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Weekly shot could replace daily growth hormone for kids

NCT ID NCT07221851

Disease control Sponsor: Ascendis Pharma A/S 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 25, 2026 · Last updated Sep 03, 2026 · Updated 11 times

Summary

This phase 3 trial compares a once-weekly growth hormone injection (lonapegsomatropin) to a daily one (somatropin) in 186 prepubertal children with growth failure due to Turner syndrome, SHOX deficiency, being small for gestational age, or idiopathic short stature. The goal is to see if the weekly shot works as well as the daily one for increasing height. Participants will receive either weekly treatment for 2 years or daily treatment for 1 year followed by weekly treatment for 1 year.

What this could mean

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

Active substance
lonapegsomatropin (once-weekly injection) and somatropin (daily injection)
What this could lead to
If successful, this could offer children with growth disorders a more convenient once-weekly injection instead of daily shots, potentially improving adherence and quality of life.
What could go wrong
This is a phase 3 trial, but it is still relatively small (186 participants) and open-label, which can introduce bias. The weekly treatment may not prove as effective as daily injections for all conditions studied.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 186 people

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

Started

Dec 2025

Expected to finish

Mar 2029

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

2 to 17 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: 1. Chronological age between ≥2 and \<18 years, at start of screening. 2. Naïve to growth hormone and growth hormone promoting therapies. 3. Prepubertal. 4. Able to stand without assistance. 5. Diagnosis of TS, SHOX-D, SGA, or ISS with impaired growth or short stature, according to the following disease-specific criteria: TS or SHOX-D (Léri-Weill dyschondrosteosis): 1. Diagnosis confirmed by a genetic test. NOTE: Historical test results are acceptable for proof of diagnosis. For karyotypes, a minimum of 20 cells must be counted. 2. Impaired growth or short stature defined as: (i.) AHV \<25th percentile over a time span of 6-16 months prior to screening utilizing a historical height properly documented in a health care setting (self-measurement record is not accepted) OR (ii.) Height \<5th percentile for sex and age according to the Centers for Disease Control Growth Charts for the United States SGA without catch-up growth: c. Birth weight and/or birth length \< -2.0 SDS for gestational age according to the 2006 World Health Organization Child Growth Standards. For infants born premature, the Fenton Preterm Infant Growth Chart (Fenton 2013) should be used. d. Impaired growth or short stature defined as: (i.) AHV \<25th percentile over a time span of 6-16 months prior to screening properly documented in a health care setting (self-measurement record is not accepted) OR (ii.) Height \< -2.0 SDS for age and sex according to the 2000 Centers for Disease Control Growth Charts for the United States for children ≥ 3 years or height \< -2.5 SDS for age and sex according to the for children ≥ 2 years and \< 3 years ISS: e. Height \< -2.25 SDS for sex and age according to the Centers for Disease Control Growth Charts for the United States with no identifiable cause for short stature. f. Documented normal GH-IGF-1 axis, defined as either: (i.)IGF-1 SDS \>0 at screening based on central laboratory OR (ii.)Historical documentation of normal peak GH upon stimulation test (as defined by local institution) g. 46,XX chromosome as determined by karyotype or microarray if female. For karyotypes, a minimum of 30 cells must be counted. 6. If on hormone replacement therapies for any hormone deficiencies other than growth hormone (e.g., adrenal, thyroid), must be on adequate and stable doses for ≥4 weeks prior to and throughout screening. 7. Written, signed informed consent provided by parent(s) or legal guardian(s) of the participant. Assent should be signed by participant as required by IRB/HREC/IEC. Exclusion Criteria: 1. Advanced bone age X-ray by central reading defined as \>20% above chronological age in months (Greulich 1959). 2. Closed epiphyses as defined as bone age of ≥14.0 years in females or ≥16.0 years in males. 3. Current clinical diagnosis of diabetic retinopathy 4. Any diagnosis or presence at screening of the following: 1. Untreated moderate or severe sleep apnea as determined by formal (local) read of an inpatient or at-home sleep study. 2. Prader Willi syndrome with severe obesity, history of severe upper airway obstruction, or severe respiratory impairment. 5. Signs/symptoms of intracranial hypertension, active proliferative retinopathy. 6. Uncontrolled hypo- or hyperthyroidism. 7. Uncontrolled diabetes mellitus (defined as: HbA1c \>7.5% from central laboratory at screening). 8. Known history or diagnosis of any gastrointestinal inflammatory condition, HIV, radiation exposure, other skeletal dysplasias, growth hormone deficiency, and/or cardio-thoracic surgery due to their independent effects on growth. 9. Any significant hepatic or renal abnormality, such as abnormal renal function (defined as eGFR \<60 mL/min/1.73m2). 10. Undiagnosed or uncontrolled hypertension. 11. Receiving treatment with any agent that might influence growth or interfere with GH secretion or action including any sex steroids and stimulants for attention-deficit/hyperactivity disorder (ADHD). 12. High dose inhaled glucocorticoid for more than 28 consecutive days total over the course of 12 months. 13. Female who is pregnant, plans to be pregnant, or breastfeeding. 14. Participation in another interventional clinical trial involving an investigational compound within 90 days prior to screening or in parallel to this trial. 15. Any disease or condition that, in the judgement of the investigator, may make the participant unlikely to comply with the requirements of the protocol or any condition that presents undue risk from the investigational product or trial procedures. 16. Exclusion Criteria only applicable to TS: 1. Presence of Y chromosome material on genetic testing without history of gonadectomy. 2. Less than 10% of 45,X mosaicism. 3. Any known, clinically significant, congenital or acquired cardiovascular dysfunction that might interfere with growth. 17. Exclusion Criteria only applicable to SGA: a. Any known clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements: (i.)Chromosomal aneuploidy, significant gene mutations, or medical syndromes with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, abnormal SHOX-1 gene analysis or absence of GH receptors. (ii.)Congenital abnormalities (causing skeletal abnormalities), including but not limited to skeletal dysplasias. 18. Exclusion Criteria only applicable to ISS: 1. Known history of any condition that causes disproportionate short stature (i.e. skeletal dysplasias), chromosomal aneuploidy, significant gene mutations, or medical syndromes with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, abnormal SHOX-1 gene analysis or absence of gH receptors.

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Conditions

The condition(s) this trial relates to.

chromosomal disorder Dwarfism endocrine system disorder Failure to Thrive fetal growth restriction isolated short stature Noonan syndrome sex chromosome disorder of sex development Sex Chromosome Disorders SHOX-related short stature Turner syndrome

As listed by the trial registrant

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

Idiopathic short stature Short stature homeobox gene mutation Small for gestational age at delivery Turner syndrome

How to take part

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

  1. The places running it

    43 sites in 7 countries. 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

  • Ascendis Pharma Investigational Site

    RECRUITING

    Palo Alto, California, 94304, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Sacramento, California, 95821, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Centennial, Colorado, 80112, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Wilmington, Delaware, 19803, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Orlando, Florida, 32806, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    St. Petersburg, Florida, 33701, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Idaho Falls, Idaho, 83404, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    New Orleans, Louisiana, 70118, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Minneapolis, Minnesota, 55454, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Saint Paul, Minnesota, 55102, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Lake Success, New York, 11042, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Oklahoma City, Oklahoma, 73104, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Edinburg, Texas, 78539, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Fort Worth, Texas, 76104, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    San Antonio, Texas, 78229, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    San Antonio, Texas, 78232, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Seattle, Washington, 98105, United States

  • Ascendis Pharma Investigational Site

    RECRUITING

    Montpellier, 34090, France

  • Ascendis Pharma Investigational Site

    RECRUITING

    Paris, 75012, France

  • Ascendis Pharma Investigational Site

    RECRUITING

    Toulouse, 31300, France

  • Ascendis Pharma Investigational Site

    RECRUITING

    Düsseldorf, 40225, Germany

  • Ascendis Pharma Investigational Site

    RECRUITING

    Hanover, 30173, Germany

  • Ascendis Pharma Investigational Site

    RECRUITING

    Tübingen, 72076, Germany

  • Ascendis Pharma Investigational Site

    RECRUITING

    Florence, 50139, Italy

  • Ascendis Pharma Investigational Site

    RECRUITING

    Naples, 80131, Italy

  • Ascendis Pharma Investigational Site

    RECRUITING

    Naples, 80138, Italy

  • Ascendis Pharma Investigational Site

    RECRUITING

    Trieste, 34123, Italy

  • Ascendis Pharma Investigational Site

    RECRUITING

    Iași, 700106, Romania

  • Ascendis Pharma Investigational Site

    RECRUITING

    Târgu Mureş, 540136, Romania

  • Ascendis Pharma Investigational Site

    RECRUITING

    Sejong, 30099, South Korea

  • Ascendis Pharma Investigational Site

    RECRUITING

    Seongnam, 13620, South Korea

  • Ascendis Pharma Investigational Site

    RECRUITING

    Seoul, 03722, South Korea

  • Ascendis Pharma Investigational Site

    RECRUITING

    Seoul, 05278, South Korea

  • Ascendis Pharma Investigational Site

    RECRUITING

    Seoul, 05505, South Korea

  • Ascendis Pharma Investigational Site

    RECRUITING

    Seoul, 06273, South Korea

  • Ascendis Pharma Investigational Site

    RECRUITING

    Suwon, 16499, South Korea

  • Ascendis Pharma Investigational Site

    RECRUITING

    Barcelona, 8950, Spain

  • Ascendis Pharma Investigational Site

    RECRUITING

    Madrid, 28046, Spain

  • Ascendis Pharma Investigational Site

    RECRUITING

    Málaga, 29011, Spain

  • Ascendis Pharma Investigational Site

    RECRUITING

    Zaragoza, 50009, Spain

  • Ascendis PharmaInvestigational Site

    RECRUITING

    Le Kremlin-Bicêtre, 94270, France

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