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Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Q

QED Therapeutics

Status

Enrolling

Conditions

Achondroplasia

Study type

Observational

Funder types

Industry

Identifiers

NCT04035811
QBGJ398-001

Details and patient eligibility

About

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.

Enrollment

250 estimated patients

Sex

All

Ages

30 months to 17 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Key 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

Key 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)
  • Have had previous guided growth surgery or limb-lengthening surgery within 12 months prior to screening.

Trial contacts and locations

32

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Central trial contact

QED Therapeutics, Inc.

Data sourced from clinicaltrials.gov

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