Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL

QED Therapeutics, Inc., a Bridgebio company25 sites in 10 countries150 target enrollmentJune 5, 2024

ConditionsHypochondroplasia

Overview

Phase: N/A
Intervention: Not specified
Conditions: Hypochondroplasia
Sponsor: QED Therapeutics, Inc., a Bridgebio company
Enrollment: 150
Locations: 25
Primary Endpoint: Annualized height velocity
Status: Recruiting
Last Updated: 6 months ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).

Detailed Description

The objective is to evaluate growth, HCH-related medical complications, health-related quality of life, functional abilities and cognitive functions of study participants. Data collected will contribute to the characterization of the natural history of children with HCH. No study medication will be administered.

Registry

clinicaltrials.gov

Start Date

June 5, 2024

End Date

October 1, 2026

Last Updated

6 months ago

Study Type

Observational

Sex

All

Investigators

Sponsor

QED Therapeutics, Inc., a Bridgebio company

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Signed informed consent.
•Aged 2.5 to \<17 years at study entry.
•Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
•Participants are ambulatory and able to stand without assistance.
•Study participants and parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.

Exclusion Criteria

•Have ACH or short stature condition other than HCH.
•In females, having had their menarche. Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening.
•Having a clinically significant disease or condition that in view of the investigator or Sponsor will interfere with the evaluation of growth, with study participation or not be in the best interest of the participant.
•Clinically significant abnormality in any laboratory test result at screening
•Current evidence of corneal or retinal disorders.
•Have used any other investigational or approved product or medical device for the treatment of HCH or short stature for ≥ 30 days or with the last dose \<6 months before screening.
•Have had regular long-term treatment (\>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).
•Previous limb-lengthening surgery or guided growth surgery with plates still in place or removed within the 6 months prior to screening.
•Having had a fracture of the long bones or spine within 12 months of screening.
•History and/or current evidence of extensive ectopic tissue calcification.

Outcomes

Primary Outcomes

Annualized height velocity

Time Frame: up to 3 years

Secondary Outcomes

HCH-related surgical procedures(up to 3 years)
Cognitive functions(up to 3 years)
Change over time in height Z-score, upper arm to forearm ratio, and upper leg to lower leg ratio(up to 3 years)
HCH-related medical events reported as medical history or NT-AEs(up to 3 years)
Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form.(up to 3 years)