Awardee OrganizationBOSTON UNIVERSITY MEDICAL CAMPUS
Description
Abstract Text
This subproject is one of many research subprojects utilizing the
resources provided by a Center grant funded by NIH/NCRR. The subproject and
investigator (PI) may have received primary funding from another NIH source,
and thus could be represented in other CRISP entries. The institution listed is
for the Center, which is not necessarily the institution for the investigator.
This open-label Phase II cross-over study will test hypotheses that 1) Pulsed AB will induce higher levels of Hb F than HU alone, and 2) assessment of factors influencing erythropoietic rates may identify patient subsets who are more likely to respond to HU + AB. The study is open only to patients who have not had resolution of their symptoms with HU alone, and have a baseline Hb F of at least 2%. Patients will serve as their own controls. Clinical events and hematologic factors will be assessed in patients on HU alone for 6 months. AB therapy will then be added, (4 days, once/ month), and Hb F parameters and clinical events will be assessed on combined therapy. If additive effects are found with the two agents, the trial may provide a basis for selecting patients most likely to benefit from AB (in addition to HU therapy) and for design of Phase III trials. (This small trial will not serve as a pivotal trial for FDA approval of AB for treatment of sickle cell disease.)
Public Health Relevance Statement
Data not available.
NIH Spending Category
No NIH Spending Category available.
Project Terms
Arginine ButyrateClinicalComputer Retrieval of Information on Scientific Projects DatabaseCross-Over StudiesEventFundingGrantInstitutionLabelPatientsPhasePhase III Clinical TrialsPhysiologic pulsePulse takingRateResearchResearch PersonnelResolutionResourcesSickle Cell AnemiaSourceSymptomsTestingUnited States Food and Drug AdministrationUnited States National Institutes of Healthbasedaytherapy design
No Sub Projects information available for 2M01RR000533-39 8137
Publications
Publications are associated with projects, but cannot be identified with any particular year of the project or fiscal year of funding. This is due to the continuous and cumulative nature of knowledge generation across the life of a project and the sometimes long and variable publishing timeline. Similarly, for multi-component projects, publications are associated with the parent core project and not with individual sub-projects.
No Publications available for 2M01RR000533-39 8137
Patents
No Patents information available for 2M01RR000533-39 8137
Outcomes
The Project Outcomes shown here are displayed verbatim as submitted by the Principal Investigator (PI) for this award. Any opinions, findings, and conclusions or recommendations expressed are those of the PI and do not necessarily reflect the views of the National Institutes of Health. NIH has not endorsed the content below.
No Outcomes available for 2M01RR000533-39 8137
Clinical Studies
No Clinical Studies information available for 2M01RR000533-39 8137
News and More
Related News Releases
No news release information available for 2M01RR000533-39 8137
History
No Historical information available for 2M01RR000533-39 8137
Similar Projects
No Similar Projects information available for 2M01RR000533-39 8137