Sickle cell drug candidate tested in first human trial – but study halted early

NCT ID NCT05660265

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This was a first-in-human study testing a single dose of an experimental drug called GSK4172239D in 11 people with sickle cell disease. The main goals were to check safety and how the drug moves through the body. The study was terminated early, so results are limited.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

GSK4172239D (a prodrug that turns into GSK4106401 in the body)

What this could lead to

If successful, this could point toward a new oral treatment option for sickle cell disease.

What could go wrong

This was a very early (Phase 1) trial with only 11 participants, and it was terminated, so results may not be available or applicable. The drug is still experimental and may not prove safe or effective.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

hematologic disorder sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • GSK Investigational Site

    Miami, Florida, 33155, United States

  • GSK Investigational Site

    Tamarac, Florida, 33321, United States

  • GSK Investigational Site

    Atlanta, Georgia, 30315, United States

  • GSK Investigational Site

    Columbus, Georgia, 31904, United States

  • GSK Investigational Site

    Riverdale, Georgia, 30274, United States

  • GSK Investigational Site

    Las Vegas, Nevada, 89113, United States