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Could a daily pill replace growth hormone shots for children?

NCT ID NCT04614337

Disease control Sponsor: Lumos Pharma Source: ClinicalTrials.gov ↗

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tests an oral medication called LUM-201 in children with growth hormone deficiency who have not yet received treatment. The goal is to see if taking a pill daily can improve growth as effectively as standard daily injections. The trial also looks for a way to identify which children are most likely to benefit from LUM-201.

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 daily pill as an alternative to daily injections for children with growth hormone deficiency.
What could go wrong
This is an early phase 2 trial with a small number of participants, so results may not confirm effectiveness or safety. The drug may not work for all children.

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

104 people

The number who actually took part.

Started

Dec 2020

Finished

Sep 2024

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 12 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: * Have an established diagnosis of idiopathic PGHD as determined by standard diagnostic criteria. Eligible subjects must be naïve-to-treatment and be prepubertal. * Morning cortisol ≥ 7 µg/dL or stimulated cortisol ≥ 14 µg/dL. * At Screening, be ≥ 3.0 years and ≤ 11.0 years for girls and ≤ 12.0 years for boys. * Have HT-SDS ≤ -2.0 or HT-SDS ≥ 2 SD below mean parental HT-SDS. * Have a baseline height velocity \< 5.5 cm/year based on at least 6 months of growth. * Have a bone age delayed by ≥ 6 months with respect to chronological age. * Have prepubertal status as evidenced by Tanner Stage I breast development in girls and testicular volume \< 4.0 mL in boys. * In girls, have genetic testing results to rule out Turner syndrome. If SHOX genetic testing results are available, they need to be negative. * Have normal thyroid function. Subjects diagnosed with hypothyroidism must have documented successful treatment for at least 30 days prior to Day 1. Exclusion Criteria: * Any medical or genetic condition which, in the opinion of the Investigator or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment. (Examples: diabetes, idiopathic short stature). * A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201 or rhGH. * Use of any medication that, in the opinion of the Investigator and/or MM, can independently cause short stature or limit the response to exogenous growth factors (Example: glucocorticoids). * Evidence or history of an intracranial mass (e.g., pituitary tumor, craniopharyngioma). * Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3 ng/mL on two prior standard of care GH stimulation tests, or pituitary deficiencies beyond GH and thyroid function. * Malnutrition as evidenced by medical history or a body weight \< 3rdth percentile for current height. * BMI \> 95th percentile. * Gestational age-adjusted birth weight \< 5th percentile (small for gestational age). * History of spinal, cranial, or total body irradiation. * Treatment with medications known to act as moderate or strong inhibitors or strong inducers of CYP3A/4, or with medications known to act as strong inhibitors of P-glycoprotein (P-gp) or potent substrates of P-gp or Multidrug and toxin extrusion protein 1 (MATE1).

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Conditions

The condition(s) this trial relates to.

isolated congenital growth hormone deficiency pituitary dwarfism

As listed by the trial registrant

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

Growth hormone deficiency

Contacts and locations

Locations

  • Atlanta Diabetes Associates

    Atlanta, Georgia, 30318, United States

  • Canberra Hospital

    Garran, Australian Capital Territory, 2605, Australia

  • Center of Excellence in Diabetes and Endocrinology

    Sacramento, California, 95821, United States

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224, United States

  • Children's Minnesota

    Saint Paul, Minnesota, 55102, United States

  • Children's National Hospital

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

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104, United States

  • Department of Pediatrics and Endocrinology- Monash Health

    Clayton, Victoria, 3168, Australia

  • Diabetes & Glandular Disease Clinic, P.A.

    San Antonio, Texas, 78229, 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 Wieku Rozwojowego UM

    Wroclaw, Poland

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

    Warsaw, Poland

  • Klinika Pediatrii, Diabetologii i Endokrynologii Gdansk

    Pomorskie, Poland

  • Klinika Pediatrii, Endokrynologii, Diabetologii z Pododdziałem Kardiologii, Uniwersytecki Dziecięcy Szpital Kliniczny im.Ludwika Zamenhofa w Białymstoku

    Bialystok, Poland

  • Liggins Institute, University of Auckland

    Auckland, New Zealand

  • M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic

    Minneapolis, Minnesota, 55454, United States

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • MultiCare Institute for Research and Innovation

    Tacoma, Washington, 98405, United States

  • NYU Grossman School of Medicine

    New York, New York, 10016, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Novak Center For Childrens Health

    Louisville, Kentucky, 40202, United States

  • Pediatric Endocrine Associates

    Greenwood Village, Colorado, 80111, United States

  • Penn State College of Medicine

    Hershey, Pennsylvania, 17033, United States

  • Queensland Children's Hospital

    South Brisbane, Australia

  • Rady Children's Hospital

    San Diego, California, 92123, United States

  • Royal Children's Hospital

    Melbourne, Victoria, 3052, Australia

  • SP Dziecięcy Szpital Kliniczny w Warszawie

    Warsaw, Poland

  • Schneider Children's Medical Center Institute for Endocrinology and Diabetes National Center

    Petah Tikva, Tiqwa, 4920235, Israel

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Sonomed - Centrum Medyczne

    Szczecin, Poland

  • State Institution 'V. P. Komissarenko Institute of Endocrinology and Metabolism of the National academy of medical science of Ukraine

    Kyiv, 04114, Ukraine

  • Texas Tech University Health Sciences Center

    Amarillo, Texas, 79106, United States

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • The Children's Mercy Hospital

    Kansas City, Missouri, 64111, United States

  • The Mount Sinai Hospital

    Mount Sinai, New York, 30093, United States

  • UBMD Pediatrics

    Buffalo, New York, 14203, United States

  • UMass Memorial Medical Center

    Worcester, Massachusetts, 01655, United States

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of Oklahoma Health Sciences Center, Pediatric Diabetes and Endocrinology

    Oklahoma City, Oklahoma, 73104, United States

  • University of Virginia Health System

    Charlottesville, Virginia, 22908, United States

  • Wellington Regional Hospital CCDHB

    Newtown, Wellington Region, 6021, New Zealand

  • klinika Pediatrii, Endokrynologii i Diabetologii Dziecięcej

    Rzeszów, Poland

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