for people ages 30 months to 10 years (full criteria)
at Oakland, California and other locations
study started
estimated completion
Principal Investigator
by Paul Harmatz, M.D.



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.

Official Title

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


Achondroplasia, Skeletal dysplasia, Endochondral ossification, ACH, Shortened proximal limbs, Fibroblast growth factor receptor 3, FGFR3, Endochondral bone formation, Short-limb disproportionate dwarfism, Bone disease, Dwarfism, Bone diseases, Musculoskeletal diseases, Osteochondrodysplasia, Genetic diseases, Inborn


You can join if…

Open to people ages 30 months to 10 years

  • 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

You CAN'T join if...

  • 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


  • Benioff Children's Hospital Oakland accepting new patients
    Oakland California 94609 United States
  • Stollery Children's Hospital accepting new patients
    Edmonton Alberta T6G 2H7 Canada

Lead Scientist at UCSF

  • Paul Harmatz, M.D.
    Professor, Pediatrics, School of Medicine. Authored (or co-authored) 211 research publications


accepting new patients
Start Date
Completion Date
QED Therapeutics, Inc.
Related Info
Study Type
Expecting 200 study participants
Last Updated