Opportunities / HEALTH AND HUMAN SERVICES, DEPARTMENT OF
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
| Notice type | Special Notice |
|---|---|
| Solicitation # | RFA-DK-23-017_RFA-DK-23-018 |
| Department | HEALTH AND HUMAN SERVICES, DEPARTMENT OF |
| Sub-tier / office | NATIONAL INSTITUTES OF HEALTH / NATIONAL INSTITUTES OF HEALTH NICHD |
| NAICS | 541715 — Research and Development in the Physical, Engineering, and Life Sciences (except Nanotechnology and Biotechnology) |
| PSC | Q999 |
| Set-aside | None |
| Posted | Sep 30, 2024 |
| Response deadline | May 31, 2029 (2029-05-31T17:00:00-04:00) |
| Place of performance | Bethesda, MD, 20817 |
| Primary contact | Edward Doo · dooe@niddk.nih.gov · 301-451-4524 |
Description (excerpt)
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: 1) 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; 2) 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 3) 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; 4) Longitudinal study of Mitochondrial Hepatopathies (MITOHEP) with 73 participants; and …