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
Hereditary Inclusion Body Myopathy
- A Phase 1 Study to Evaluate the Safety and Tolerability of ManNAc in Subjects with Hereditary Inclusion Body Myopathy
- A Natural History Study of Patients with Hereditary Inclusion Body 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
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
NIH announces start of clinical trial using cyclodextrin as a potential treatment for Niemann-Pick disease type C1 — a rare, fatal neurological disorder. The collaborative phase I trial, which stems from pilot work pursued by the TRND program, will test the drug in nine patients.
Pre-clinical scientists at NCATS have also published promising work on another treatment candidate for Niemann-Pick type C1: delta-tocopherol, a form of vitamin E.