A Phase 2/3 Study in Adult and Pediatric Participants with SCD

Phase 1

• Conditions: Sickle Cell Disease; MedDRA version: 21.0Level: PTClassification code: 10040644Term: Sickle cell disease Class: 100000004850; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

• Registration Number: CTIS2023-508766-14-00
• Lead Sponsor: Global Blood Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-04-05
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 229

Inclusion Criteria

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

Exclusion Criteria

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified