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Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04035811
Recruitment Status : Recruiting
First Posted : July 29, 2019
Last Update Posted : April 8, 2022
Information provided by (Responsible Party):
QED Therapeutics, Inc.

Brief Summary:
This is a long-term, multi-center, observational study in children 2.5 to 10 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications and treatments of study participants. No study medication will be administered.

Condition or disease

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Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
Actual Study Start Date : August 12, 2019
Estimated Primary Completion Date : June 2026
Estimated Study Completion Date : June 2026

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. Annualized height velocity [ Time Frame: Up to 2 years ]

Secondary Outcome Measures :
  1. Collection of natural history of achondroplasia symptoms in children with achondroplasia [ Time Frame: Up to 2 years ]
  2. To characterize achondroplasia symptoms in children with achondroplasia [ Time Frame: Up to 2 years ]

Biospecimen Retention:   Samples With DNA
Collect blood for central assessment of blood biomarkers

Information from the National Library of Medicine

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Ages Eligible for Study:   30 Months to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Children with achondroplasia

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 10 years (inclusive) at study entry
  • Diagnosis of ACH
  • Ambulatory and able to stand without assistance
  • Study participants and parent(s) or LAR(s) are willing and able to comply with study visits and study procedures

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 received a C-type natriuretic peptide (CNP) analog or treatment targeting fibroblast growth factor receptor (FGFR) inhibition at any time
  • Have had regular long-term treatment (>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
  • Have used any other investigational product or investigational medical device for the treatment of ACH or short stature
  • Have had previous limb-lengthening surgery

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04035811

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Contact: QED Therapeutics 1-877-280-5655 PROPELstudyinfo@QEDTX.com

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Sponsors and Collaborators
QED Therapeutics, Inc.
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Study Director: QED Therapeutics VP, Clinical Development QED Therapeutics
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: QED Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04035811    
Other Study ID Numbers: QBGJ398-001
First Posted: July 29, 2019    Key Record Dates
Last Update Posted: April 8, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by QED Therapeutics, Inc.:
Skeletal dysplasia
Endochondral ossification
Shortened proximal limbs
Fibroblast growth factor receptor 3
Endochondral bone formation
Short-limb disproportionate dwarfism
Bone disease
Bone diseases
Musculoskeletal diseases
Genetic diseases
Additional relevant MeSH terms:
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Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn