Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease

Summary

This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to \< 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.

Timeline

Start

2020-11-11

Primary completion

2024-01-18

Completion

2024-11-06

Outcome

Met primary endpoint

Stopped: “Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment.”

registry Voxelotor vs Placebo; p = 0.0048; Difference in Least Square (LS) mean -7.77 (95% CI -13.18 to -2.37) NCT04218084 ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Voxelotor	Small molecule	1500 mg	Oral

Indications