Status:
ACTIVE_NOT_RECRUITING
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
Lead Sponsor:
QED Therapeutics, a BridgeBio company
Conditions:
Achondroplasia
Eligibility:
All Genders
30-17 years
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 h...
Eligibility Criteria
Inclusion
- Key
- 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
Exclusion
- 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.
Key Trial Info
Start Date :
August 12 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 1 2026
Estimated Enrollment :
271 Patients enrolled
Trial Details
Trial ID
NCT04035811
Start Date
August 12 2019
End Date
June 1 2026
Last Update
April 11 2025
Active Locations (32)
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1
Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
3
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, United States, 19803
4
Johns Hopkins School of Medicine
Baltimore, Maryland, United States, 21211