Could a pill replace daily shots for growth hormone deficiency?
NCT ID NCT05250063
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
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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 Hide 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
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Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
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Indiana University School of Medicine
Indianapolis, Indiana, 46202, United States
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Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki
Lodz, Poland
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Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka
Warsaw, Poland
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M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic
Minneapolis, Minnesota, 55454, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Rady Children's Hospital
San Diego, California, 92123, United States
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Sonomed - Centrum Medyczne
Szczecin, Poland
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The Children's Mercy Hospital
Kansas City, Missouri, 64111, United States
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University of Virginia Health System
Charlottesville, Virginia, 22908, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Growth hormone treatment under Long-Term watch in thousands of children
- Weekly shot could replace daily hormone injections for growth hormone deficiency
- Could a daily pill replace growth hormone shots for children?