Abstract

No treatment has been found that prevents or reverses the progressive muscle wasting in myotonic dystrophy (DM). DM is unusual among the muscular dystrophies in that muscle wasting is associated with decreased anabolism, rather than increased catabolism. Circulating levels of DHEAS, a steroid hormone that is anabolic for skeletal muscle are markedly reduced in DM. Results of a recent open label study suggest that daily IV DHEAS improves strength and function in patients with DM. This study is an open label trial of oral DHEAS in 12 subjects with DM. The primary aim is to assess the safety and tolerability of oral DHEAS. The secondary aim is to compare the pharmacokinetics of oral and IV DHEAS, ascertian trough levels during chronic administration or oral DHEAS at 2 dose levels & refine outcome measures.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000044-39
Application #
6407568
Study Section
General Clinical Research Centers Committee (CLR)
Project Start
1974-10-01
Project End
2001-02-28
Budget Start
Budget End
Support Year
39
Fiscal Year
2000
Total Cost
Indirect Cost

  Institution

Name
University of Rochester
Department
Type
DUNS #
208469486
City
Rochester
State
NY
Country
United States
Zip Code
14627

  Publications

Askie, Lisa M; Darlow, Brian A; Finer, Neil et al. (2018) Association Between Oxygen Saturation Targeting and Death or Disability in Extremely Preterm Infants in the Neonatal Oxygenation Prospective Meta-analysis Collaboration. JAMA 319:2190-2201
Di Fiore, Juliann M; Martin, Richard J; Li, Hong et al. (2017) Patterns of Oxygenation, Mortality, and Growth Status in the Surfactant Positive Pressure and Oxygen Trial Cohort. J Pediatr 186:49-56.e1
Ternullo, Sharon R; Abdolahi, Amir; Williams, Geoffrey C (2017) Study of monotherapy versus combination therapy for tobacco dependence among heavily addicted smokers. J Am Pharm Assoc (2003) 57:77-81.e1
Younge, Noelle; Goldstein, Ricki F; Bann, Carla M et al. (2017) Survival and Neurodevelopmental Outcomes among Periviable Infants. N Engl J Med 376:617-628
Williams, Geoffrey C; Niemiec, Christopher P; Patrick, Heather et al. (2016) Outcomes of the Smoker's Health Project: a pragmatic comparative effectiveness trial of tobacco-dependence interventions based on self-determination theory. Health Educ Res 31:749-759
Archer, Stephanie Wilson; Carlo, Waldemar A; Truog, William E et al. (2016) Improving publication rates in a collaborative clinical trials research network. Semin Perinatol 40:410-417
Ettensohn, David B; Frampton, Mark W; Nichols, Joan E et al. (2016) Human Alveolar Macrophages May Not Be Susceptible to Direct Infection by a Human Influenza Virus. J Infect Dis 214:1658-1665
Phelps, Dale L; Ward, Robert M; Williams, Rick L et al. (2016) Safety and pharmacokinetics of multiple dose myo-inositol in preterm infants. Pediatr Res 80:209-17
Foglia, Elizabeth E; Nolen, Tracy L; DeMauro, Sara B et al. (2015) Short-term Outcomes of Infants Enrolled in Randomized Clinical Trials vs Those Eligible but Not Enrolled. JAMA 313:2377-9
O'Connor, Thomas G; Wang, Hongyue; Moynihan, Jan A et al. (2015) Observed parent-child relationship quality predicts antibody response to vaccination in children. Brain Behav Immun 48:265-73

Showing the most recent 10 out of 509 publications