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

QED Therapeutics, Inc.32 sites in 11 countries271 target enrollmentAugust 12, 2019

ConditionsAchondroplasia

Overview

Phase: N/A
Intervention: Not specified
Conditions: Achondroplasia
Sponsor: QED Therapeutics, Inc.
Enrollment: 271
Locations: 32
Primary Endpoint: Annualized height velocity (cm/year)
Status: Active, Not Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

August 12, 2019

End Date

June 2026

Last Updated

last year

Study Type

Observational

Sex

All

Investigators

Sponsor

QED Therapeutics, Inc.

Responsible Party

Sponsor

Eligibility Criteria

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 \<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

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
•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/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)