C5351004 - A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease - C5351004

Global Blood Therapeutics Inc.0 个研究点目标入组 229 人开始时间: 2024年4月5日最近更新: 2024年8月21日

适应症

试验速览

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•Part B/C 1. Participant with SCD. Documentation of SCD genotype HbSS or HbSB, may be based on history of laboratory testing or must be confirmed by laboratory testing during Screening., Part B 2. Age 12 years and older, inclusive at Screening. Participants age 12 to < 18 years will only be enrolled after evaluation of safety in this age cohort in Part C Cohort C1., Part B 3. More than or equal to 2 and = 10 VOCs, within 12 months of Screening., Part B 4./C 3. Hb = 5.5 and = 10.5 g/dL during Screening and considered stable by the Investigator., Part B 5./C 4. For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the Informed Consent Document or Assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator and no sign of hematological toxicity., Part C 2. Age by cohort at Screening: a. Cohort C1: 12 to < 18 years; b. Cohort C2: 6 to < 12 years; c. Cohort C3: 2 to < 6 years; d. Cohort C4: 6 months to < 2 years

•Part B 1. / C2. Female participant who is breastfeeding or pregnant., Part B 2./C 3. Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or has received an RBC or exchange transfusion for any reason within 90 days of Day 1., Part B 3./ C 4. Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the Informed Consent Document., Part B 4./ C5. Screening laboratory test of ALT > 4 × ULN for age., Part B 5./ C 6. Acute illness or clinically significant bacterial, fungal, parasitic, or viral infection which requires therapy, including acute bacterial infection requiring antibiotics within 14 days prior to the study drug administration., Part C 1. More than 10 VOCs within 12 months of Screening.

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