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No treatment, just tracking: PROPEL study watches how kids with achondroplasia grow

NCT ID NCT04035811

Knowledge-focused Sponsor: QED Therapeutics, a BridgeBio company 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 followed 271 children with achondroplasia, ages 2.5 to 17, to understand their growth, medical issues, and quality of life over time. No medication or treatment was given. The goal was to gather natural history data to help design future clinical trials.

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.

Participants

271 people

The number who actually took part.

Started

Aug 2019

Finished

Aug 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.

Who is studied

Children with achondroplasia

Ages

30 months 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.

Key Inclusion Criteria: * Signed informed consent by study participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the study participant (when applicable) * Aged 2.5 to \<17 years at study entry * Diagnosis of ACH * Study participants and parent(s) or LAR(s) are willing and able to comply with study visits and study procedures Key Exclusion Criteria: * Have hypochondroplasia or short stature condition other than ACH (e.g. trisomy 21, pseudoachondroplasia, psychosocial short stature) * In females, having had their menarche * Height \< -2 or \> +2 standard deviations for age and sex based on reference tables on growth in children with ACH * Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening * Current evidence of corneal or retinal disorder/keratopathy * Current evidence of endocrine alterations of calcium/phosphorus homeostasis * Have a concurrent disease or condition that in the view of the Investigator and/or Sponsor, may impact growth or where the treatment is known to impact growth. * Significant abnormality in screening laboratory results. * Have been treated with growth hormone, insulin-like growth factor 1 (IGF 1), or anabolic steroids in the previous 6 months or long-term treatment (\>3 months) at any time * Have had regular long-term treatment (\>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable) * Have had previous guided growth surgery or limb-lengthening surgery within 12 months prior to screening.

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Conditions

The condition(s) this trial relates to.

achondroplasia bone disorder Dwarfism hereditary disease Mucopolysaccharidosis IV musculoskeletal system disorder osteochondrodysplasia

As listed by the trial registrant

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

Achondroplasia

Contacts and locations

Locations

  • Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

  • Birmingham Children's Hospital

    Birmingham, United Kingdom

  • Bristol Royal Hospital for Children

    Bristol, England, United Kingdom

  • Children's Hospital - London Health Sciences Center

    London, Ontario, N6A 5W9, Canada

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlino

    Milan, 20122, Italy

  • Haukeland Universitetssjukehus

    Bergen, 5009, Norway

  • Hopital Femme Mere Enfant

    Lyon, France

  • Hopital Necker-Enfants Malades

    Paris, France

  • Hopital des Enfants

    Toulouse, France

  • Hospital Universitario La Paz

    Madrid, 24086, Spain

  • Hospital Universitario Virgen de la Victoria

    Málaga, Spain

  • Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan

    Buenos Aires, Argentina

  • Johns Hopkins School of Medicine

    Baltimore, Maryland, 21211, United States

  • KK Women's and Children's Hospital

    Singapore, 229899, Singapore

  • Manchester University Children's Hospital

    Manchester, United Kingdom

  • Murdoch Children's Research Institute

    Parkville, Australia

  • Nemours Alfred I. Dupont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • Oslo Universitetssykehus

    Oslo, 0372, Norway

  • Otto-von-Guericke-University Magdeburg Medical Fakulty

    Magdeburg, 39120, Germany

  • Queen Elizabeth University Hospital

    Glasgow, United Kingdom

  • Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS

    Rome, 00168, Italy

  • Sheffield Children's Hospital

    Sheffield, United Kingdom

  • St. Thomas' Hospital

    London, United Kingdom

  • Stollery Children's Hospital

    Edmonton, Alberta, T6G 2H7, Canada

  • University of Missouri

    Columbia, Missouri, 65211, United States

  • University of Montreal

    Montreal, Quebec, H3C 3J7, Canada

  • University of Ottawa

    Ottawa, Ontario, K1N 6N5, Canada

  • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

    Madison, Wisconsin, 53705, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

  • Vithas Hospital San Jose

    Barcelona, 08305, Spain

More trials for these conditions

Other studies related to the condition(s) this trial covers.