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New pill could tame phenylketonuria by blocking amino acid absorption

NCT ID NCT07685210

Recruiting now Disease control Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Jul 06, 2026 · Last updated Jul 07, 2026 · Updated 1 time

Summary

This early-stage trial tests a single dose of an experimental oral drug called GenSci144 in healthy adults. The drug aims to block a transporter in the gut to reduce phenylalanine levels, which could help manage phenylketonuria (PKU). The study focuses on safety and how the body handles the drug, not yet on treating the disease.

What this could mean

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

Active substance
GenSci144 (SLC6A19 transporter inhibitor)
What this could lead to
If successful, this could lead to a new oral treatment option for people with phenylketonuria, potentially helping manage their condition more easily.
What could go wrong
This is an early Phase 1 trial in healthy volunteers, not patients. The drug may prove unsafe, ineffective, or cause side effects. Success is far from guaranteed.

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.

phenylketonuria

As listed by the trial registrant

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

PHENYLKETONURIA

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • The First Affiliated Hospital of Shandong First Medical University (Shandong Qianfoshan Hospital).

    RECRUITING

    Jinan, Shandong, 250014, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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