6R-BH4 IN SUBJECTS WITH SICKLE CELL DISEASE

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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. Purpose: This study is designed to assess the safety and biologic activity of once daily oral administration of 4 escalating doses of 6R-BH4 to subjects with sickle cell disease. Subjects: Up to 40 subjects will be screened and enrolled at 5-6 sites in the United States. Procedures: After screening, eligible study participants will receive oral, once-daily doses of 6R-BH4 during a 16-week dose-escalation phase, with dose levels increasing within subjects every 4 weeks. Prior to dose escalation, the Investigator will evaluate each subject s safety profile during the previous dose level, using predefined parameters. If a subject does not meet the criteria for escalation to the next dose level or is de-escalated in dose, they will receive their highest tolerated dose for the duration of the 16-week period. The overall safety profile of 6R-BH4 will be evaluated during periodic safety review meetings between the Sponsor and Investigators. Following the 16-week dose escalation phase,participants may continue on the highest tolerated study drug in an extension phase lasting up to 2 years.