The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Seeks Industry Partners for Clinical Research Collaborations on Therapeutics, Diagnostics or Devices for Childhood Cholestatic Liver Diseases
Special Notice from the National Institutes of Health, for work in Bethesda, Maryland. Due May 31, 2029, 5:00 PM (UTC-4), 956 days left.
| Solicitation number | RFA-DK-23-017_RFA-DK-23-018 |
|---|---|
| Agency | National Institutes of Health |
| Office | National Institutes of Health Nichd |
| Notice type | Special Notice |
| Set-aside | None |
| Trade (NAICS) | Research and Development in the Physical, Engineering, and Life Sciences (except Nanotechnology and Biotechnology) (541715) |
| Product or service code | Q999 |
| Place of performance | Bethesda, Maryland |
| Posted | Sep 30, 2024 |
| Response deadline | May 31, 2029, 5:00 PM (UTC-4) (956 days left) |
Official notice and attachments
Description
The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Seeks Industry Partners for Clinical Research Collaborations on Therapeutics, Diagnostics or Devices for Childhood Cholestatic Liver Diseases The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) of the National Institutes of Health (NIH) of the Department of Health and Human Services (DHHS) seeks industry collaborators to provide novel therapeutic agents, diagnostic markers, biomarkers and devices for use in NIH-sponsored multi-center clinical trials and ancillary studies in patients with childhood cholestatic liver diseases, including biliary atresia, alpha-one antitrypsin deficiency, cystic fibrosis liver disease, Alagille syndrome, progressive familial intrahepatic cholestasis (PFIC), bile acid synthesis defects, mitochondrial respiratory chain and fatty acid oxidation defects, primary sclerosing cholangitis, and idiopathic neonatal hepatitis; and the liver disease related complications emanating from these diseases.
BACKGROUND: The Childhood Liver Disease Research and Education Network (ChiLDReN) is a multi-center consortium of 10 clinical sites in the United States plus a scientific data coordinating center funded by NIDDK to study rare childhood liver diseases that result in cholestasis, including primary sclerosing cholangitis, biliary atresia, alpha-one antitrypsin deficiency, cystic fibrosis liver disease, Alagille syndrome, progressive familial intrahepatic cholestasis (PFIC), bile acid synthesis defects, mitochondrial respiratory chain and fatty acid oxidation defects, and idiopathic neonatal hepatitis.
Five ongoing studies being conducted by ChiLDReN include:
- A Prospective Database of Infants with Cholestasis (PROBE), a longitudinal observational study starting in infancy which has enrolled 890 infants with biliary atresia and 621 with intrahepatic cholestasis;
- Biliary Atresia Study in Infants and Children (BASIC), a longitudinal study of children with biliary atresia both before and after liver transplant, which has enrolled over 1768 children with biliary atresia
- Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC), a longitudinal study of intrahepatic cholestasis diseases, which has enrolled 474 subjects with Alagille syndrome, 572 with alpha-1 antitrypsin deficiency liver disease, 355 with PFIC and 36 with bile acid defects;
- Longitudinal study of Mitochondrial Hepatopathies (MITOHEP) with 73 participants; and
- Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH), a study of the prognostic value of hepatic ultrasound on the development of liver fibrosis in CF, which has enrolled over 770 subjects.
In addition, ChiLDReN completed a placebo-controlled clinical trial to determine if post-operative corticosteroid therapy improves serum bilirubin and survival without liver transplantation in 140 infants with biliary atresia who have undergone the Kasai surgery ("START"). Ancillary studies to evaluate the natural history, clinical outcomes and quality of life, pathogenesis, genetics and genomics, proteomics, metabolomics, lipidomics, imaging studies, and determinants of progression and severity of these cholestatic liver diseases present a variety of research collaboration opportunities.
ChilDReN is also interested in conducting clinical trials of promising therapies to improve clinical outcomes for these cholestatic liver diseases. Finally, improved diagnostics or screening procedures for biliary atresia and cholestasis in infants are of great interest to ChiLDReN. STUDY GOALS: The overall goal of ChiLDReN is to determine the etiology, genetics, pathogenesis, natural history, clinical outcomes, complications, predictors of outcomes and optimal therapy of the cholestatic liver diseases.
ChiLDReN studies have enrolled and are following a relatively large and well characterized population of children and young adults with these rare disorders. ChiLDReN is interested in conducting research that will lead to improved clinical outcomes in children and young adults with these cholestatic liver diseases with industry collaborators to: • Evaluate the natural history, pathogenesis, diagnosis, genetic factors, genomics, proteomics, metabolomics, lipidomics, epigenomics, imaging studies, and determinants of progression and severity of biliary atresia, alpha-one antitrypsin deficiency, cystic fibrosis liver disease, Alagille syndrome, PFIC, bile acid synthesis defects, mitochondrial respiratory chain and fatty acid oxidation defects, primary sclerosing cholangitis, or idiopathic neonatal hepatitis. • 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, genomic, proteomic, metabolomic, and lipidomic variables • Explore the utility of these serum markers as surrogate markers of therapeutic response in study subjects participating in treatment trials (e.g., START study for biliary atresia) • Investigate proprietary drugs (such as novel choleretic agents, anti-fibrogenic agents, autophagy inducers, chloride channel inducers, fat-soluble vitamin supplements, bone mineral density agents, etc.), reagents, or devices in controlled randomized clinical trials as potential diagnostics or therapies for biliary atresia, alpha-one antitrypsin deficiency, cystic fibrosis liver disease, Alagille syndrome, PFIC, bile acid synthesis defects, mitochondrial respiratory chain and fatty acid oxidation defects, primary sclerosing cholangitis, or idiopathic neonatal hepatitis • Evaluate novel newborn or infant screening techniques or devices for biliary atresia or cholestasis. • Evaluate noninvasive imaging methods for assessing fibrosis, biliary disease, and parenchymal pathology in these diseases including but not limited to the use of elastography, nuclear magnetic resonance imaging, and molecular imaging. • Evaluate the
The description continues in the official notice.
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