NIH Protocol RegistrationPrincipal Investigator, MD, PhD
Title of Study
NIH Registration of a Human Gene Transfer Protocol
Title of Protocol
Principal Investigator, MD, PhD
X Professor, Department
Duke University Medical Center
Date of Submission
Table of Contents
Table of Contents
1.Scientific Abstract
2.Proposed Clinical Protocol
3.Summary of Preclinical Studies
4.Description of the Product
4a. Derivation of the Delivery Vector System and Modifications
4b. Genetic Content
4c. Other Materials
4d. Methods for Replication-Competent Virus Testing
4e. Intended Target Cells and Transduction Efficiency
4f. Gene Transfer Agent Delivery Method
5.Proposed Informed Consent Document
6.Documentation from the IBC/IRB
1.Scientific Abstract
2.Proposed Clinical Protocol
Include a copy of the proposed clinical protocol, including tables, figures, and any relevant publications. The protocol does not need to be incorporated into this document, but you may simply state “See Attached.”
3.Summary of Preclinical Studies
A summary of preclinical studies conducted in support of the proposed clinical trial or reference to the specific section of the protocol providing this information.
4.Description of the Product
4a. Derivation of the Delivery Vector System and Modifications
Describe the derivation of the delivery vector system including the source (e.g., viral, bacterial, or plasmid vector); and modifications (e.g., deletions to attenuate or self-inactivate, encapsulation in any synthetic complex, changes to tropisms, etc.). Please reference any previous clinical experience with this vector or similar vectors.
4b. Genetic Content
Describe the genetic content of the transgene or nucleic acid delivered including the species source of the sequence and whether any modifications have been made (e.g. mutations, deletions, and truncations). What are the regulatory elements contained in the construct?
4c.Other Materials
Describe any other material to be used in preparation of the agent (vector and transgene) that will be administered to the human research subject (e.g., helper virus, packaging cell line, carrier particles).
4d. Methods for Replication-Competent Virus Testing
Describe the methods for replication-competent virus testing, if applicable.
4e. Intended Target Cells and Transduction Efficiency
Describe the intended ex vivo or in vivo target cells and transduction efficiency.
4f. Gene Transfer Agent Delivery Method
Describe the gene transfer agent delivery method.
5.Proposed Informed Consent Document
Include a copy of the proposed informed consent document. The informed consent does not need to be incorporated into this document, but you may simply state “See Attached.”
6.Documentation from the IBC/IRB
Specifically for submission to the NIH Office of Science Policy (OSP), provide additional documentation originating from oversight bodies (IBC or IRB) involved in the review at an initial site regarding their assessment of whether public RAC review is warranted. In the event that review is requested, a justification that the NIH RAC review criteria (see Section III–C– 1) are met shall be included.
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