HYDROXYUREA FAILURE

Clinical trials for HYDROXYUREA FAILURE explained in plain language.

Never miss a new study

Get alerted when new HYDROXYUREA FAILURE trials appear

Sign up with your email to follow new studies for HYDROXYUREA FAILURE, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.

Our safety recommendation!

By submitting, you agree to our Terms of use

Not yet finished but already full! 1

CRISPR breakthrough: One-Shot gene therapy aims to free kids from sickle cell pain crises

Disease control Ongoing

This study is testing a one-time treatment called CTX001 in children with severe sickle cell disease who don't respond well to the standard medication, hydroxyurea. The treatment uses the patient's own modified stem cells (a type of gene therapy using CRISPR technology) to try to…

Matched conditions: HYDROXYUREA FAILURE

Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control

Last updated Mar 20, 2026 14:48 UTC

HYDROXYUREA FAILURE

Get alerted when new HYDROXYUREA FAILURE trials appear

CRISPR breakthrough: One-Shot gene therapy aims to free kids from sickle cell pain crises