TRND Clinical Research Studies
The following are current clinical research studies conducted by the TRND program and its collaborators. For details about these studies, click on the links below or visit clinicaltrials.gov.
Active Research Studies
Chronic Lymphocytic Leukemia
GNE Myopathy (previously known as Hereditary Inclusion Body Myopathy)
- A Phase II Study of DEX-M74 (ManNac) in Subjects with GNE Myopathy
- A Phase I Study to Evaluate the Safety and Tolerability of ManNAc in Subjects GNE Myopathy (Concluded)
- A Natural History Study of Patients with GNE Myopathy
Niemann-Pick Type C Disease
- Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
- Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Completed Research Studies
Sickle Cell Disease
- A Single Dose Study of the Safety, Blood Levels and Biological Effects of Aes-103 Compared to Placebo in Subjects with Stable Sickle Cell Disease
Industry Acquires First Drug Candidate from TRND Program
On July 9, 2014, NIH announced that biopharmaceutical company Baxter International has acquired a drug candidate developed by NCATS' Therapeutics for Rare and Neglected Diseases (TRND) researchers and collaborators. The small molecule, Aes-103, is designed to treat sickle cell disease, a genetic blood disorder that affects millions worldwide, including approximately 100,000 people in the United States — among them, 1 in 500 African-Americans.