Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
- Conditions
- Achondroplasia
- Registration Number
- NCT04035811
- Lead Sponsor
- QED Therapeutics, Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 271
- 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
Key
- 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)
- Have had previous guided growth surgery or limb-lengthening surgery within 12 months prior to screening.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Annualized height velocity (cm/year) Up to 2 years
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (32)
Benioff Children's Hospital Oakland
🇺🇸Oakland, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Nemours Alfred I. Dupont Hospital for Children
🇺🇸Wilmington, Delaware, United States
Johns Hopkins School of Medicine
🇺🇸Baltimore, Maryland, United States
University of Missouri
🇺🇸Columbia, Missouri, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
🇺🇸Madison, Wisconsin, United States
Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
🇦🇷Buenos Aires, Argentina
Murdoch Children's Research Institute
🇦🇺Parkville, Australia
Scroll for more (22 remaining)Benioff Children's Hospital Oakland🇺🇸Oakland, California, United States