MDA Translational
Research Program
Resources
(bookmarks to major headings)
FDA Investigational
New Drug Application Resources
CDER
Investigational New Drug Applications
http://www.fda.gov/cder/regulatory/applications/ind_page_1.htm
IND Drug Review Process (flowchart)
http://www.fda.gov/cder/handbook/ind.htm
Guidance for Industry Q & A - Content and Format
of INDs for Phase 1 Studies of Drugs, Including Well-Characterized,
Therapeutic, Biotechnology-Derived Products
http://www.fda.gov/cder/guidance/3591fnl.htm
Office of Drug Evaluation IV (ODE IV) Pre-Investigational
New Drug Application (IND) Consultation Program
http://www.fda.gov/cder/ode4/preind/default.htm
CBER (blood, vaccines, cellular/gene
therapy, tissue, devices)
Investigational New Drug Applications
http://www.fda.gov/cber/ind/ind.htm
CBER Frequently Asked Questions
http://www.fda.gov/cber/faq.htm
Guidance Documents
http://www.fda.gov/cber/guidelines.htm
FDA: GENERAL RESOURCES
REGULATORY
Office of Orphan Products Development
http://www.fda.gov/orphan/
Manufacturers Assistance and Technical Training Branch
(MATTB)
The Manufacturers Assistance and Technical Training Branch
(MATTB) informs industry and trade associations of the
status of CBER policies and initiatives through regular
information dissemination and training. MATTB also serves
as the CBER focal point for industry and trade associations
to provide meeting support, and coordinates external meetings
with other FDA Centers.
http://www.fda.gov/cber/manufacturer.htm
Draft Guidance for Industry: Information Program on Clinical
Trials for Serious or Life-Threatening Diseases and Conditions
(Revision 1) - 1/26/2004 - (PDF),
(Text)
Guidance for Industry: Fast Track Drug Development Programs
- Designation, Development, and Application Review - 7/21/2004
- (PDF),
(Text)
Appendix 2 (PDF)
Appendix 3 - CDER
MAPP 6020.3 (PDF), CBER
SOPP 8405(Text)
Appendix 4 (PDF)
Draft Guidance for FDA Review Staff and Sponsors: Content
and Review of Chemistry, Manufacturing, and Control (CMC)
Information for Human Gene Therapy Investigational New
Drug Applications (INDs) - 11/8/2004 - (PDF),
(Text)
Draft Guidance for Reviewers: Instructions and Template
for Chemistry, Manufacturing, and Control (CMC) Reviewers
of Human Somatic Cell Therapy Investigational New Drug
Applications (INDs) - 8/15/2003 - (PDF),
(Text)
Draft Guidance for Clinical Trial Sponsors on the Establishment
and Operation of Clinical Trial Data Monitoring Committees
- 11/15/2001 - (PDF),
(Text)
Guidance for Human Somatic Cell Therapy and Gene Therapy
http://www.fda.gov/cber/gdlns/somgene.pdf
- 1998
Guidance for Industry: Supplemental Guidance on Testing
for Replication Competent Retrovirus in Retroviral Vector
Based Gene Therapy Products and During Follow-up of Patients
in Clinical Trials Using Retroviral Vectors
http://www.fda.gov/cber/gdlns/retrogt1000.pdf
- 2000
Points to Consider in the Characterization of Cell Lines
Used to Produce Biologicals
http://www.fda.gov/cber/gdlns/ptccell.pdf
Good Clinical Practice in FDA-Regulated Clinical Trials
http://www.fda.gov/oc/gcp/default.htm
FUNDING OPPORTUNITIES
Orphan Products Grant Program
http://www.fda.gov/orphan/grants/index.htm
NIH RESOURCES
NIH CLINICAL TRIAL
RESOURCES
Office of Biotechnology Activities
http://www4.od.nih.gov/oba/
Recombinant DNA and Gene Transfer
http://www4.od.nih.gov/oba/Rdna.htm
RAC – Recombinant DNA Advisory Committee
http://www4.od.nih.gov/oba/rac/aboutrdagt.htm
GeMCRIS – Genetic Modification Clinical Research
Information System
http://www4.od.nih.gov/oba/RAC/GeMCRIS/GeMCRIS.htm
NIH Guidelines for Research Involving Recombinant DNA
Molecules
http://www4.od.nih.gov/oba/rac/guidelines/guidelines.html
Office of Human Subjects Research
http://www.nihtraining.com/ohsrsite/
Bioethics Resources on the Web
http://www.nih.gov/sigs/bioethics/casestudies.html
Overall NINDS Clinical Trials Program Information http://www.ninds.nih.gov/funding/research/clinical_research/index.htm
NINDS Clinical Trial Planning Grant
http://grants2.nih.gov/grants/guide/pa-files/PAR-03-051.html
NINDS Clinical Trial Planning Grant (R34) Program
http://grants.nih.gov/grants/guide/pa-files/PA-04-008.html
Pilot Studies for Clinical Trials in Neurological Disorders
http://grants.nih.gov/grants/guide/pa-files/PAR-03-174.html
NIH TRAINING RESOURCES
Ruth L. Kirschstein National Research Service Awards
for Postdoctoral Fellowships in Muscle Disease Research
http://grants.nih.gov/grants/guide/pa-files/PA-05-052.html
NINDS Mentored Research Scientist Development Awards
in Translational Research
http://grants.nih.gov/grants/guide/pa-files/PAR-02-138.html
Mentored Clinical Investigator Career Development Awards
in Muscle Disease Research
http://grants.nih.gov/grants/guide/pa-files/PA-05-051.html
NIH TRANSLATIONAL RESEARCH
FUNDING OPPORTUNITIES
Overall NINDS Translational Research Program http://www.ninds.nih.gov/funding/research/translational/index.htm
NINDS Exploratory/Developmental Projects in Translational
Research http://grants.nih.gov/grants/guide/pa-files/PAR-02-138.html
NINDS Cooperative Program in Translational Research
http://grants.nih.gov/grants/guide/pa-files/PAR-02-139.html
NINDS Mentored Research Scientist Development Awards
in Translational Research http://grants.nih.gov/grants/guide/pa-files/PAR-02-140.html
Announcement of NINDS High Throughput Drug Screening
Service and Call for Assay Proposals http://grants1.nih.gov/grants/guide/notice-files/NOT-NS-04-005.html
Administrative Supplements for the Sharing and Distribution
of Mouse Genetic Models http://grants2.nih.gov/grants/guide/notice-files/NOT-NS-04-009.html
Novel Approaches to Enhance Animal Stem Cell Research
http://grants.nih.gov/grants/guide/pa-files/PA-04-125.html
Centers of Excellence in Translational Human Stem Cell
Research http://grants.nih.gov/grants/guide/rfa-files/RFA-NS-05-005.html
Small Business Innovation Research (SBIR) and Small Business
Technology Transfer (STTR) to Improve The Chemistry and
Targeted Delivery of RNAi Molecules http://grants.nih.gov/grants/guide/pa-files/PA-05-041.html
NIH GENERAL RESOURCES
Muscular Dystrophy Coordinating Committee
http://www.ninds.nih.gov/find_people/groups/mdcc/
CINCH (Consortium for Clinical Investigations of
Neurological Channelopathies )
http://www.rarediseasesnetwork.org/cinch/index.htm
NINDS Human Genetics Resource Center
http://locus.umdnj.edu/ninds/
NINDS: Increasing Quality of Life in Mobility Disorders
http://grants.nih.gov/grants/guide/pa-files/PA-02-111.html
NINDS Institutional Center Core Grants to Support Neuroscience
Research http://grants.nih.gov/grants/guide/pa-files/PAR-02-059.html
Precursor Cells in Skeletal Muscle Repair and Hypertrophy
http://grants.nih.gov/grants/guide/pa-files/PA-02-136.html
Muscular Dystrophy: Pathogenesis and Therapies
http://grants.nih.gov/grants/guide/pa-files/PA-05-038.html
The SMA Project: A Collaborative Program to Accelerate
Therapeutics Development for Spinal Muscular Atrophy (SMA)
http://www.smaproject.org/Solicitations/opensolicitations.htm
NIAMS, NICHD, NINDS: Support for Muscular Dystrophy Workshops
and Research Conferences
http://grants1.nih.gov/grants/guide/pa-files/par-03-176.html
DHHS, NIH, NINDS: Assay Development for High Throughput
Molecular Screening (R03/R21)
http://grants.nih.gov/grants/guide/rfa-files/rfa-rm-06-004.html
CENTERS FOR DISEASE CONTROL
Duchenne/Becker Muscular Dystrophy Projects Page (MD
StarNet, Newborn screening, Family Needs Assessment and
Cardiac Health in Female Carriers)
http://www.cdc.gov/ncbddd/duchenne/cdc.htm
BURROUGHS WELLCOME FUND
RESOURCES
Clinical Scientists Awards in Translational Research
http://www.bwfund.org/programs/translational/clinical_scientists_main.html
HUMAN
SUBJECT PROTECTION
ADVERSE EVENT REPORTING
Gene Therapy (FDA)
http://www.fda.gov/cber/ltr/gt110599.htm
Gene Therapy (NIH and FDA): GeMCRIS – Genetic Modification
Clinical Research Information System
http://www4.od.nih.gov/oba/RAC/GeMCRIS/GeMCRIS.htm
MedWatch: The FDA Safety Information and Adverse Event
Reporting Program(Post-marketing surveillance)
http://www.fda.gov/medwatch/
GENERAL HUMAN SUBJECT
PROTECTION
NIH – Office of Human Subjects Research
http://www.nihtraining.com/ohsrsite/
NIH – Bioethics Resources on the Web
http://www.nih.gov/sigs/bioethics/casestudies.html
DHHS - Office for Human Research Protections
http://ohrp.osophs.dhhs.gov/
FDA - Guidance for Institutional Review Boards and Clinical
Investigators
http://www.fda.gov/oc/ohrt/irbs/default.htm
FDA - A Guide to Informed Consent
http://www.fda.gov/oc/ohrt/irbs/informedconsent.html
The Belmont Report: Ethical Principles and Guidelines
for the Protection of Human
Subjects of Research
http://ohsr.od.nih.gov/mpa/belmont.php3
Nuremberg Code
http://ohsr.od.nih.gov/nuremberg.php3
Helsinki Declaration
http://ohsr.od.nih.gov/helsinki.php3
Federal Policy for the Protection of Human Subjects
http://ohrp.osophs.dhhs.gov/humansubjects/guidance/45cfr46.htm
ANIMAL WELFARE
USDA Animal Welfare Act
http://www.aphis.usda.gov/ac/awa.html
Institutional Animal Care and Use Committees (IACUC)
http://www.iacuc.org/
NIH - Office of Animal Care and Use (OACU)
http://oacu.od.nih.gov/
American College of Laboratory Animal Medicine (ACLAM)
http://www.aclam.org/
Association for the Assessment and Accreditation of
Laboratory Animal Care International
http://www.aaalac.org/
TISSUE AND REAGENT
BANKS
Brain and Tissue Bank Resources
http://www.ninds.nih.gov/find_people/voluntary_orgs/vol_org_sub_BB.htm
Cord Blood Registry
http://www.cordblood.com
BrainNet Europe
http://www.brainnet-europe.org
Medical Solutions Biomaterials Resource
http://www.biomaterialsresource.com/
Facioscapulohumeral Muscular Dystrophy (FSHD) Tissue Bank
http://www.mda.org/research/ct-fshtissue.html
Morris Neuromuscular Antibody Collection
www.rjah.nhs.uk/cind/morrisge/mabs.htm
ANIMAL MODELS
JACKSON LABORATORIES
CMT 1A Mouse
http://jaxmice.jax.org/jaxmicedb/html/model_989.shtml
Duchenne/Becker - mdx
http://jaxmice.jax.org/jaxmicedb/html/model_1022.shtml
Congential muscular dystrophy (CMD) - merosin deficient
http://jaxmice.jax.org/jaxmicedb/html/model_1019.shtml
LGMD 2F – delta-sarcoglycan deficient
http://jaxmice.jax.org/jaxmicedb/html/model_1503.shtml
LGMD 2B – dysferlin deficiency
http://jaxmice.jax.org/jaxmicedb/html/model_1503.shtml
Myotonia Congenita (Thomsen)
http://jaxmice.jax.org/jaxmicedb/html/model_1023.shtml
Amyotrophic lateral sclerosis (ALS) – SOD1 mouse
http://jaxmice.jax.org/jaxmicedb/html/model_982.shtml
Neuromuscular models
http://jaxmice.jax.org/jaxmicedb/html/model_121.shtml
Spinal muscular atrophy (SMA)
http://jaxmice.jax.org/jaxmicedb/html/model_1733.shtml
OTHER SOURCES
Amyotrophic lateral sclerosis (ALS) – SOD1 rat
http://www.taconic.com/emerging/002148.htm
REGISTRIES
National Registry of Veterans with Amyotrophic Lateral
Sclerosis
http://hsrd.durham.med.va.gov/ERIC/ALS/ALSregistry.htm
ALS Patient Care Database
http://www.umassmed.edu/outcomes/als/
The Myositis Association
http://www.myositis.org/patient_registry/index.html
The International Spinal Muscular Atrophy Patient Registry
http://www.fsma.org/registry2002.shtml
National Registry of Myotonic Dystrophy & FSHD Patients
and Family Members
http://www.urmc.rochester.edu/nihregistry/
North American Charcot-Marie-Tooth disease (CMT) Database
http://www.med.wayne.edu/neurology/clin_programs/labs/CMT/index.htm
The Utah Dystrophinopathy Project
http://dystrophy.genetics.utah.edu/
CLINICAL STUDIES AND
DATABASES
CIDD (Clinical Investigation of Duchenne Dystrophy);
Clinical research study group that conducted natural history
and therapeutic studies between 1979 and 1987
http://archneur.ama-assn.org/cgi/content/abstract/44/8/808
Leiden Mutation Databases
http://www.dmd.nl/
Human Gene Mutation Database
http://archive.uwcm.ac.uk/uwcm/mg/hgmd0.html
MD STARnet
http://www.cdc.gov/ncbddd/duchenne/MD%20STARNet%20Factsheet.pdf
and http://www.cdc.gov/ncbddd/duchenne/cdc.htm
Utah dystrophinopathy Project
http://dystrophy.genetics.utah.edu
National Initiative for Families with Duchenne (NIFD)
Survey http://www.cdc.gov/ncbddd/duchenne/cdc.htm
CINRG, Cooperative International Neuromuscular Research
Group http://www.cinrgresearch.org
UC Davis Longitudinal Assessment of Impairment, Activities,
QOL & Secondary Conditions in DMD
http://nmdinfo.net/rrtc/programoverview/teamrrtc.asp#project1
CINCH (Consortium for Clinical Investigations of
Neurological Channelopathies )
http://www.rarediseasesnetwork.org/cinch/index.htm
PRACTICE PARAMETERS
AND CONSENSUS STATEMENTS
Pulmonary
Respiratory Care of the Patient With Duchenne Muscular
Dystrophy. American Thoracic Society Consensus Statement
– March 2004.
http://www.thoracic.org/adobe/statements/duchenne1-10.pdf
Steroids
Corticosteroid Treatment of Duchenne Muscular Dystrophy.
Report of the Quality Standards Subcommittee of the American
Academy of Neurology and the Practice Committee of the
Child Neurology Society – January 2005.
http://www.neurology.org/cgi/reprint/64/1/13.pdf
Treatment of Duchenne Muscular Dystrophy: Defining the
gold standards of management in the use of corticosteroids.
Report on the 124 ENMC International Workshop –
April 2004.
Glucocorticoid corticosteroids for Duchenne muscular
dystrophy. Cochrane Database System Review. – 2005.
http://www.mrw.interscience.wiley.com/cochrane/clsysrev/articles/CD003725/frame.html
Cardicac
The management of cardiac involvement in muscular
dystrophy and myotonic dystrophy. 107th ENMC
International Workshop – June 2002.http://download.journals.elsevierhealth.com/pdfs/journals/0960-8966/PIIS0960896602002134.pdf
PUBLICATIONS
Cornetta K and Smith F. Regulatory Issues for Clinical
Gene Therapy Trials. Human Gene Therapy. 2002;13:1143-1149.
Escolar D, Henricson K, Pasquali L, Gorni K, and Hoffman
E. Collaborative translational research leading to multicenter
clinical trials in Duchenne muscular dystrophy: the Cooperative
International Neuromuscular Research Group (CINRG). Neuromuscular
Disorders. 2002; S147-S154.
Grilley B, and Gee A. Gene transfer: Regulatory issues
and their impact on the clinical investigator and the
good manufacturing production facility. Cytotherapy. 2003;5(3):197-207.
Hibberd PL, Weiner DL. Monitoring participant safety
in phase I and II interventional trials: Options and controversies.
J Investig Med. 2004 Nov;52(7):446-52.
DiMasi J, Hansen R, and Grabowski H. The price of innovation:
New estimates of drug development costs. Journal of Health
Economics. 2003; 22:151-185.
http://www.phase-ii.co.uk/drug1.pdf
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