Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Last updated: April 8, 2025
Sponsor: QED Therapeutics, Inc.
Overall Status: Active - Not Recruiting

Phase

N/A

Condition

Severe Short Stature

Treatment

N/A

Clinical Study ID

NCT04035811
QBGJ398-001
  • Ages 30-17
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Signed informed consent by study participant or parent(s) or legally authorizedrepresentative (LAR) and signed informed assent by the study participant (whenapplicable)

  • 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 studyvisits and study procedures

Exclusion

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 ongrowth in children with ACH

  • Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior toscreening

  • 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/orSponsor, 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), oranabolic steroids in the previous 6 months or long-term treatment (>3 months) at anytime

  • Have had regular long-term treatment (>1 month) with oral corticosteroids (low-doseongoing inhaled steroid for asthma is acceptable)

  • Have had previous guided growth surgery or limb-lengthening surgery within 12 monthsprior to screening.

Study Design

Total Participants: 271
Study Start date:
August 12, 2019
Estimated Completion Date:
June 30, 2026

Connect with a study center

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

    Buenos Aires,
    Argentina

    Site Not Available

  • Murdoch Children's Research Institute

    Parkville,
    Australia

    Site Not Available

  • Stollery Children's Hospital

    Edmonton, Alberta T6G 2H7
    Canada

    Site Not Available

  • Children's Hospital - London Health Sciences Center

    London, Ontario N6A 5W9
    Canada

    Site Not Available

  • University of Ottawa

    Ottawa, Ontario K1N 6N5
    Canada

    Site Not Available

  • SickKids - The Hospital for Sick Children

    Toronto, Ontario M5G1X8
    Canada

    Site Not Available

  • University of Montreal

    Montréal, Quebec H3C 3J7
    Canada

    Site Not Available

  • Hopital Femme Mere Enfant

    Lyon,
    France

    Site Not Available

  • Hopital Necker-Enfants Malades

    Paris,
    France

    Site Not Available

  • Hopital des Enfants

    Toulouse,
    France

    Site Not Available

  • Otto-von-Guericke-University Magdeburg Medical Fakulty

    Magdeburg, 39120
    Germany

    Site Not Available

  • Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlino

    Milan, 20122
    Italy

    Site Not Available

  • Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS

    Rome, 00168
    Italy

    Site Not Available

  • Haukeland Universitetssjukehus

    Bergen, 5009
    Norway

    Site Not Available

  • Oslo Universitetssykehus

    Oslo, 0372
    Norway

    Site Not Available

  • KK Women's and Children's Hospital

    Singapore, 229899
    Singapore

    Site Not Available

  • Vithas Hospital San Jose

    Barcelona, 08305
    Spain

    Site Not Available

  • Hospital Universitario La Paz

    Madrid, 24086
    Spain

    Site Not Available

  • Hospital Universitario Virgen de la Victoria

    Málaga,
    Spain

    Site Not Available

  • Bristol Royal Hospital for Children

    Bristol, England
    United Kingdom

    Site Not Available

  • Birmingham Children's Hospital

    Birmingham,
    United Kingdom

    Site Not Available

  • Queen Elizabeth University Hospital

    Glasgow,
    United Kingdom

    Site Not Available

  • St. Thomas' Hospital

    London,
    United Kingdom

    Site Not Available

  • Manchester University Children's Hospital

    Manchester,
    United Kingdom

    Site Not Available

  • Sheffield Children's Hospital

    Sheffield,
    United Kingdom

    Site Not Available

  • Benioff Children's Hospital Oakland

    Oakland, California 94609
    United States

    Site Not Available

  • Children's Hospital Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Nemours Alfred I. Dupont Hospital for Children

    Wilmington, Delaware 19803
    United States

    Site Not Available

  • Johns Hopkins School of Medicine

    Baltimore, Maryland 21211
    United States

    Site Not Available

  • University of Missouri

    Columbia, Missouri 65211
    United States

    Site Not Available

  • Cincinnati Children's Hospital

    Cincinnati, Ohio 45229
    United States

    Site Not Available

  • Vanderbilt University Medical Center

    Nashville, Tennessee 37232
    United States

    Site Not Available

  • Cook's Children Medical Center

    Dallas, Texas 75207
    United States

    Site Not Available

  • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

    Madison, Wisconsin 53705
    United States

    Site Not Available

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