral intervention) that has known safety and tolerability in humans and may already be approved for another indication or used by clinicians (off label) with little or no evidence to treat symptoms of HD.  The study should be controlled by involving randomized assignment to experimental treatments, and preferably (although not required) include active and placebo-controlled treatments. The randomized  trial should last no more than 8 to 12 weeks per participant and involve no fewer than 16 ambulatory HD patients who consent to research participation under the oversight of an institutional (ethics) review board and appropriate regulatory review by Health Canada or the FDA.  

Proposals are requested from investigators in Canada, the United States, Australia or Europe who will propose the compound or intervention to be tested, the design and conduct of the clinical trial, and the pre-specified methods of analyses.  It is expected that a report of the study will be prepared for peer-reviewed publication within 90 days of completing the final research visit. The successful applicant should also demonstrate experience in conducting clinical research and the ability to recruit sufficient numbers of subjects for the required sample size.  

If a pharmaco-therapeutic agent is being evaluated, applicants must assure a sufficient supply of active drug (and matching placebo as required) to complete the study.  The supplier/sponsor of the active drug may supplement the amount of the award within the framework of this request for applications.

3. Required Letter of Intent (Due:  February 1, 2010)

Individuals are asked to obtain feedback prior to the submission of a formal application by submitting a letter of intent by Feb. 1, 2010.  The letter, not to exceed two pages, should contain an adequate description of the planned research (see below), including information about the proposed experimental treatment and, as appropriate, the source(s) for its procurement.  An NIH biosketch of the investigator should be attached, as well as a single page to include a brief budget outlining the preparation and implementation phases of the proposal with justification, key references, and any additional relevant information.  Application letters should be sent as a Microsoft Word attachment to an email to HSGGrant2010@ctcc.rochester.edu that includes the candidate’s name, address, professional title, institution and the title of the study.  Letters of intent will be reviewed by the HSG Clinical Trials Initiative Working Group and applicants will be sent a written response by March 1, 2009, that includes a brief analysis of strengths and weaknesses and recommendations for the full proposal.

4.  Full Proposals (Deadline: March 23, 2010)

Full proposals require the following information:

a. Candidates must propose a controlled clinical research plan that is designed to evaluate an experimental treatment that would improve the cognitive (intellectual), motor, behavioral or weight maintenance problems facing patients with manifest HD. 
   
This research plan does not need to be hypothesis-based but must include specific, measurable objectives; the rationale for the study; significance of the research; research design and methods; sample size and statistical analysis; logistical considerations, source of experimental treatment (drug and placebo supply, if required); project timetable and critical milestones; and assurances of timely institutional (ethics) and regulatory review - not to exceed 5 pages. IRB approval is not required at the time of the application.  The application should clearly define the unmet clinical need and anticipated clinical impact of the experimental treatment on that need.

b.   Description of the standardized and validated properties of the intervention or the pharmacological properties and summary of preliminary and background studies.  If applicable, provide the source and availability of the proposed experimental pharmacotherapeutic agent; and a description of CNS access and activity.

c. Information about statistical power, including sample size, estimated effect size and variance

d. Literature cited.

e. NIH biographical sketch for all investigators.

f. Amount of funding requested and time period, including proposed budget for the prepatory and implementation phases of the study. The budget for the proposed study may include the cost of study drug. Overhead or institutional support cannot exceed 10% of the total budget request.

g. Copy of the proposed consent form.  An optional one-page letter to address concerns pertaining to protection of human subjects and safety may also be appended.

h. An electronic copy of the proposal in Microsoft Word must be received on or before March 23, 2010.  Applications and supporting materials should be sent by email to HSGGrant2010@ctcc.rochester.edu. Materials that cannot be sent electronically may be forwarded by mail to:  Suzanne Nicholl, Huntington Study Group, 1351 Mt. Hope Avenue, Suite 223, Rochester, NY 14620.  All materials must be received on or before the March 23, 2010 deadline.  Incomplete applications will not be considered. 

5. The Review Process

Full proposals will be reviewed by a Committee comprising representatives of the Huntington Society of Canada and the Scientific Review Committee of the Huntington Study Group. Committee members will be scientists, clinical investigators, members of the HD community and individuals with business backgrounds and consumer experience.   This Committee will also meet as required to monitor the progress of the study in line with the milestones established when the grant was awarded.

Members of the Scientific Review Committee who are applying for the award, or who have an institutional or professional relationship with applicants, that would represent a conflict of interest, will be excused from the review process.

6. Decision Criteria

The criteria for reviewing proposals will include the following:

a. The extent to which the proposal meets all elements of the goal of the project, namely to test experimental treatments that show promise in the near term of providing demonstrable symptomatic benefit  for the cognitive (intellectual), motor, behavioral or weight maintenance problems facing patients with manifest HD.  It is anticipated that such research might provoke interest among commercial entities to further develop the treatment for HD. 

b. The extent to which the proposed symptom and the objectives of the study are clear and achievable.

c. The extent to which expected outcomes can be adequately measured by way of a validated scale or instrument.

d. The qualifications and experience of the applicant and the overall quality of the proposal, including the scientific merit, research design, sample size, etc.

e. If applicable, the extent to which the preparatory work has been done to ensure access to quality supplies of the proposed compound.

f. The extent to which the logistical elements of the proposal can be successfully implemented in the time allowed.

7. Timelines

Letters of intent must be received electronically by February 1, 2010.  A constructive critique will be sent to the applicant by March 1, 2010.  All proposals are due by March 23, 2010.  The Huntington Study Group will strive to notify successful applicants by June 30, 2010.

8. Enquiries

Questions regarding the Request for Proposals or any of the above information should be directed to Suzanne Robertson at Suzanne.Robertson@ctcc.rochester.edu.

Click here for a PDF copy of the Request for Proposals