A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease.

Status

Not yet recruiting

Phases

Phase 2Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-515202-84-00

Acronym

AG348-C-020

Enrollment

Registered

2024-08-13

Start date

Unknown

Completion date

Unknown

Last updated

2026-01-26

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

Conditions

Anemia in subjects with sickle cell disease (SCD)

Brief summary

Hemoglobin (Hb) response, defined as a ≥1.0 g/dL increase in average Hb concentration from Week 24 through Week 52 compared with baseline, Annualized rate of SCPCs (as defined in Section 8.5.2.1)

Detailed description

Average change from baseline in Hb concentration from Week 24 through Week 52, Average change from baseline in indirect bilirubin from Week 24 through Week 52, Average change from baseline in percent reticulocyte from Week 24 through Week 52, Average change from baseline in Patient-Reported Outcomes Measurement Information System®(PROMIS) Fatigue 13a Short Form (SF) scores from Week 24 through Week 52, Annualized frequency of hospitalizations for SCPC

Interventions

DRUGMITAPIVAT

DRUG"Placebo to Match Mitapivat Tablets

DRUG5 mg and 20 mg

DRUGare supplied as film-coated

DRUGblue

DRUGround tablets for oral administration Placebo to Match Mitapivat Tablets

DRUG50 mg or 100 mg

DRUGoblong tablets for oral administration. Placebo to Match Mitapivat Granules

DRUG1 mg

DRUGwhite

DRUGround granules for oral administration"

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Secondary

Measure	Time frame
Average change from baseline in Hb concentration from Week 24 through Week 52, Average change from baseline in indirect bilirubin from Week 24 through Week 52, Average change from baseline in percent reticulocyte from Week 24 through Week 52, Average change from baseline in Patient-Reported Outcomes Measurement Information System®(PROMIS) Fatigue 13a Short Form (SF) scores from Week 24 through Week 52, Annualized frequency of hospitalizations for SCPC	—

Measure

Time frame

—

Primary

Measure	Time frame
Hemoglobin (Hb) response, defined as a ≥1.0 g/dL increase in average Hb concentration from Week 24 through Week 52 compared with baseline, Annualized rate of SCPCs (as defined in Section 8.5.2.1)	—

Countries

Belgium, France, Germany, Italy, Netherlands

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026