Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
Purpose
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.
Condition
- Achondroplasia
Eligibility
- Eligible Ages
- Between 30 Months and 17 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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 - 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.
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Case-Only
- Time Perspective
- Prospective
Recruiting Locations
Vanderbilt University Medical Center
Nashville, Tennessee 37232
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- QED Therapeutics, Inc.