GTRP - Instructions for Submitting a Request For Service Application (RSA)

Who can submit an RSA?

Investigators may submit an RSA after:

Completing the online Investigator Registration application form on the NHLBI Gene Therapy Resource Program (GTRP) main webpage (http://www.gtrp.org) and
Receiving registration approval by the NHLBI Gene Therapy Group (GTG) allowing him/her to submit an RSA to the GTRP. For a registration to be considered for approval, an investigator must be conducting gene therapy research at an institution located in the United States.

What are the requirements for the various types of RSAs?

For all types of RSAs for laboratory services

The scientific research in gene therapy that is the subject of the RSA must be consistent with the NHLBI Mission (http://www.nhlbi.nih.gov/about/org/mission.htm) or, if the research addresses a disease area consistent with the mission of another NIH Institute, that Institute must agree to cover the cost of the service.
The investigator must complete the on-line Registration Form on the GTRP main web page and receive approval by the NHLBI Gene Therapy Group (GTG) allowing the investigator to submit an RSA.
RSA approval and implementation is contingent upon availability of funds in the GTRP.
Services provided through an approved RSA are at no cost to the investigator.
The various types of RSAs are independent of one another. An applicant Investigator may submit any type of RSA for a qualified gene therapy research project regardless of past or future utilization of GTRP resources for the project.

In Addition

For Preclinical Vector Production

The applicant must be working in basic or translational gene therapy research in a disease area funded by the NIH.
Funding must already be secured, and demonstrated in writing, for the project for which the vector is requested.
View University of Pennsylvania, NHLBI GTRP Core Vector Laboratory webpage for further information. (lab_penn.aspx).

For Pharmacology/Toxicology Studies

The applicant must provide documentation of a pre-IND FDA meeting/review and FDA-specified pharmacology/toxicology testing recommendations.
A brief description of the anticipated gene therapy clinical trial must be provided by the applicant.
View Lovelace Biomedical and Environmental Research Institute, the NHLBI GTRP Core Pharmacology/ Toxicology Laboratory webpage for further information (lab_lovelace.aspx).

For Clinical-Grade AAV or Lentiviral Vector Production

The applicant must have adequate preclinical data and the requisite regulatory and other oversight reviews for the proposed clinical protocol in which the clinical-grade vector will be used.
Funding must already be secured and documented for the proposed clinical trial.
View the Children’s Hospital of Philadelphia, NHLBI GTRP Core AAV Clinical-grade Vector Laboratory webpage (lab_aav.aspx) or the Indiana University, NHLBI GTRP Core Lentiviral Clinical-grade Vector Laboratory webpage (lab_ind.aspx) for further information.

For Regulatory Support

The applicant must be, or have been, an NHLBI grantee. However, the gene therapy project for which the regulatory assistance is requested does not have to be part of an NHLBI grant.
View the NHLBI GTRP Clinical Coordinating Center webpage for further information (ccc.aspx).

For Phase I or II Clinical Trial Funding Assistance

The applicant must:
- Be an NHLBI investigator
- Provide the clinical protocol for which funding is sought
- Provide an implementation plan for recruitment and enrollment of research participants
- Provide documentation of any secured financial support for the clinical trial
View the NHLBI GTRP Clinical Coordinating Center webpage for further information (ccc.aspx)

What is the RSA Submission Process?

Once approved as an Applicant Investigator by the NHLBI Gene Therapy Group (GTG) you will be notified by the Clinical Coordinating Center (CCC). You will use the User Name (email address) and password which you created during the Registration Application process to access the secure area of
the website.
To access the RSA forms and other information available to Approved Investigators, click on the “Request for Service Applications” menu item located near the top of the GTRP Home page. The initial information displayed is available to the public. Near the bottom of the page, click on Member Login. Use your User Name and password to Login and view the Investigator’s Main Page.

Select the type of GTRP service you are interested in. You will be asked to submit information specific to the service being requested. From the Main GTRP webpage, you can access the webpage(s) for each of the service areas to determine the type of information that is required on the RSA form. Go to the FAQ section of the specific service area webpage and click on “What type of information will an approved applicant investigator need to provide to the laboratory as part of the Request for Service Application?”

You will be allowed to enter information and save your RSA in stages; you will initially submit the RSA as a “preliminary” submission.

Each RSA will be assigned a unique identifier at the time of the initial “save”. Refer to this number in all correspondence or submission of supplemental forms and materials.

You will be able to edit “preliminary” submissions.

Following your “preliminary” submission you must discuss your request with the respective laboratory or the clinical coordinating center (if applicable) before submitting it as a “final” RSA. Only you, as the RSA author, will have edit rights to your preliminary RSA. If needed, you may submit multiple “preliminary” versions of the RSA before submitting a “final” version.

Once submitted as a “final” the RSA can not be edited.

Once the RSA is submitted as “final” it enters a queue for review and subsequent actions. The review process is described in the document “The Request for Service Application Review Process”.

Before your RSA can receive program review, you must submit the following additional forms to the clinical coordinating center via email. These forms can be downloaded from the RSA page.
- Investigator and Institution Certifications and Agreement (for all vector production RSAs and the Pharmacology/Toxicology RSA)
- Conflict of Interest Statement for Investigators (for all investigators submitting an RSA)

Any additions, changes, or clarifications to a “final” RSA that may be required following any step in the review process, must be submitted as an “Amendment”.

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Supplemental information for preclinical RSA submissions

Guidance for Investigators Submitting a Preclinical RSA Form

Describe the particular research study for which vectors are being requested in the following fields of the RSA form:

Study Title
Study Abstract
Rationale for Conducting the Study
Study Design

In Section V of the RSA form, the investigator can request multiple vector serotypes, promoters, and genes by clicking on the button “Save and Add More Vectors” at the bottom of the page. Information on each of the vectors needed for the study, including controls, must be provided in the designated data fields - not just described in the text fields.

The text describing the study, rationale and design should correlate with the vectors requested in the discreet vector fields. This frequently requires modification after the investigator’s discussions with the Core Laboratory prior to submitting a “final” RSA.

Make every effort to estimate the quantity required for each vector and control to fully complete the study.

When to Submit Multiple RSAs

A particular research study may be part of a broad multi-year and/or multi-step research plan. A broad research plan should not be submitted in a single RSA but may require submission of multiple RSAs to address discrete study questions.

The following factors would require submission of more than one RSA:

Sequential study questions: If a research question addressed in one study needs to be answered before a second study can be started, then a separate RSA needs to be submitted for the second study after completion of the first study.
Multiple Aims: If a research plan has discrete aims using different vectors, animal models, modes of deliver, study design, and/or study questions, then separate RSAs need to be submitted addressing each aim.
Different animal models or test systemsIf a vector is to be tested in different animal models or test systems to answer different questions then separate RSAs are required (e.g. vector “A” used in rats to test gene function and used in dogs to test safety).
Vector comparisons: Comparisons of different vector types, such as a Lentivirus and an AAV vector, require separate RSAs. In addition, a new RSA would be required to continue a study using different vector serotypes than what was requested in the original RSA submission.

When to Use the Amendment/Supplement Form

The Amendment/Supplement Form is available for investigators to use under the following circumstances:

To upload documents containing information that is an adjunct to the RSA data fields or information requested by the Core Laboratory during the preliminary RSA submission process.
To request additional quantity of vector already provided in the original “final” RSA if warranted to complete a study but not to address a new question or hypothesis.

Note: The Amendment/Supplement form should not be used to request different vectors or controls for the original study. A new RSA would be required.

Because the information submitted through the Amendment/Supplement Form is not captured in the GTRP database, this form should be used sparingly.