Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

March 30, 2026 updated by: QED Therapeutics, a BridgeBio company

Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial

This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
271

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Argentina
      • Buenos Aires, Argentina
        • Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
  • Australia
      • Parkville, Australia
        • Murdoch Children's Research Institute
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2H7
        • Stollery Children's Hospital
    • Ontario
      • London, Ontario, Canada, N6A 5W9
        • Children's Hospital - London Health Sciences Center
      • Ottawa, Ontario, Canada, K1N 6N5
        • University of Ottawa
    • Quebec
      • Montreal, Quebec, Canada, H3C 3J7
        • University of Montreal
  • France
      • Lyon, France
        • Hôpital Femme Mère Enfant
      • Paris, France
        • Hôpital Necker-Enfants Malades
      • Toulouse, France
        • Hopital Des Enfants
  • Germany
      • Magdeburg, Germany, 39120
        • Otto-von-Guericke-University Magdeburg Medical Fakulty
  • Italy
      • Milan, Italy, 20122
        • Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlino
      • Rome, Italy, 00168
        • Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS
  • Norway
      • Bergen, Norway, 5009
        • Haukeland Universitetssjukehus
      • Oslo, Norway, 0372
        • Oslo Universitetssykehus
  • Singapore
      • Singapore, Singapore, 229899
        • KK Women's and Children's Hospital
  • Spain
      • Barcelona, Spain, 08305
        • Vithas Hospital San Jose
      • Madrid, Spain, 24086
        • Hospital Universitario La Paz
      • Málaga, Spain
        • Hospital Universitario Virgen de la Victoria
  • United Kingdom
      • Birmingham, United Kingdom
        • Birmingham Children's Hospital
      • Glasgow, United Kingdom
        • Queen Elizabeth University Hospital
      • London, United Kingdom
        • St. Thomas' Hospital
      • Manchester, United Kingdom
        • Manchester University Children's Hospital
      • Sheffield, United Kingdom
        • Sheffield Children's Hospital
    • England
      • Bristol, England, United Kingdom
        • Bristol Royal Hospital for Children
  • United States
    • California
      • Oakland, California, United States, 94609
        • Benioff Children's Hospital Oakland
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Nemours Alfred I. Dupont Hospital for Children
    • Maryland
      • Baltimore, Maryland, United States, 21211
        • Johns Hopkins School of Medicine
    • Missouri
      • Columbia, Missouri, United States, 65211
        • University of Missouri
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center
    • Wisconsin
      • Madison, Wisconsin, United States, 53705
        • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 year to 6 years (Child)

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study Population

Children with achondroplasia

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Annualized height velocity (cm/year)
Time Frame: Up to 2 years
Up to 2 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Collection of natural history of achondroplasia symptoms in children with achondroplasia
Time Frame: Up to 2 years
Up to 2 years
To characterize achondroplasia symptoms in children with achondroplasia
Time Frame: Up to 2 years
Up to 2 years

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Time Frame
Change from baseline in height Z score
Time Frame: Up to 2 years
Up to 2 years
ACH-related NT-AEs
Time Frame: Up to 2 years
Up to 2 years
ACH-related surgical procedures
Time Frame: Up to 2 years
Up to 2 years
Changes in health-related quality of life as assessed by Pediatric Quality of Life Inventory (PedsQoL)
Time Frame: Up to 2 years
Up to 2 years
Changes in overall body pain as assessed by Numeric Rating Scale for pain (Pain-NRS)
Time Frame: Up to 2 years
Up to 2 years
Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)
Time Frame: Up to 2 years
Up to 2 years
Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test)
Time Frame: Up to 2 years
Up to 2 years
Change in attention assessed by age-appropriate computerized tests (Identification Test)
Time Frame: Up to 2 years
Up to 2 years
Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)
Time Frame: Up to 2 years
Up to 2 years
Change in working memory assessed by age-appropriate computerized tests (One Back Test)
Time Frame: Up to 2 years
Up to 2 years
Change from baseline in upper to lower body ratio (cm)
Time Frame: Up to 2 years
Up to 2 years
Change from baseline in upper arm to forearm ratio (cm)
Time Frame: Up to 2 years
Up to 2 years
Change from baseline in upper leg to lower leg ratio (cm)
Time Frame: Up to 2 years
Up to 2 years
Bone biomarkers (blood)
Time Frame: Up to 2 years
Up to 2 years
Changes in health-related quality of life as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)
Time Frame: Up to 2 years
Up to 2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
QED Therapeutics, a BridgeBio company

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: QED Therapeutics, Inc. VP, Clinical Development, QED Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 12, 2019

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 27, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 27, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 20, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 24, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 29, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 3, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 30, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • QBGJ398-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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