Concept Proposal Template for NIDCD Clinical Trials
(For phase I trials, skip those items which do not apply.)
1) List the title of the proposed research, the Principal Investigator(s) and institution(s).
2) If prior clinical trial experience is minimal, appoint clinical trial expert(s) as key personnel to help with study design and execution.
3) Appoint a biostatistician – preferably with clinical trial experience -- as key personnel.
4) Describe the target disease, primary study objective and any secondary study objectives.
1) Briefly describe the scientific rationale for the study.  State the compelling public health need and the trial’s potential impact on medical care.
5) Briefly describe the study design and indicate, in general terms, how the design will fulfill the intent of the study.  For a preliminary phase I/II clinical trial, describe how it will be used to formulate a definitive phase III clinical trial.
6) R01 and R34 applications require compelling preliminary data.  Are preliminary data adequate to use one of these mechanisms?
2) Describe the informed consent process and human subjects protections.  If risks are more than minimal, identify an independent medical officer to manage adverse events.  Include an adverse event plan (see attached NIDCD guidelines). 
7) Describe ethical considerations of the proposed research.  For example, placebo-controlled trials may be unethical if a standard-of-care exists.
8) Describe the number of subjects to be enrolled, and the number of clinical centers needed to meet target enrollment.
9) List specific inclusion and exclusion criteria.  How will these criteria impact on subject recruitment and retention?  Describe the method for identifying and recruiting subjects.
10) Describe the statistical and clinical basis for the sample size calculation.
11) Describe the extent and type of blinding/masking.  Persons measuring the primary outcome should be masked.
12) Describe the randomization procedure.
13) Describe how the intervention will be administered, including dose and duration as applicable.
14) How will fidelity checks be used to insure that each subject receives the correct treatment?
15) Define the primary and secondary outcomes and how they will be measured.  Only one primary outcome measure is permitted; all other measurements are observational.
16) List the statistical methods to be used to analyze these outcomes.  Specify whether an intention-to-treat analysis will be performed.  Explain how missing data, outliers, noncompliance and losses to follow-up will be handled in the analyses.
17) Describe how data will be created, handled and stored.  How will data integrity and confidentiality at individual sites be confirmed?  Double-blind trials require an independent data coordinating center.
18) For phase II and III trials, describe the interim monitoring plan, including the schedule of interim analyses and guidelines for stopping the study for reasons of efficacy, safety, futility, or poor study performance.
19) Do not underestimate trial duration.  Allow ample time for start-up, planned enrollment, follow-up to endpoint, and close-out.
20) Do not underestimate trial cost.  For phase II and III trials, budget for a clinical coordinating center, data coordinating center, and Data Safety Monitoring Board, as needed.
21) For a new drug or device, try to obtain an IND, IDE or written exemption from the FDA before submitting the application.  
22) Name the participating pharmaceutical or device manufacturing company, if any.
23) Do you or any member of your group have a financial conflict of interest or hold a patent with the use of the intervention on this protocol?
24) For phase II and III trials, have you considered collaborating with one of the NIDCD clinical research networks such as CHEER?
25) Estimate direct costs for all years of funding.
26) Indicate a planned submission date.

For additional information contact:

Gordon B. Hughes, M.D.
Director, Clinical Trials, Division of Scientific Programs
National Institute on Deafness and Other Communication Disorders/NIH
6120 Executive Boulevard
Room 400C MSC 7180
Bethesda, MD 20892
Phone 301-435-4085
Fax 301-402-6251
Email hughesg@nidcd.nih.gov