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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

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

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