Summary

Eligibility
for people ages 30 months to 10 years (full criteria)
Location
at Oakland, California and other locations
Dates
study started
estimated completion

Description

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.

Official Title

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

Keywords

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

Eligibility

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

Locations

  • Benioff Children's Hospital Oakland not yet accepting patients
    Oakland California 94609 United States
  • Vanderbilt University Medical Center not yet accepting patients
    Nashville Tennessee 37232 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
QED Therapeutics, Inc.
Links
Related Info
ID
NCT04035811
Study Type
Observational
Last Updated