Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

April 2, 2024 updated by: QED Therapeutics, Inc.

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

Recruiting

Conditions

Study Type

Observational

Enrollment (Estimated)

250

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 Locations

  • Argentina
      • Buenos Aires, Argentina
        • Recruiting
        • Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
  • Australia
      • Parkville, Australia
        • Recruiting
        • Murdoch Children'S Research Institute
        • Contact:
        • Principal Investigator:
          • Ravi Savarirayan, M.D.
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2H7
    • Ontario
      • London, Ontario, Canada, N6A 5W9
        • Recruiting
        • Children's Hospital - London Health Sciences Center
      • Ottawa, Ontario, Canada, K1N 6N5
        • Recruiting
        • University of Ottawa
    • Quebec
      • Montréal, Quebec, Canada, H3C 3J7
        • Recruiting
        • University of Montreal
  • France
      • Lyon, France
        • Recruiting
        • Hôpital Femme Mère Enfant
        • Contact:
        • Principal Investigator:
          • Marc Nicolino, M.D., Ph.D.
      • Paris, France
        • Recruiting
        • Hopital Necker-Enfants Malades
      • Toulouse, France
        • Recruiting
        • Hopital des Enfants
        • Contact:
        • Principal Investigator:
          • Jean Pierre Salles, M.D., Ph.D.
  • Germany
      • Magdeburg, Germany, 39120
        • Recruiting
        • Otto-von-Guericke-University Magdeburg Medical Fakulty
  • Italy
      • Rome, Italy, 00168
        • Recruiting
        • Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS
  • Norway
      • Bergen, Norway, 5009
        • Recruiting
        • Haukeland Universitetssjukehus
      • Oslo, Norway, 0372
        • Recruiting
        • Oslo Universitetssykehus
  • Singapore
      • Singapore, Singapore, 229899
        • Recruiting
        • KK Women's and Children's Hospital
  • Spain
      • Barcelona, Spain, 08305
        • Recruiting
        • Vithas Hospital San Jose
        • Principal Investigator:
          • Jorge Knörr, M.D.
        • Contact:
      • Madrid, Spain, 24086
        • Recruiting
        • Hospital Universitario La Paz
        • Contact:
        • Principal Investigator:
          • Fernando Santos Simarro, M.D.
      • Málaga, Spain
        • Recruiting
        • Hospital Universitario Virgen de la Victoria
        • Contact:
        • Principal Investigator:
          • Antonio Leiva Gea, M.D.
  • United Kingdom
      • Birmingham, United Kingdom
        • Recruiting
        • Birmingham Children's Hospital
        • Contact:
        • Principal Investigator:
          • Vrinda Saraff, MBBS
      • Glasgow, United Kingdom
        • Recruiting
        • Queen Elizabeth University Hospital
        • Contact:
        • Principal Investigator:
          • Helen McDevitt, MB ChB
      • London, United Kingdom
      • Manchester, United Kingdom
        • Recruiting
        • Manchester University Children's Hospital
      • Sheffield, United Kingdom
        • Recruiting
        • Sheffield Children's Hospital
        • Contact:
        • Principal Investigator:
          • Paul Arundel, MBBS
    • England
      • Bristol, England, United Kingdom
        • Recruiting
        • Bristol Royal Hospital for Children
        • Contact:
        • Principal Investigator:
          • Christine Burren, MBBS
  • United States
    • California
      • Oakland, California, United States, 94609
        • Recruiting
        • Benioff Children's Hospital Oakland
        • Contact:
        • Principal Investigator:
          • Paul Harmatz, M.D.
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • Children's Hospital Colorado
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Completed
        • Nemours Alfred I. Dupont Hospital for Children
    • Maryland
      • Baltimore, Maryland, United States, 21211
        • Recruiting
        • Johns Hopkins School of Medicine
        • Contact:
        • Principal Investigator:
          • Julie Hoover-Fong, MD
    • Missouri
      • Columbia, Missouri, United States, 65211
        • Recruiting
        • University of Missouri
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital
        • Contact:
        • Principal Investigator:
          • Howard Saal, M.D.
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt University Medical Center
        • Principal Investigator:
          • John Phillips, III, M.D.
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75207
        • Withdrawn
        • Cook's Children Medical Center
    • Wisconsin
      • Madison, Wisconsin, United States, 53705
        • Recruiting
        • 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

Ages Eligible for Study

1 year to 8 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
  • 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

Outcome Measure
Time Frame
Change from baseline in height Z score
Time Frame: Up to 2 years
Up to 2 years
Change from baseline in upper to lower body segment ratio
Time Frame: Up to 2 years
Up to 2 years

Secondary Outcome Measures

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

Outcome Measure
Time Frame
Change from baseline in other growth parameters (height Z score, body proportions, etc),
Time Frame: Up to 2 years
Up to 2 years
Bone biomarkers
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 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
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

Collaborators and Investigators

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

Sponsor

QED Therapeutics, Inc.

Investigators

  • 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)

August 12, 2019

Primary Completion (Estimated)

June 1, 2026

Study Completion (Estimated)

June 1, 2026

Study Registration Dates

First Submitted

July 20, 2019

First Submitted That Met QC Criteria

July 24, 2019

First Posted (Actual)

July 29, 2019

Study Record Updates

Last Update Posted (Actual)

April 4, 2024

Last Update Submitted That Met QC Criteria

April 2, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID 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

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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