A Phase 2/3 Study in Adult and Pediatric Participants with Sickle Cell Disease (SCD)

A Phase 2/3, Randomized, Multicenter Study of GBT021601 Administered Orally to Participants with Sickle Cell Disease and an Open- Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease.

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

PACTR

Registry ID

PACTR202208531640912

Enrollment

480

Registered

2022-08-10

Start date

2022-06-30

Completion date

Unknown

Last updated

2026-08-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Haematological Disorders

Interventions

Drug GBT021601

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Inclusion Criteria: Part A, Part B, and Part C: • Male or female with SCD • Participants with stable hemoglobin value • Participants on HU should be on stable dose for at least 90 days prior to signing ICF Part B: • Participants with SCD ages 12 to 65 years, inclusive • Participants with more than or equal to 2 and less than or equal to 10 VOCs inclusive, within 12 months of Screening

Exclusion criteria

Exclusion criteria: Part A, Part B, and Part C: • Participants who had more than 10 VOC within 12 months of screening • Female participant who is breastfeeding or pregnant • Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1 • Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF

Design outcomes

Primary

Measure	Time frame
1.Part A [ Time Frame: Through week 12] Number of adult participants with change from baseline in hemoglobin (Hb) through week 12 2.Part B [ Time Frame: Through week 48] Proportion of participants with an increase from baseline of >1 g/dL in Hb at week 48 3.Part C [ Time Frame: Through Week 2] Assess the pharmacokinetics, while observing maximum concentration (Cmax) after a single dose 4.Part C [ Time Frame: Through Week 2] Assess the pharmacokinetics, while observing minimum concentration (Cmin) and maximum concentration after multiple dose administration	—

Measure

Time frame

1.Part A [ Time Frame: Through week 12] Number of adult participants with change from baseline in hemoglobin (Hb) through week 12 2.Part B [ Time Frame: Through week 48] Proportion of participants with an increase from baseline of >1 g/dL in Hb at week 48 3.Part C [ Time Frame: Through Week 2] Assess the pharmacokinetics, while observing maximum concentration (Cmax) after a single dose 4.Part C [ Time Frame: Through Week 2] Assess the pharmacokinetics, while observing minimum concentration (Cmin) and maximum concentration after multiple dose administration

—

Secondary

Measure	Time frame
1. Part A [ Time Frame: Through week 12] Proportion of participants with an increase from baseline of >1 g/dL in Hb through Week12 2. Part B [ Time Frame: Through week 48] Annualized rate of vaso-occlusive crisis (VOC)through end of Week48 3. Part C [ Time Frame: Through Week 2] Change from baseline in Hb at MD Week 2 (Day 14).	—

Countries

Kenya, Nigeria, Uganda

Contacts

Public ContactJessica Guider

Senior Clinical Trial Manager

jguider@gbt.com+16303642275

Outcome results

None listed

Source: PACTR (via WHO ICTRP) · Data processed: Aug 9, 2026