Download Biosafety Application, Human Gene Transfer Clinical Trials

UCDenver Biosafety Application
Human Gene Transfer Clinical Trials
IBC Application Number:
-HG
Please return the completed form and the required supporting documentation electronically to
[email protected]
Documents to Submit:
 A scientific abstract
 The proposed Clinical Protocol, including
tables, figures, and any relevant
publications
 The proposed Investigator’s Brochure with
any appendices
 Summary of preclinical studies conducted
in support of the proposed clinical trial or
reference to the specific section of the
protocol providing this information.




The proposed informed consent
document
Letter or certification of registration
with the NIH (GEMCRIS)
CV of PI(s) in NIH bio-sketch format
IBC HGT Protocol Review Form
(Description of the product)
Section I. Administrative Information
FDA IND Number:
PRINCIPAL INVESTIGATOR:
SPONSOR/GRANT AGENCY:
DEPARTMENT/DIVISION:
PHONE NO: 303-724-5541
SITE(S) WHERE RESEARCH DRUG
PREPARATION AND ADMINISTRATION WILL
BE DONE:
Mailstop:
Email:
CO-INVESTIGATORS:
Drug Preparation
Building (s):
OTHER PERSONNEL (List those involved in
THIS clinical trial):
Drug Administration
Building (s):
Room No. (s):
PROJECT TITLE:
Other Committee Reviews, if applicable:
Room No. (s):
UCDenver Application for Human Gene Transfer Clinical Trials—BSF-003, Rev July 2016
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UCDenver Biosafety Application
Human Gene Transfer Clinical Trials
I understand that for any experiment involving the deliberate transfer of recombinant DNA, or
DNA or RNA derived from recombinant DNA, into human research participants (Human Gene
Transfer), no research participant may be enrolled, until the entire review and approval process
has been completed in accordance with all CUDenver Institutional Biosafety Committee and
COMIRB requirements and stipulations.
I acknowledge that I am aware of all the requirements and restrictions of the most current
NIH Guidelines for the Human Gene Transfer Clinical trial to be conducted. I accept
responsibility for the safe conduct of the clinical trial to be conducted at the CUDenver |
Anschutz Medical Campus.
I understand that it is my responsibility to assure that all personnel working on this clinical trial
are informed of and trained about any of the potential hazards of the recombinant DNA or gene
transfer material, the proper actions for safe use, the appropriate steps to take in case of
accidents, spills or exposures and that they are provided with all necessary safety
equipment/personal protective equipment and are instructed in its use.
_________________ _______________________________________________
Date
Signature of Principal Investigator
PLEASE FILL OUT THE REST OF THIS FORM BY ANSWERING ALL SECTIONS
APPLICABLE TO THIS PROJECT AT UCDenver.
Please attach additional page(s) if necessary. The responses MUST be specific for clinical
trials at CUDenver. You may request assistance from the study sponsor for the appropriate
responses to these questions.
UCDenver Application for Human Gene Transfer Clinical Trials—BSF-003, Rev August, 2016
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UCDenver Biosafety Application
Human Gene Transfer Clinical Trials
Section II.
Human gene transfer experiments that may require NIH RAC review in addition to the
local IBC review:
Check any and all that apply to this protocol:
______ The protocol uses a new vector, genetic material, or delivery methodology that represents a firstin-human experience, thus presenting an unknown risk;
_____ The protocol relies on preclinical safety data that were obtained using a new preclinical model
system of unknown and unconfirmed value;
_____ The proposed vector, gene construct, or method of delivery is associated with possible toxicities
that are not widely known and that may render it difficult for oversight bodies (i.e. IBC, IRB) involved in
the review at an initial site(s) to evaluate the protocol rigorously
If any of these conditions may apply to the protocol, the IBC or IRB may request that the NIH RAC
(Recombinant DNA Advisory Committee) conduct a public RAC review. If this is requested, it must occur
before the local IBC or IRB proceeds with the review/approval of the protocol.
All HGT clinical trials MUST register with the Office of Science Policy/Office of
Biotechnology Activities in the Genetic Modification Clinical Research Information
System (GEMCRIS) (http://osp.od.nih.gov/office-biotechnology-activities/biomedicaltechnology-assessment/hgt/gemcris ) and submit documentation of this registration to
the local IBC before the review commences.
UCDenver Application for Human Gene Transfer Clinical Trials—BSF-003, Rev August, 2016
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UCDenver Biosafety Application
Human Gene Transfer Clinical Trials
Section III. A description of the product: Appendix M-1-A
1. A description of the product: Appendix M-1-A
a. Describe the derivation of the delivery vector system including the source (e.g., viral,
bacterial, or plasmid vector); give specifics (i.e. lentiviral vector with tat, env, gag
deleted). Also address the following:
i. Is it replication incompetent?
ii. Describe the methods for replication competent virus testing, if applicable.
b. Describe modifications (e.g., deletions to attenuate or self-inactivate, encapsulation in
any synthetic complex, changes to tropisms, etc.).
c. Include references to any previous clinical experience with this vector or similar vectors.
d. Describe the genetic content of the transgene or nucleic acid delivered
i.
Including the species source of the sequence
ii.
Whether any modifications have been made (e.g. mutations, deletions, and
truncations).
iii.
What are the regulatory elements contained in the construct?
iv.
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)
e. Indicate whether the intended target cells are ex vivo or in vivo
a. Describe the intended target cells and transduction efficiency.
b. If ex vivo, where will this be done (i.e. sponsor’s facility at “xxxxxxx”, PI’s
laboratory in research tower “yyyyyy”)
f. Describe the gene transfer agent delivery method and location (i.e. method of
administration: intra-tumor injection during surgery, hepatic artery with an infusion
pump, out-patient clinic at “zzzzz” hospital, etc.)
g. Will shedding studies be conducted? Is any vector shedding expected?
h. What measures will be undertaken to mitigate the risks, if any, of contamination by or
dissemination of the vector to other patients, health-care workers, family members or
the environment (facility, home or community)? (i.e. disinfection procedures, restrictions
on demographics of family such as no infants younger than 6 months or
immunocompromised persons in the home)
i. Is there a risk of vertical transmission to offspring?
UCDenver Application for Human Gene Transfer Clinical Trials—BSF-003, Rev August, 2016
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UCDenver Biosafety Application
Human Gene Transfer Clinical Trials
a. Will pregnancy tests be performed?
b. Will birth control measures be recommended to subjects?
i. For what period of time?
j. List any biological safety cabinet to be used in the preparation of the study drug, with:
location:
If in university laboratory or UCH pharmacy: serial number ______________
Date of its most recent certification___________________
k. What specific protocols are to be followed for the receipt, storage, preparation and
disposal of study drug?
l. Explain how safety reporting for all Adverse Events or Serious Adverse Events is to be
handled. Who will be responsible for the notification to NIH and the FDA, COMIRB and
the IBC?
This space is for Institutional Biosafety Committee Use ONLY
IBC Application Number _________________-HG
Application Received by IBC Office
Date: __________________
Reviewer 1
Date: __________________
Reviewer 2
Date: __________________
Full Committee Review
Date: __________________
Approved
COMIRB #
Deferred
Disapproved
Review Panel & Date
__________________
UCDenver Application for Human Gene Transfer Clinical Trials—BSF-003, Rev August, 2016
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