SPECIAL NOTICE
A -- The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Seeks Industry Collaborators for NAFLD, NASH & Cryptogenic Cirrhosis Clinical Studies
- Notice Date
- 9/6/2013
- Notice Type
- Special Notice
- NAICS
- 541712
— Research and Development in the Physical, Engineering, and Life Sciences (except Biotechnology)
- Contracting Office
- Department of Health and Human Services, National Institutes of Health, Nat'l Institute of Diabetes, Digestive, & Kidney Diseases, 2 Democracy Plaza, Suite 700W, 6707 Democracy Blvd., MSC 5455, Bethesda, Maryland, 20892-5455
- ZIP Code
- 20892-5455
- Solicitation Number
- NIHLM2013-DK01
- Archive Date
- 1/15/2014
- Point of Contact
- Edward J Kostolansky, Phone: 301-594-4758
- E-Mail Address
-
ekostola@niddk.nih.gov
(ekostola@niddk.nih.gov)
- Small Business Set-Aside
- N/A
- Description
- The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Seeks Industry Collaborators for NAFLD, NASH & Cryptogenic Cirrhosis Clinical Studies National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) of the National Institutes of Health of the Department of Health and Human Services (DHHS) seeks Industry collaborators interested in using their novel therapeutic agents, diagnostic markers and devices in NIH-sponsored multi-center clinical trials and studies in patients with nonalcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), or cryptogenic cirrhosis. BACKGROUND: In 2002, the NIDDK established a Clinical Research Network (CRN), the goal of which was to facilitate and perform clinical, scientific, epidemiological and therapeutic research in NASH. The NASH CRN is anticipated to enter its third cycle of funding commencing on May 2014, with the mission of continuing and expanding the research in NASH and NAFLD with new specific goals. Completed studies - the NAFLD Database 2 Study was a prospective database of adult and pediatric cases of nonalcoholic fatty liver disease or cryptogenic cirrhosis was created by the NIDDK-funded NASH Clinical Research Network (CRN), accrued 1388 adults and 441 pediatric subjects from 2009 to 2013. The Database includes a comprehensive collection of clinical and liver histological data as well as a tissue bank. In addition to the NAFLD Database study, the NASH CRN has completed two treatment trials: (1) PIVENS: to evaluate whether 96 weeks of treatment with either pioglitazone or vitamin E lowers NASH activity as determined from hepatic histology in nondiabetic adults with NASH compared to treatment with placebo, (2) TONIC: to determine whether 96 weeks of treatment with either metformin or vitamin E leads to sustained reduction in serum alanine aminotransferase in nondiabetic children with NAFLD compared to treatment with placebo. The primary results of the PIVENS and TONIC Trials have been published in the New England Journal of Medicine 2010; 362: 1675-85 and Journal of the American Medical Association 2011; 305(16):1659-68, respectively. Current & future studies -In addition to the NAFLD Database 2 Study, two new clinical treatment trials in adults (FLINT) and children (CyNCh) are in progress, both with liver biopsy entry and upon exit from the studies. Further information on FLINT and CyNCh may be found at www.clinicaltrials.gov. Ancillary studies to evaluate the natural history, pathogenesis, genetic factors, proteomics, metabolomics, lipidomics, imaging studies, and determinants of progression and severity of nonalcoholic fatty liver disease present a variety of opportunities for collaborations. STUDY GOALS: There are an estimated 40-90 million individuals within the United States with NAFLD, 10-30% of who have NASH and may develop NASH-related cirrhosis. The overall goal of the NASH Clinical Research Network sponsored by the NIDDK is to focus on the etiology, diagnosis, contributing factors, natural history, complications, and therapy of nonalcoholic steatohepatitis. The NASH CRN studies currently comprise a large and well characterized population of individuals with various stages of nonalcoholic fatty liver disease, including steatosis, steatohepatitis, and cirrhosis. The NASH CRN is interested in conducting research that will lead to improved clinical outcomes in patients with nonalcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), and cryptogenic cirrhosis patients with industry collaborators to: • Evaluate the natural history, pathogenesis, diagnosis, genetic factors, proteomics, metabolomics, lipidomics, epigenomics, imaging studies and determinants of progression and severity of nonalcoholic fatty liver disease (NAFLD) and/or NASH as well as clinical trials to assess optimal treatment of adult and pediatric patients with NAFLD, NASH and/or cryptogenic cirrhosis. • Develop serum/plasma proteomic, metabolomic, lipidomic, microbiomic and expression arrays that are diagnostic of fatty liver disease or NASH or cryptogenic cirrhosis and that would provide staging and grading of the degree of cell injury, steatosis and fibrosis in the liver as well as insights into the pathogenesis of this disease. • Explore use of serum markers for fibrosis and serum markers for disease activity to predict hepatic histology either by themselves or in combination with other clinical, laboratory, proteomic, metabolomic, and lipidomic variables in the NAFLD Database study. • Explore the utility of these serum markers as surrogate markers of therapeutic response in study subjects participating in adult (PIVENS) and pediatric (TONIC) treatment trials. • Evaluate a panel of serological assays that reflect hepatic fibrosis, inflammation, insulin resistance, and oxidative stress to differentiate among NAFLD, NASH, or cryptogenic cirrhosis. • Investigate proprietary drugs, reagents, or devices in controlled randomized clinical trials as potential therapy for NASH, NAFLD and/or cryptogenic cirrhosis in children and/or adults. • Evaluate noninvasive imaging methods for assessing fat, inflammation, disease activity and/or fibrosis in NAFLD/NASH including but not limited to the use of elastography, ultrasonography, nuclear magnetic resonance imaging, and molecular imaging. • Identify proteomic alterations in patients with nonalcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), or cryptogenic cirrhosis. • Evaluate the role of lipid peroxidation and oxidative stress in the pathogenesis of NASH or NAFLD and analysis of the effect of various therapies on the levels of serum markers of oxidative stress in patients with NASH or NAFLD participating in treatment trials. • Evaluate the use of cytokine assays for analyses of serum/plasma cytokine levels as markers of necroinflammatory or fibrotic activity in NAFLD or NASH and as surrogate markers of histologic improvement in therapeutic trials of NAFLD or NASH. • Develop and evaluate specific reagents or tests that can be performed on formalin-fixed paraffin embedded tissue sections that would allow pathologists to positively identify and quantify ballooning hepatocellular injury or other specific hepatic injuries in liver biopsies as an adjunct to diagnosis in NAFLD and NASH. • Develop and validate image analysis techniques that would allow segmentation of the pathological features of NAFLD and NASH for better quantitation and characterization of the histological changes for cross-sectional or longitudinal studies. CAPABILITY STATEMENTS: Commercial organizations interested in pursuing clinical collaborations with NIDDK for NAFLD, NASH or cryptogenic cirrhosis are required to submit a Capability Statement to NIDDK. The Capability Statements submitted in response to this announcement will be used to evaluate and select industry collaborators. The Capability Statement should not exceed ten pages of narrative (not including appendices) and must include the following information: 1. A description of the therapeutic, diagnostic or device proposed to be used in the clinical research study (Note: The proposed therapeutic or device must have been tested already in a Phase I trial in humans). 2. A rationale for using the NASH CRN cohort. Projects that are synergistic with the mission of the NASH-CRN and will have the greater likelihood of advancing science in NASH would be prioritized. Applications that especially target a unique and/or understudied (such as pediatric or geriatric) population will be given priority based upon their novelty, innovation and significance to the mission of the NASH-CRN 3. Specific details of the methods to be utilized in the investigation of the pharmacologic, surgical or device intervention in patients with NAFLD or NASH and clearly describe important issues surrounding the evaluation of disease progression in these patients. 4. A detailed plan demonstrating the ability to commit sufficient quantities of the laboratory test agent or device in a timely manner for the duration of the study. 5. A description of laboratory tests that are needed including assays and required amount of specimens, to determine specific biomarker levels along with appropriate methods for performing such tests. 6. A description of other core facilities and interactions with core facilities that are needed. 7. A description of the methods that would be used to assure patient privacy and maintain confidentiality of data. 8. A letter of cross reference to the sponsor of the NASH studies to be included with any US FDA filing that contains the chemistry, manufacturing and control information for the drug substance and drug product or device Master File. 9. For novel agents, dosing and pharmacokinetic data from human studies. 10. Any related clinical adverse event profiles. 11. (Optional) outcome measures of interest to the Collaborator. The specifics of the proposed outcome measures and the proposed support should include but not be limited to treatment and evaluation of NAFLD or NASH or cryptogenic cirrhosis. 12. Any other resources the organization proposes to commit to the collaboration (in addition to the proposed therapeutic, diagnostic or device), which may include any of the following: equipment, reagents, supplies, access to facilities, personnel or services, and, if appropriate, funding (pursuant to a Cooperative Research and Development Agreement (CRADA) authorized under 15 U.S.C. § 3710a), to help support the conduct of the clinical study. 13. Affirmative statements that the organization will: a. agree to an independent review of data and statistical analyses and methodology. We encourage involvement of the NASH-CRN investigators in planning and execution of these tasks. b. agree to share (with NASH CRN) all safety data from other studies involving their preparation or device as well as relevant efficacy data from other studies (updated Investigator Brochure, etc). c. agree to have the results of the study published, irrespective of what those results may be. d. include the NASH-CRN investigators as co-authors where appropriate on all publications. The NASH-CRN strongly adheres to the International Committee of Medical Journal Editors criteria for authorship. e. work within the PHS intellectual property policies (see http://www.ott.nih.gov/policy/phspat_policy.aspx) and the NIDDK Biosample and Data repository requirements (see https://www.niddkrepository.org) SUBMISSION DEADLINE: December 31, 2013, 5:00pm Eastern Time. (Capability Statements received by NIDDK after the submission deadline above will not be considered.) Prospective collaborators may be invited to attend, at their own expense, a NASH Steering Committee meeting held in the Baltimore-Washington, DC area to discuss the information in their Capability Statement with the Steering Committee members. NOTE: NO FUNDS WILL BE PROVIDED BY NIDDK TO ANY ORGANIZATION SELECTED FOR THIS CLINICAL RESEARCH COLLABORATION OPPORTUNITY. SUBMISSION AND INQUIRIES: Submit Capability Statements to: National Institute of Diabetes and Digestive and Kidney Diseases E-mail: Dooe@niddk.nih.gov or averell.sherker@nih.gov. For Scientific Inquiries contact: Edward Doo, MD Director, Liver Diseases Research Program Division of Digestive Diseases and Nutrition National Institute of Diabetes and Digestive and Kidney Diseases National Institutes of Health Phone: 301-451-4524 Fax: 301-480-8300 E-mail: Dooe@niddk.nih.gov or Averell H. Sherker, MD, FRCP(C) Scientific Advisor for Viral Hepatitis and Liver Diseases Liver Diseases Research Branch National Institute of Diabetes and Digestive and Kidney Diseases National Institutes of Health 6707 Democracy Blvd, Room 653 Bethesda, MD. 20892-5450 (use 20817 for express mail) Telephone 301 451 6207 FAX: 301 480 8300 Email: averell.sherker@nih.gov For Inquiries Regarding Partnering with NIDDK (Intellectual Property Matters and Collaboration Agreements) contact: S. Vandana Chawla, JD, MPH Senior Technology Transfer Specialist Technology Advancement Office NIDDK/NIH Phone: 301-594-6762 chawlav@niddk.nih.gov The Notice of Opportunity is also posted at: http://techdev.niddk.nih.gov/collabs.shtml
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