for people ages 30 months to 17 years (full criteria)
at Oakland, California and other locations
study started
completion around
Principal Investigator
by Paul Harmatz, M.D.
Headshot of Paul Harmatz
Paul Harmatz



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.

Official Title

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


Achondroplasia, Skeletal dysplasia, Endochondral ossification, Achondroplasia (ACH), Shortened proximal limbs, Fibroblast growth factor receptor 3, FGFR3, Endochondral bone formation, Short-limb disproportionate dwarfism, Quality of life in achondroplasia, Dwarfism, Bone diseases, Musculoskeletal diseases, Osteochondrodysplasia, Genetic diseases, Inborn, Functional abilities, Average growth velocity, Average height velocity, growth


You can join if…

Open to people ages 30 months to 17 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 <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

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 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.


  • Benioff Children's Hospital Oakland accepting new patients
    Oakland California 94609 United States
  • Children's Hospital Colorado accepting new patients
    Aurora Colorado 80045 United States

Lead Scientist at UCSF

  • Paul Harmatz, M.D.
    Dr. Paul Harmatz is a gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases (genetic disorders in which a lack of certain enzymes results in progressive damage to cells and organ systems). He leads a team of specialists who diagnose and care for patients with these rare diseases, offering therapies such as weekly enzyme infusion.


accepting new patients
Start Date
Completion Date
QED Therapeutics, Inc.
Related Info
Study Type
Expecting 250 study participants
Last Updated