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New pill could tame phenylketonuria by blocking amino acid absorption

NCT ID NCT07685210

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 48 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2026

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 45 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Healthy men and women aged 18 to 45, with at least 3 people of the same gender in each group. * BMI 19\~26 kg/m², men weigh ≥50 kg, women ≥45 kg. * Voluntarily sign the informed consent form, fully aware of the research risks and procedures. * Use reliable contraception for 3 months after the last dose, no plans for pregnancy or sperm/egg donation. Exclusion Criteria: * A family history of sudden death and serious neuropsychiatric disorders. * Major underlying conditions like gastrointestinal absorption problems, chronic liver, kidney, heart, and endocrine diseases. * Liver function, kidney function with eGFR\<90, abnormal QTc interval, positive for various infectious diseases. * Drug allergies, drug abuse, recent participation in other clinical trials. * Drinking alcohol, smoking, taking grapefruit/amino acid supplements, and having a chaotic routine during the screening period. * Pregnant or breastfeeding women, people who can't tolerate blood draws or get faint from needles.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

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

    RECRUITING

    Jinan, Shandong, 250014, China

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