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Could a pill replace daily shots for growth hormone deficiency?

NCT ID NCT05250063

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested an oral drug called LUM-201 in 15 children with growth hormone deficiency who had already received daily injections for a year. The goal was to see if LUM-201 could maintain or improve growth over 12 months. The drug was given once daily by mouth, and researchers measured height changes and monitored for side effects.

What this could mean

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

Active substance
LUM-201 (oral drug)
What this could lead to
If successful, this could offer a more convenient oral treatment option for children with growth hormone deficiency, reducing the need for daily injections.
What could go wrong
This is a small, early-phase study with only 15 participants, so results may not apply to all children. The drug may not work as well as current treatments or could have side effects.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

15 people

The number who actually took part.

Started

Feb 2022

Finished

Jan 2025

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

3 to 13 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

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: * Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable. * Must have previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial. * Is eligible for study participation as confirmed by the principal investigator (PI) Exclusion Criteria: * Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201. * Uses any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors. * Has planned or is receiving current long-term treatment with medications known to act as substrates, inducers, or inhibitors of the cytochrome system CYP3A4 that metabolizes LUM-201

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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

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010, United States

  • Indiana University School of Medicine

    Indianapolis, Indiana, 46202, United States

  • Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki

    Lodz, Poland

  • Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka

    Warsaw, Poland

  • M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic

    Minneapolis, Minnesota, 55454, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Rady Children's Hospital

    San Diego, California, 92123, United States

  • Sonomed - Centrum Medyczne

    Szczecin, Poland

  • The Children's Mercy Hospital

    Kansas City, Missouri, 64111, United States

  • University of Virginia Health System

    Charlottesville, Virginia, 22908, United States

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